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Clinical Trial Results:
PhaseOut DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)

Summary
EudraCT number	2015-004333-27
Trial protocol	GB
Global end of trial date	11 Sep 2018

Results information
Results version number	v1
This version publication date	20 Nov 2018
First version publication date	25 Oct 2018
Other versions	v2

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

SMT C11005

ISRCTN number

US NCT number

NCT02858362

WHO universal trial number (UTN)

Sponsor organisation name

Summit (Oxford) Limited

Sponsor organisation address

136a Eastern Avenue, Milton Park, Abingdon, United Kingdom,

Public contact

Clinical Trial Information, Summit (Oxford) Limited, clinicaltrials@summitplc.com

Scientific contact

Clinical Trial Information, Summit (Oxford) Limited, clinicaltrials@summitplc.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Final

Date of interim/final analysis

11 Sep 2018

Is this the analysis of the primary completion data?

Yes

Primary completion date

11 Apr 2018

Global end of trial reached?

Yes

Global end of trial date

11 Sep 2018

Was the trial ended prematurely?

Yes

Main objective of the trial

To investigate changes in leg magnetic resonance imaging (MRI)/magnetic resonance spectroscopy (MRS) in paediatric patients with Duchenne Muscular Dystrophy (DMD), following treatment with SMT C1100 (Cohorts 1 and 2). To investigate the relationships between changes in leg MRI/MRS with plasma concentrations of SMT C1100 and its metabolites in paediatric patients with DMD, following treatment with SMT C1100 (Cohorts 1 and 2). To assess the safety and tolerability of SMT C1100 and its metabolites in paediatric patients with DMD.

Protection of trial subjects

The study was conducted in accordance with the Declaration of Helsinki, The International Council on Harmonisation of technical requirements for pharmaceuticals for human use (ICH) harmonized tripartite guideline regarding Good Clinical Practice (ICH-GCP E6 (R2) Consolidated Guidance, November 2016), all applicable subject privacy requirements and the ethical principles that are outlined in the Declaration of Helsinki (revised version of Fortaleza, Brazil, 2013). This includes but is not limited to: Independent IRB/EC review and approval of study protocol and any subsequent amendments, subject informed consent, and investigator reporting requirements. Prior to initiation of a study site, the Sponsor obtained approval from the appropriate regulatory agency to conduct the study in accordance with the ICH GCP and applicable country specific regulatory requirements. The study was conducted in accordance with all applicable regulatory requirements. The Investigator was to ensure that this protocol was conducted in full conformance with these principles or with the laws and regulations of the locality in which the research was conducted, whichever afforded the greater protection of the individual. Written informed consent and assent was obtained from each patient (and their parents/guardian) prior to participation in the study. Written informed consent was collected following a review of the patient information leaflet by the potential patient and their parents/guardian and a discussion between the subject and their parents/guardian and the Investigator or suitably qualified designee.

Background therapy

Evidence for comparator

Actual start date of recruitment

16 Jun 2016

Long term follow-up planned

Yes

Long term follow-up rationale

Safety, Efficacy, Ethical reason

Long term follow-up duration

3 Years

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

United Kingdom: 21

Country: Number of subjects enrolled

United States: 22

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Cohort 1 was conducted in the United Kingdom (UK) and the United States (US). Cohort 2 was conducted in the US. Cohort 3 was conducted in the UK.

Screening details

43 male patients aged between 5 and 12 years, with a diagnosis of Duchenne Muscular Dystrophy (confirmed by phenotypic and genetic evidence) were enrolled in Cohort 1, Cohort 2 or Cohort 3. Patients were enrolled to Cohort 3 if they had previously received SMT C1100, but were not eligible for Cohort 1 or Cohort 2.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Cohort 1: Microfluidised Oral Suspension F3

Arm description

Patients were to receive 2.5 g of SMT C1100 microfluidised aqueous oral suspension formulation (F3) twice-daily (BID) for 48 weeks.

Arm type

Experimental

Investigational medicinal product name

SMT C1100

Investigational medicinal product code

Ezutromid

Other name

Pharmaceutical forms

Oral suspension

Routes of administration

Oral use

Dosage and administration details

Patients received 2.5 g of SMT C1100 microfluidised aqueous suspension formulation (F3) BID for 48 weeks.

Cohort 2: Powder for Oral Suspension F6

Arm description

Patients were to receive 1 g of SMT C1100 as a powder for oral suspension (F6) BID for 48 weeks.

Arm type

Experimental

Investigational medicinal product name

SMT C1100

Investigational medicinal product code

Ezutromid

Other name

Pharmaceutical forms

Powder for oral suspension

Routes of administration

Oral use

Dosage and administration details

Patients received 1 g of SMT C1100 as a powder for oral suspension (F6) BID for 48 weeks.

Cohort 3: Microfluidised Oral Suspension F3

Arm description

Patients were to receive to receive 2.5 g of SMT C1100 microfluidised aqueous oral suspension formulation (F3) twice-daily (BID) for 48 weeks. All 3 patients in Cohort 3 discontinued from the study prior to Week 24 due to premature study termination.

Arm type

Experimental

Investigational medicinal product name

SMT C1100

Investigational medicinal product code

Ezutromid

Other name

Pharmaceutical forms

Oral suspension

Routes of administration

Oral use

Dosage and administration details

Patients were to receive 2.5 g of SMT C1100 microfluidised aqueous suspension formulation (F3) BID for 48 weeks.

Number of subjects in period 1	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6	Cohort 3: Microfluidised Oral Suspension F3
Started	30	10	3
Completed	29	9	0
Not completed	1	1	3
Consent withdrawn by subject	1	-	-
Discontinued due to study termination	-	-	3
Protocol deviation	-	1	-

Baseline characteristics

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Reporting group title

Cohort 1: Microfluidised Oral Suspension F3

Reporting group description

Patients were to receive 2.5 g of SMT C1100 microfluidised aqueous oral suspension formulation (F3) twice-daily (BID) for 48 weeks.

Reporting group title

Cohort 2: Powder for Oral Suspension F6

Reporting group description

Patients were to receive 1 g of SMT C1100 as a powder for oral suspension (F6) BID for 48 weeks.

