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Clinical Trial Results:
An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.

Summary
EudraCT number	2015-001955-54
Trial protocol	NL DE BE ES
Global end of trial date	07 Sep 2016

Results information
Results version number	v