Reporting group title

Cohort 3: Microfluidised Oral Suspension F3

Reporting group description

Reporting group values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6	Cohort 3: Microfluidised Oral Suspension F3	Total
Number of subjects	30	10	3	43
Age categorical
Units: Subjects
Children (2-11 years)	30	10	0	40
Adolescents (12-17 years)	0	0	3	3
Age continuous
Units: years
median (full range (min-max))	8.820 (5.22 to 10.02)	8.835 (6.82 to 10.10)	12.21 (11.27 to 12.56)	-
Gender categorical
Units: Subjects
Female	0	0	0	0
Male	30	10	3	43
Race
Units: Subjects
White	26	9	3	38
Asian	1	1	0	2
Other	3	0	0	3
Ethnicity
Units: Subjects
Hispanic or Latino	1	2	0	3
Not Hispanic or Latino	29	8	3	40

Subject analysis set title

Overall Open Label Arms

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1, Cohort 2 and Cohort 3.

Subject analysis set title

Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to week 24 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Change from baseline to Week 24 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 24 Baseline (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 24 utrophin intensity. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Observed Values (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 utrophin intensity. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Baseline (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Baseline for Week 24 percentage developmental myosin. Data from Week 24 and Week 48 are from different subjects.

Subject analysis set title

Week 24 Observed Values (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 percentage developmental myosin. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Baseline (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 24 fibre diameter. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Observed Value (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 fibre diameter. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Baseline (Forced Expiratory Volume in 1 Second [FEV1])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for FEV1.

Subject analysis set title

Week 12 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Change from baseline to Week 12 for FEV1.

Subject analysis set title

Week 24 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for FEV1.

Subject analysis set title

Week 36 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for FEV1.

Subject analysis set title

Week 48 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for FEV1.

Subject analysis set title

Baseline (Forced Vital Capacity [FVC])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for FVC.

Subject analysis set title

Week 12 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for FVC.

Subject analysis set title

Week 24 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for FVC.

Subject analysis set title

Week 36 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for FVC.

Subject analysis set title

Week 48 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for FVC.

Subject analysis set title

Baseline (Maximum Inspiratory Pressure [MIP])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MIP.

Subject analysis set title

Week 12 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MIP.

Subject analysis set title

Week 24 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MIP.

Subject analysis set title

Week 36 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MIP.

Subject analysis set title

Week 48 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MIP.

Subject analysis set title

Baseline (Maximum Expiratory Pressure [MEP])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MEP.

Subject analysis set title

Week 12 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MEP.

Subject analysis set title

Week 24 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MEP.

Subject analysis set title

Week 36 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MEP.

Subject analysis set title

Week 48 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MEP.

Subject analysis set title

Baseline (Peak Expiratory Flow [PEF])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for PEF.

Subject analysis set title

Week 12 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for PEF.

Subject analysis set title

Week 24 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for PEF.

Subject analysis set title

Week 36 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for PEF.

Subject analysis set title

Week 48 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for PEF.

Subject analysis set title

Cohort 1 and Cohort 2 Total

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2.

Subject analysis sets values	Overall Open Label Arms	Baseline (MRS FF Vastus Lateralis)	Week 12 Change from Baseline (MRS FF Vastus Lateralis)	Week 24 Change from Baseline (MRS FF Vastus Lateralis)	Week 36 Change from Baseline (MRS FF Vastus Lateralis)	Week 48 Change from Baseline (MRS FF Vastus Lateralis)	Baseline (MRS FF Soleus)	Week 12 Change from Baseline (MRS FF Soleus)	Week 24 Change from Baseline (MRS FF Soleus)	Week 36 Change from Baseline (MRS FF Soleus)	Week 48 Change from Baseline (MRS FF Soleus)	Baseline (MRS WTRT Vastus Lateralis)	Week 12 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 24 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 36 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 48 Change from Baseline (MRS WTRT Vastus Lateralis)	Baseline (MRS WTRT Soleus)	Week 12 Change from Baseline (MRS WTRT Soleus)	Week 24 Change from Baseline (MRS WTRT Soleus)	Week 36 Change from Baseline (MRS WTRT Soleus)	Week 48 Change from Baseline (MRS WTRT Soleus)	Week 24 Baseline (Utrophin Intensity)	Week 24 Observed Values (Utrophin Intensity)	Week 48 Baseline (Utrophin Intensity)	Week 48 Observed Values (Utrophin Intensity)	Week 24 Baseline (Percentage Developmental Myosin)	Week 24 Observed Values (Percentage Developmental Myosin)	Week 48 Baseline (Percentage Developmental Myosin)	Week 48 Observed Values (Percentage Developmental Myosin)	Week 24 Baseline (Fibre Diameter)	Week 24 Observed Value (Fibre Diameter)	Week 48 Baseline (Fibre Diameter)	Week 48 Observed Values (Fibre Diameter)	Baseline (Forced Expiratory Volume in 1 Second [FEV1])	Week 12 Change from Baseline (FEV1)	Week 24 Change from Baseline (FEV1)	Week 36 Change from Baseline (FEV1)	Week 48 Change from Baseline (FEV1)	Baseline (Forced Vital Capacity [FVC])	Week 12 Change from Baseline (FVC)	Week 24 Change from Baseline (FVC)	Week 36 Change from Baseline (FVC)	Week 48 Change from Baseline (FVC)	Baseline (Maximum Inspiratory Pressure [MIP])	Week 12 Change from Baseline (MIP)	Week 24 Change from Baseline (MIP)	Week 36 Change from Baseline (MIP)	Week 48 Change from Baseline (MIP)	Baseline (Maximum Expiratory Pressure [MEP])	Week 12 Change from Baseline (MEP)	Week 24 Change from Baseline (MEP)	Week 36 Change from Baseline (MEP)	Week 48 Change from Baseline (MEP)	Baseline (Peak Expiratory Flow [PEF])	Week 12 Change from Baseline (PEF)	Week 24 Change from Baseline (PEF)	Week 36 Change from Baseline (PEF)	Week 48 Change from Baseline (PEF)	Cohort 1 and Cohort 2 Total
Number of subjects	43	39	39	36	37	36	40	40	38	38	37	39	39	36	37	36	40	40	38	38	37	22	22	15	15	22	22	16	16	22	22	16	16	38	38	37	36	33	39	39	38	37	35	36	36	35	31	32	35	35	34	31	31	35	32	34	32	32	40
Age categorical
Units: Subjects
Children (2-11 years)	40	39	39	36	37	36	40	40	38	38	37	39	39	36	37	36	40	40	38	38	37	22	22	15	15	22	22	16	16	22	22	16	16	38	38	37	36	33	39	39	38	37	35	36	36	35	31	32	35	35	34	31	31	35	32	34	32	32	40
Adolescents (12-17 years)	3
Age continuous
Units: years
median (full range (min-max))	8.519 (1.2633 to )
Gender categorical
Units: Subjects
Female	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0	0
Male	40	39	39	36	37	36	40	40	38	38	37	39	39	36	37	36	40	40	38	38	37
Race
Units: Subjects
White	35
Asian	2
Other	3
Ethnicity
Units: Subjects
Hispanic or Latino	3
Not Hispanic or Latino	37

End points

Top of page

Reporting group title

Cohort 1: Microfluidised Oral Suspension F3

Reporting group description

Patients were to receive 2.5 g of SMT C1100 microfluidised aqueous oral suspension formulation (F3) twice-daily (BID) for 48 weeks.

Reporting group title

Cohort 2: Powder for Oral Suspension F6

Reporting group description

Patients were to receive 1 g of SMT C1100 as a powder for oral suspension (F6) BID for 48 weeks.

Reporting group title

Cohort 3: Microfluidised Oral Suspension F3

Reporting group description

Subject analysis set title

Overall Open Label Arms

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1, Cohort 2 and Cohort 3.

Subject analysis set title

Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS FF Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS FF vastus lateralis leg muscle parameter.

Subject analysis set title

Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS FF Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS FF soleus leg muscle parameter.

Subject analysis set title

Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to week 24 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS WTRT Vastus Lateralis)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS WTRT vastus lateralis leg muscle parameter.

Subject analysis set title

Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 12 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 24 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Change from baseline to Week 24 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 36 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 48 Change from Baseline (MRS WTRT Soleus)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MRS WTRT soleus leg muscle parameter.

Subject analysis set title

Week 24 Baseline (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 24 utrophin intensity. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Observed Values (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 utrophin intensity. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Utrophin Intensity)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Baseline (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Baseline for Week 24 percentage developmental myosin. Data from Week 24 and Week 48 are from different subjects.

Subject analysis set title

Week 24 Observed Values (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 percentage developmental myosin. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Percentage Developmental Myosin)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Baseline (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 24 fibre diameter. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 24 Observed Value (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 24 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Baseline (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for Week 48 fibre diameter. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Week 48 Observed Values (Fibre Diameter)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Observed values for subjects at Week 48 visit. Data from Week 24 and Week 48 are from different patients.

Subject analysis set title

Baseline (Forced Expiratory Volume in 1 Second [FEV1])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for FEV1.

Subject analysis set title

Week 12 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Change from baseline to Week 12 for FEV1.

Subject analysis set title

Week 24 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for FEV1.

Subject analysis set title

Week 36 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for FEV1.

Subject analysis set title

Week 48 Change from Baseline (FEV1)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for FEV1.

Subject analysis set title

Baseline (Forced Vital Capacity [FVC])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for FVC.

Subject analysis set title

Week 12 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for FVC.

Subject analysis set title

Week 24 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for FVC.

Subject analysis set title

Week 36 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for FVC.

Subject analysis set title

Week 48 Change from Baseline (FVC)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for FVC.

Subject analysis set title

Baseline (Maximum Inspiratory Pressure [MIP])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MIP.

Subject analysis set title

Week 12 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MIP.

Subject analysis set title

Week 24 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MIP.

Subject analysis set title

Week 36 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MIP.

Subject analysis set title

Week 48 Change from Baseline (MIP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MIP.

Subject analysis set title

Baseline (Maximum Expiratory Pressure [MEP])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for MEP.

Subject analysis set title

Week 12 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for MEP.

Subject analysis set title

Week 24 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for MEP.

Subject analysis set title

Week 36 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for MEP.

Subject analysis set title

Week 48 Change from Baseline (MEP)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for MEP.

Subject analysis set title

Baseline (Peak Expiratory Flow [PEF])

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Baseline for PEF.

Subject analysis set title

Week 12 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 12 for PEF.

Subject analysis set title

Week 24 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 24 for PEF.

Subject analysis set title

Week 36 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 36 for PEF.

Subject analysis set title

Week 48 Change from Baseline (PEF)

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2. Change from baseline to Week 48 for PEF.

Subject analysis set title

Cohort 1 and Cohort 2 Total

Subject analysis set type

Intention-to-treat

Subject analysis set description

Inclusive of Cohort 1 and Cohort 2.

Primary: MRS Fat Fraction (FF) Vastus Lateralis Leg Muscle Parameter

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End point title

MRS Fat Fraction (FF) Vastus Lateralis Leg Muscle Parameter ^[1]

End point description

Value of 99999 has been used as there is no confidence interval data for baseline measure. The standard deviation for the baseline measure is 13.3788.

End point type

Primary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.

End point values	Baseline (MRS FF Vastus Lateralis)	Week 12 Change from Baseline (MRS FF Vastus Lateralis)	Week 24 Change from Baseline (MRS FF Vastus Lateralis)	Week 36 Change from Baseline (MRS FF Vastus Lateralis)	Week 48 Change from Baseline (MRS FF Vastus Lateralis)
Number of subjects analysed	39	39	36	37	36
Units: percent
arithmetic mean (confidence interval 95%)	14.954 (-99999 to 99999)	1.779 (0.939 to 2.620)	3.914 (2.695 to 5.132)	5.238 (3.563 to 6.913)	7.142 (4.866 to 9.417)

No statistical analyses for this end point

Primary: MRS FF Soleus Leg Muscle Parameter

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End point title

MRS FF Soleus Leg Muscle Parameter ^[2]

End point description

Value of 99999 has been used as there is no confidence interval data for baseline measure. The standard deviation for baseline measure is 8.6310.

End point type

Primary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

Notes
[2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.

End point values	Baseline (MRS FF Soleus)	Week 12 Change from Baseline (MRS FF Soleus)	Week 24 Change from Baseline (MRS FF Soleus)	Week 36 Change from Baseline (MRS FF Soleus)	Week 48 Change from Baseline (MRS FF Soleus)
Number of subjects analysed	40	40	38	38	37
Units: percent
arithmetic mean (confidence interval 95%)	9.123 (-99999 to 99999)	0.615 (0.068 to 1.162)	1.108 (0.350 to 1.865)	2.384 (1.287 to 3.481)	2.584 (1.258 to 3.909)

No statistical analyses for this end point

Primary: MRS Water Transverse Relaxation Time (WTRT) Vastus Lateralis Leg Muscle Parameter

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End point title

MRS Water Transverse Relaxation Time (WTRT) Vastus Lateralis Leg Muscle Parameter ^[3]

End point description

Value of 99999 has been used as there is no confidence interval data for baseline measure. The standard deviation for the baseline measure is 1.9954.

End point type

Primary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

Notes
[3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint, which include the 95% confidence interval for the mean changes from baseline.

End point values	Baseline (MRS WTRT Vastus Lateralis)	Week 12 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 24 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 36 Change from Baseline (MRS WTRT Vastus Lateralis)	Week 48 Change from Baseline (MRS WTRT Vastus Lateralis)
Number of subjects analysed	39	39	36	37	36
Units: milliseconds
arithmetic mean (confidence interval 95%)	32.226 (-99999 to 99999)	-0.559 (-1.190 to 0.072)	-0.486 (-1.193 to 0.221)	-0.849 (-1.454 to -0.244)	-0.822 (-1.673 to 0.028)

No statistical analyses for this end point

Primary: MRS WTRT Soleus Leg Muscle Parameter

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End point title

MRS WTRT Soleus Leg Muscle Parameter ^[4]

End point description

Value of 99999 has been used as there is no confidence interval data for baseline measure. The standard deviation for baseline measure is 1.9235.

End point type

Primary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

Notes
[4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint, which include the 95% confidence interval for the mean changes from baseline.

End point values	Baseline (MRS WTRT Soleus)	Week 12 Change from Baseline (MRS WTRT Soleus)	Week 24 Change from Baseline (MRS WTRT Soleus)	Week 36 Change from Baseline (MRS WTRT Soleus)	Week 48 Change from Baseline (MRS WTRT Soleus)
Number of subjects analysed	40	40	38	38	37
Units: milliseconds
arithmetic mean (confidence interval 95%)	31.878 (-99999 to 99999)	-0.655 (-1.209 to -0.101)	-0.861 (-1.440 to -0.281)	-0.447 (-1.085 to 0.190)	-0.119 (-0.747 to 0.509)

No statistical analyses for this end point

Primary: Trough Concentration (Ctrough) Steady State Plasma Pharmacokinetic Parameter

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End point title

Trough Concentration (Ctrough) Steady State Plasma Pharmacokinetic Parameter ^[5] ^[6]

End point description

Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (geometric coefficient of variation)	17 ( 140 )	80 ( 83.1 )

No statistical analyses for this end point

Primary: Simulated Maximum Concentration (Cmax) Steady State Plasma Pharmacokinetic Parameter

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End point title

Simulated Maximum Concentration (Cmax) Steady State Plasma Pharmacokinetic Parameter ^[7] ^[8]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	135 (97 to 185)	415 (303 to 640)

No statistical analyses for this end point

Primary: Simulated Average Concentration (Cav) Steady State Plasma Pharmacokinetic Parameter

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End point title

Simulated Average Concentration (Cav) Steady State Plasma Pharmacokinetic Parameter ^[9] ^[10]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	54 (37 to 82)	163 (114 to 272)

No statistical analyses for this end point

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol I (DHD I)

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End point title

Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol I (DHD I) ^[11] ^[12]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[11] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (geometric coefficient of variation)	155 ( 61 )	365 ( 55 )

No statistical analyses for this end point

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD I

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End point title

Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD I ^[13] ^[14]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	1897 (1690 to 2158)	2829 (2597 to 3072)

No statistical analyses for this end point

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD I

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End point title

Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD I ^[15] ^[16]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[15] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[16] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	742 (685 to 836)	1109 (1028 to 1217)

No statistical analyses for this end point

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol III (DHD III)

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End point title

Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol III (DHD III) ^[17] ^[18]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[17] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[18] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (geometric coefficient of variation)	484 ( 67 )	1206 ( 68 )

No statistical analyses for this end point

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD III

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End point title

Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD III ^[19] ^[20]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[19] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[20] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	3162 (2392 to 4053)	4652 (3802 to 6116)

No statistical analyses for this end point

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD III

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End point title

Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD III ^[21] ^[22]

End point description

End point type

Primary

End point timeframe

Week 1 to Week 48

Notes
[21] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Descriptive statistics are presented for this endpoint.
[22] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: Summary includes data from Cohorts 1 and 2. Cohort 3 patients were a different study population (i.e. different eligibility criteria), and as only limited data was available, their data has been excluded.

End point values	Cohort 1: Microfluidised Oral Suspension F3	Cohort 2: Powder for Oral Suspension F6
Number of subjects analysed	30	10
Units: ng/mL
geometric mean (full range (min-max))	1359 (972 to 1790)	2211 (1707 to 3066)

No statistical analyses for this end point

Primary: Number of Patients Reporting One or More Treatment-Emergent Adverse Events (TEAEs)

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End point title

Number of Patients Reporting One or More Treatment-Emergent Adverse Events (TEAEs) ^[23]

End point description

Data provided includes up to week 48.

End point type

Primary

End point timeframe

Baseline to Week 48

Notes
[23] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: There is no statistical analysis associated with this endpoint, as this is a count of patients who experienced TEAEs.

End point values	Overall Open Label Arms
Number of subjects analysed	43
Units: Participants	43

No statistical analyses for this end point

Secondary: Utrophin Intensity by Time Point

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End point title

Utrophin Intensity by Time Point

End point description

Data entered for Weeks 24 and Week 48 are for different subjects.

End point type

Secondary

End point timeframe

Week 24 Baseline, Week 24, Week 48 Baseline, Week 48

End point values	Week 24 Baseline (Utrophin Intensity)	Week 24 Observed Values (Utrophin Intensity)	Week 48 Baseline (Utrophin Intensity)	Week 48 Observed Values (Utrophin Intensity)
Number of subjects analysed	22	22	15	15
Units: Arbitrary Units
arithmetic mean (standard deviation)	0.3686 ( 0.0553 )	0.3918 ( 0.0536 )	0.3520 ( 0.0357 )	0.3634 ( 0.0572 )

Week 24 Change from Baseline

Statistical analysis description

The number of subjects included in this statistical analysis was 23 and not 44. 22 subjects had evaluable data at both baseline and Week 24, and 1 subject only had evaluable data at baseline. The analysis used a mixed effect model.

Comparison groups

Week 24 Baseline (Utrophin Intensity) v Week 24 Observed Values (Utrophin Intensity)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

0.023

Confidence interval

level

95%

sides

2-sided

lower limit

-0.002

upper limit

0.048

Week 48 Change from Baseline

Statistical analysis description

The number of subjects included in this statistical analysis was 16 and not 30. 15 subjects had evaluable data at both baseline and Week 48, and 1 subject only had evaluable data at baseline. The analysis used a mixed effect model.

Comparison groups

Week 48 Baseline (Utrophin Intensity) v Week 48 Observed Values (Utrophin Intensity)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

0.006

Confidence interval

level

95%

sides

2-sided

lower limit

-0.03

upper limit

0.042

Secondary: Developmental Myosin by Time Point

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End point title

Developmental Myosin by Time Point

End point description

Data entered for Weeks 24 and Week 48 are for different subjects.

End point type

Secondary

End point timeframe

Week 24 Baseline, Week 24, Week 48 Baseline, Week 48

End point values	Week 24 Baseline (Percentage Developmental Myosin)	Week 24 Observed Values (Percentage Developmental Myosin)	Week 48 Baseline (Percentage Developmental Myosin)	Week 48 Observed Values (Percentage Developmental Myosin)
Number of subjects analysed	22	22	16	16
Units: Percent (%)
arithmetic mean (standard deviation)	11.1392 ( 3.3915 )	8.8226 ( 3.1082 )	12.7340 ( 3.9410 )	13.9588 ( 5.8819 )

Week 24 Change from Baseline

Statistical analysis description

The number of subjects included in this statistical analysis was 24 and not 44. 22 subjects had evaluable data at both baseline and Week 24, 1 subject only had evaluable data at baseline, and 1 subject only had evaluable data at Week 24. The analysis used a mixed effect model.

Comparison groups

Week 24 Observed Values (Percentage Developmental Myosin) v Week 24 Baseline (Percentage Developmental Myosin)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

-2.611

Confidence interval

level

95%

sides

2-sided

lower limit

-4.324

upper limit

-0.898

Week 48 Change from Baseline

Statistical analysis description

The number of subjects included in this statistical analysis was 16 and not 32. 16 subjects had evaluable data at both baseline and Week 48. The analysis used a mixed effect model.

Comparison groups

Week 48 Baseline (Percentage Developmental Myosin) v Week 48 Observed Values (Percentage Developmental Myosin)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

1.166

Confidence interval

level

95%

sides

2-sided

lower limit

-1.103

upper limit

3.435

Secondary: Fibre Diameter by Time Point

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End point title

Fibre Diameter by Time Point

End point description

Data for Week 24 and Week 48 are from different subjects.

End point type

Secondary

End point timeframe

Week 24 Baseline, Week 24, Week 48 Baseline, Week 48

End point values	Week 24 Baseline (Fibre Diameter)	Week 24 Observed Value (Fibre Diameter)	Week 48 Baseline (Fibre Diameter)	Week 48 Observed Values (Fibre Diameter)
Number of subjects analysed	22	22	16	16
Units: Micrometers (µm)
arithmetic mean (standard deviation)	42.2288 ( 6.0723 )	40.3083 ( 6.9697 )	44.7365 ( 4.7681 )	46.8001 ( 5.1765 )

Week 24 Change from Baseline

Statistical analysis description

Comparison groups

Week 24 Observed Value (Fibre Diameter) v Week 24 Baseline (Fibre Diameter)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

-1.837

Confidence interval

level

95%

sides

2-sided

lower limit

-3.989

upper limit

0.315

Week 48 Change from Baseline

Statistical analysis description

The number of subjects included in this statistical analysis was 16 and not 32. 16 subjects had evaluable data at both baseline and Week 48. The analysis used a mixed effect model.

Comparison groups

Week 48 Baseline (Fibre Diameter) v Week 48 Observed Values (Fibre Diameter)

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

Method

Parameter type

Difference in Least Square Mean

Point estimate

2.21

Confidence interval

level

95%

sides

2-sided

lower limit

0.096

upper limit

4.324

Secondary: Forced Expiratory Volume (FEV) in 1 Second by Time Point

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End point title

Forced Expiratory Volume (FEV) in 1 Second by Time Point

End point description

Summary includes data from Cohorts 1 and 2. No data was available for Cohort 3 from Weeks 12 to 48.

End point type

Secondary

End point timeframe

Baseline, Week, 12, Week 24, Week 36, Week 48

End point values	Baseline (Forced Expiratory Volume in 1 Second [FEV1])	Week 12 Change from Baseline (FEV1)	Week 24 Change from Baseline (FEV1)	Week 36 Change from Baseline (FEV1)	Week 48 Change from Baseline (FEV1)
Number of subjects analysed	38	38	37	36	33
Units: percent
arithmetic mean (standard deviation)	95.0 ( 23.18 )	-3.4 ( 20.86 )	-2.5 ( 24.88 )	-7.3 ( 24.26 )	2.0 ( 18.31 )

No statistical analyses for this end point

Secondary: Forced Vital Capacity (FVC) by Time Point

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End point title

Forced Vital Capacity (FVC) by Time Point

End point description

Summary includes data from Cohorts 1 and 2. No data was available for Cohort 3 from Weeks 12 to 48.

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

End point values	Baseline (Forced Vital Capacity [FVC])	Week 12 Change from Baseline (FVC)	Week 24 Change from Baseline (FVC)	Week 36 Change from Baseline (FVC)	Week 48 Change from Baseline (FVC)
Number of subjects analysed	39	39	38	37	35
Units: percent
arithmetic mean (standard deviation)	94.3 ( 19.52 )	-4.0 ( 16.31 )	1.1 ( 16.75 )	-3.4 ( 18.39 )	1.1 ( 16.29 )

No statistical analyses for this end point

Secondary: Maximum Inspiratory Pressure (MIP) by Time Point

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End point title

Maximum Inspiratory Pressure (MIP) by Time Point

End point description

Summary includes data from Cohorts 1 and 2. No data was available for Cohort 3 from Weeks 12 to 48.

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

End point values	Baseline (Maximum Inspiratory Pressure [MIP])	Week 12 Change from Baseline (MIP)	Week 24 Change from Baseline (MIP)	Week 36 Change from Baseline (MIP)	Week 48 Change from Baseline (MIP)
Number of subjects analysed	36	36	35	31	32
Units: cm H2O
arithmetic mean (standard deviation)	45.0 ( 20.23 )	3.6 ( 32.68 )	3.0 ( 25.00 )	9.0 ( 18.40 )	8.7 ( 20.30 )

No statistical analyses for this end point

Secondary: Peak Expiratory Flow (PEF) by Time Point

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End point title

Peak Expiratory Flow (PEF) by Time Point

End point description

Summary includes data from Cohorts 1 and 2. No data was available for Cohort 3 from Weeks 12 to 48.

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 24, Week 36, Week 48

End point values	Baseline (Peak Expiratory Flow [PEF])	Week 12 Change from Baseline (PEF)	Week 24 Change from Baseline (PEF)	Week 36 Change from Baseline (PEF)	Week 48 Change from Baseline (PEF)
Number of subjects analysed	35	32	34	32	32
Units: percent
arithmetic mean (standard deviation)	82.5 ( 29.64 )	0.5 ( 20.65 )	-0.1 ( 23.86 )	-0.4 ( 23.46 )	9.6 ( 30.09 )

No statistical analyses for this end point

Secondary: Number of Patients with a Change from Baseline for Vital Sign Measurements

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End point title

Number of Patients with a Change from Baseline for Vital Sign Measurements

End point description

Summary includes data from Cohorts 1 and 2. Only 1 Cohort 3 patient had measurements recorded after first day of dosing with study treatment, and all were within 20% of their baseline value, with the exception of one patient with vitals >20% for systolic blood pressure recorded post dose on Day 1.

End point type

Secondary

End point timeframe

Baseline to Week 48

End point values	Cohort 1 and Cohort 2 Total
Number of subjects analysed	40
Units: Participants
Systolic Blood Pressure (SBP): < 20% Change	25
SBP: >= 20% Reduction and < 20% Increase	2
SBP: >= 20% Increase and < 20% Reduction	13
SBP: >= 20% Reduction and >= 20% Increase	0
Diastolic Blood Pressure (DBP): < 20% Change	14
DBP: >= 20% Reduction and < 20% Increase	11
DBP: >= 20% Increase and < 20% Reduction	14
DBP: >= 20% Reduction and >= 20% Increase	1
Pulse: < 20% Change	19
Pulse: >= 20% Reduction and < 20% Increase	7
Pulse: >= 20% Increase and < 20% Reduction	13
Pulse: >= 20% Reduction and >= 20% Increase	1

No statistical analyses for this end point

Secondary: Number of Patients with a Change from Baseline for Echocardiogram Measurements

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End point title

Number of Patients with a Change from Baseline for Echocardiogram Measurements

End point description

Summary includes data from Cohorts 1 and 2. Data was not collected for Cohort 3.

End point type

Secondary

End point timeframe

Baseline to Week 48

End point values	Cohort 1 and Cohort 2 Total
Number of subjects analysed	40
Units: Participants
Baseline: Normal	38
Baseline: Abnormal, Not Clinically Significant	2
Baseline: Abnormal, Clinically Significant	0
Week 24: Normal	33
Week 24: Abnormal, Not Clinically Significant	5
Week 24: Abnormal, Clinically Significant	0
Week 24: Missing the Visit	2
Week 48: Normal	33
Week 48: Abnormal, Not Clinically Significant	2
Week 48: Abnormal, Clinically Significant	1
Week 48: Missing the Visit	4

No statistical analyses for this end point

Secondary: Number of Patients with Liver Function Test Results of Potential Clinical Concern

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End point title

Number of Patients with Liver Function Test Results of Potential Clinical Concern

End point description

Laboratory measurements for alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin (TB), alkaline phosphatase (ALP), and glutamate dehydrogenase (GLDH).

End point type

Secondary

End point timeframe

Baseline to End of Study

End point values	Overall Open Label Arms
Number of subjects analysed	43
Units: Participants
ALT >= ULN (Upper Limit of Normal)	43
ALT >= 2*ULN	43
ALT >= 3*ULN	42
AST >= ULN	43
AST >= 2*ULN	42
AST >= 3*ULN	42
TB >= ULN	0
ALP >= 1.5*ULN	0
GLDH >= ULN Excluding Hemolysed Samples	28
GLDH >= ULN Including Hemolysed Samples	31
GLDH >= 2.5*ULN Excluding Hemolysed Samples	4
GLDH >= 2.5*ULN Including Hemolysed Samples	4
Subjects Meeting Hy's Law	0

No statistical analyses for this end point

Secondary: Number of Patients with a Change from Baseline for 12-Lead Electrocardiogram Results

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End point title

Number of Patients with a Change from Baseline for 12-Lead Electrocardiogram Results

End point description

Summary includes data from Cohorts 1 and 2. Data was not collected for Cohort 3. Change from Baseline to Week 48 for maximum PR interval (MPRI), heart rate (HR), and maximum increase from baseline in QT interval, heart rate corrected using Fridericia’s formula (QTcF).

End point type

Secondary

End point timeframe

Baseline to Week 48

End point values	Cohort 1 and Cohort 2 Total
Number of subjects analysed	40
Units: Participants
MPRI: < 20% Change	35
MPRI: >= 20% Reduction and < 20% Increase	2
MPRI: >= Increase and < 20% Reduction	3
MPRI: >= 20% Reduction and >= 20% Increase	0
HR: < 20% Change	7
HR: >= 20% Reduction and < 20% Increase	6
HR: >= 20% Increase and < 20% Reduction	22
HR: >= 20% Reduction and >= 20% Increase	5
Maximum Increase from Baseline in QTcF: < 30 ms	32
Maximum Increase from Baseline in QTcF: 30 - 59 ms	8
Maximum Increase from Baseline in QTcF: >= 60 ms	0

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

The condition of each patient was monitored throughout the study. Treatment emergent adverse events (TEAEs [all causalities]) are presented in this record. All data to the end of the study have been included.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

19.0

Reporting group title

Open Label Treatment Arm

Reporting group description

Serious adverse events	Open Label Treatment Arm
Total subjects affected by serious adverse events
subjects affected / exposed	4 / 43 (9.30%)
number of deaths (all causes)	0
number of deaths resulting from adverse events	0
Injury, poisoning and procedural complications
Spinal compression fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Femur fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
General disorders and administration site conditions
Non-cardiac chest pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	1 / 1
deaths causally related to treatment / all	0 / 0
Peripheral swelling
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	1 / 1
deaths causally related to treatment / all	0 / 0
Vomiting
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Renal and urinary disorders
Chromaturia
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Musculoskeletal and connective tissue disorders
Muscle spasms
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Rhabdomyolysis
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Infections and infestations
Viral infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Tonsillitis bacterial
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0
Gastritis viral
subjects affected / exposed	1 / 43 (2.33%)
occurrences causally related to treatment / all	0 / 1
deaths causally related to treatment / all	0 / 0

Frequency threshold for reporting non-serious adverse events: 0%

Non-serious adverse events	Open Label Treatment Arm
Total subjects affected by non serious adverse events
subjects affected / exposed	42 / 43 (97.67%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Eye haemangioma
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Melanocytic naevus
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Skin papilloma
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Vascular disorders
Hypertension
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Hypotension
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Pallor
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Surgical and medical procedures
Eyeglasses therapy
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
General disorders and administration site conditions
Catheter site bruise
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Chest pain
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Facial pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Fatigue
subjects affected / exposed	5 / 43 (11.63%)
occurrences all number	5
Feeling hot
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Impaired healing
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Malaise
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Pyrexia
subjects affected / exposed	8 / 43 (18.60%)
occurrences all number	12
Thirst
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Unevaluable event
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Vessel puncture site pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Gait disturbance
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Immune system disorders
Multiple allergies
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	12 / 43 (27.91%)
occurrences all number	15
Dyspnoea
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Epistaxis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	5
Nasal congestion
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Oropharyngeal pain
subjects affected / exposed	10 / 43 (23.26%)
occurrences all number	10
Pharyngeal erythema
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Rhinorrhoea
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	4
Sinus congestion
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Throat irritation
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Wheezing
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Psychiatric disorders
Abnormal behaviour
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	3
Aggression
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Anger
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Anxiety
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Asocial behaviour
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Belligerence
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Enuresis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Initial insomnia
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Irritability
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Mood altered
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Sleep disorder
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Depressed mood
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Investigations
Aspartate aminotransferase increased
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Blood urea increased
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Gamma-glutamyltransferase increased
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Glutamate dehydrogenase increased
subjects affected / exposed	4 / 43 (9.30%)
occurrences all number	4
Protein urine present
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Thyroxine increased
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Weight increased
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Weight decreased
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Injury, poisoning and procedural complications
Contusion
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	5
Ear injury
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Fall
subjects affected / exposed	8 / 43 (18.60%)
occurrences all number	16
Foot fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Head injury
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Humerus fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Joint injury
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Ligament sprain
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Limb injury
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Post procedural contusion
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Procedural nausea
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Procedural pain
subjects affected / exposed	7 / 43 (16.28%)
occurrences all number	7
Skin abrasion
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Spinal fracture
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Torus fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Spinal compression fracture
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Scratch
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Thermal burn
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Congenital, familial and genetic disorders
Cryptorchism
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Nervous system disorders
Disturbance in attention
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Dizziness
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Headache
subjects affected / exposed	11 / 43 (25.58%)
occurrences all number	27
Lethargy
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Sensory processing disorder
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Ear and labyrinth disorders
Ear pain
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Excessive cerumen production
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Motion sickness
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Tinnitus
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Vertigo
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Eye disorders
Astigmatism
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Cataract
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Chalazion
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Dry eye
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Erythema of eyelid
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Eye pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Eye inflammation
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Hypermetropia
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Scleral disorder
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Vitreous floaters
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Gastrointestinal disorders
Abdominal discomfort
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	4
Abdominal distension
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Abdominal pain upper
subjects affected / exposed	12 / 43 (27.91%)
occurrences all number	24
Constipation
subjects affected / exposed	4 / 43 (9.30%)
occurrences all number	4
Dental plaque
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Diarrhoea
subjects affected / exposed	18 / 43 (41.86%)
occurrences all number	21
Dyspepsia
subjects affected / exposed	4 / 43 (9.30%)
occurrences all number	4
Faeces discoloured
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Faeces pale
subjects affected / exposed	24 / 43 (55.81%)
occurrences all number	29
Frequent bowel movements
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Gastritis
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Gingival recession
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Haemorrhoids
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Lip swelling
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	4
Nausea
subjects affected / exposed	7 / 43 (16.28%)
occurrences all number	13
Oral mucosal eruption
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Rectal haemorrhage
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Rectal prolapse
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Toothache
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Abdominal pain
subjects affected / exposed	7 / 43 (16.28%)
occurrences all number	11
Vomiting
subjects affected / exposed	23 / 43 (53.49%)
occurrences all number	79
Gastric hypomotility
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Hypoaesthesia oral
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Skin and subcutaneous tissue disorders
Dry skin
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Eczema
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Erythema
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Ingrowing nail
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Pruritus
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Rash
subjects affected / exposed	5 / 43 (11.63%)
occurrences all number	17
Rash pruritic
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Rash maculo-papular
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Skin discolouration
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Swelling face
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Urticaria
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Renal and urinary disorders
Micturition urgency
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Chromaturia
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Pollakiuria
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Endocrine disorders
Cushingoid
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Growth hormone deficiency
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	5 / 43 (11.63%)
occurrences all number	5
Back pain
subjects affected / exposed	10 / 43 (23.26%)
occurrences all number	14
Flank pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Musculoskeletal chest pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Musculoskeletal discomfort
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Musculoskeletal stiffness
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Musculoskeletal pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Myalgia
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Neck pain
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Pain in extremity
subjects affected / exposed	8 / 43 (18.60%)
occurrences all number	12
Spinal pain
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Muscle spasms
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Infections and infestations
Campylobacter gastroenteritis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Cellulitis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Cystitis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Ear infection
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Eye infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Gastroenteritis
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Gastrointestinal viral infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Gingival abscess
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Impetigo
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Localised infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Lower respiratory tract infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Molluscum contagiosum
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Nail infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Nasopharyngitis
subjects affected / exposed	11 / 43 (25.58%)
occurrences all number	14
Otitis media
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Paronychia
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	2
Pharyngitis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Pharyngitis streptococcal
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Post procedural infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Rhinitis
subjects affected / exposed	4 / 43 (9.30%)
occurrences all number	6
Sinusitis
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Upper respiratory tract infection
subjects affected / exposed	4 / 43 (9.30%)
occurrences all number	6
Urinary tract infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Viral upper respiratory tract infection
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2
Gastroenteritis viral
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Adenovirus infection
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Conjunctivitis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Oral candidiasis
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Metabolism and nutrition disorders
Decreased appetite
subjects affected / exposed	3 / 43 (6.98%)
occurrences all number	3
Increased appetite
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	3
Overweight
subjects affected / exposed	1 / 43 (2.33%)
occurrences all number	1
Vitamin D deficiency
subjects affected / exposed	2 / 43 (4.65%)
occurrences all number	2

More information

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Were there any global substantial amendments to the protocol? Yes

08 Dec 2015

Amendment 1 (dated 08 December 2015) added additional information on potential interactions of ezutromid with the Cytochrome P-450 pathway, particularly CYP2B6, and introduced the prohibition of taking medications known to be Cytochrome P450 inhibitors, inducers, and substrates during the study.

07 Oct 2016

Amendment 3 (dated 07 October 2016) added Cohort 2 (10 subjects) to study a microfluidized aqueous oral suspension at a dose of 1000 mg twice daily. Cohort 2 subjects were only enrolled at US sites. The number of subjects in the original cohort was correspondingly reduced from 40 to 30. The amendment also provided additional information on Summit Study ezutromid, in which the suspension formulation was previously tested, and made various minor administrative changes.

24 Feb 2017

Amendment 4 (24 February 2017) added Cohort 3 (15 subjects), which consisted of patients who had previously received ezutromid and were not eligible for Cohorts 1 or 2. These patients entered a safety arm and underwent additional cardiac MRI scans and pulmonary function tests. Cohort 3 patients were only enrolled at UK sites. The amendment also added the Extension Phase into the study design. All patients were eligible to continue into the Extension Phase. The amendment also made various minor administrative changes.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results: PhaseOut DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: MRS Fat Fraction (FF) Vastus Lateralis Leg Muscle Parameter

Primary: MRS Fat Fraction (FF) Vastus Lateralis Leg Muscle Parameter

Primary: MRS FF Soleus Leg Muscle Parameter

Primary: MRS FF Soleus Leg Muscle Parameter

Primary: MRS Water Transverse Relaxation Time (WTRT) Vastus Lateralis Leg Muscle Parameter

Primary: MRS Water Transverse Relaxation Time (WTRT) Vastus Lateralis Leg Muscle Parameter

Primary: MRS WTRT Soleus Leg Muscle Parameter

Primary: MRS WTRT Soleus Leg Muscle Parameter

Primary: Trough Concentration (Ctrough) Steady State Plasma Pharmacokinetic Parameter

Primary: Trough Concentration (Ctrough) Steady State Plasma Pharmacokinetic Parameter

Primary: Simulated Maximum Concentration (Cmax) Steady State Plasma Pharmacokinetic Parameter

Primary: Simulated Maximum Concentration (Cmax) Steady State Plasma Pharmacokinetic Parameter

Primary: Simulated Average Concentration (Cav) Steady State Plasma Pharmacokinetic Parameter

Primary: Simulated Average Concentration (Cav) Steady State Plasma Pharmacokinetic Parameter

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol I (DHD I)

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol I (DHD I)

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD I

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD I

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD I

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD I

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol III (DHD III)

Primary: Ctrough Steady State Plasma Pharmacokinetic Parameter for Dihydrodiol III (DHD III)

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD III

Primary: Simulated Cmax Steady State Plasma Pharmacokinetic Parameter for DHD III

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD III

Primary: Simulated Cav Steady State Plasma Pharmacokinetic Parameter for DHD III

Primary: Number of Patients Reporting One or More Treatment-Emergent Adverse Events (TEAEs)

Primary: Number of Patients Reporting One or More Treatment-Emergent Adverse Events (TEAEs)

Secondary: Utrophin Intensity by Time Point

Secondary: Utrophin Intensity by Time Point

Secondary: Developmental Myosin by Time Point

Secondary: Developmental Myosin by Time Point

Secondary: Fibre Diameter by Time Point

Secondary: Fibre Diameter by Time Point

Secondary: Forced Expiratory Volume (FEV) in 1 Second by Time Point

Secondary: Forced Expiratory Volume (FEV) in 1 Second by Time Point

Secondary: Forced Vital Capacity (FVC) by Time Point

Secondary: Forced Vital Capacity (FVC) by Time Point

Secondary: Maximum Inspiratory Pressure (MIP) by Time Point

Secondary: Maximum Inspiratory Pressure (MIP) by Time Point

Secondary: Peak Expiratory Flow (PEF) by Time Point

Secondary: Peak Expiratory Flow (PEF) by Time Point

Secondary: Number of Patients with a Change from Baseline for Vital Sign Measurements

Secondary: Number of Patients with a Change from Baseline for Vital Sign Measurements

Secondary: Number of Patients with a Change from Baseline for Echocardiogram Measurements

Secondary: Number of Patients with a Change from Baseline for Echocardiogram Measurements

Secondary: Number of Patients with Liver Function Test Results of Potential Clinical Concern

Secondary: Number of Patients with Liver Function Test Results of Potential Clinical Concern

Secondary: Number of Patients with a Change from Baseline for 12-Lead Electrocardiogram Results

Secondary: Number of Patients with a Change from Baseline for 12-Lead Electrocardiogram Results

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
PhaseOut DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)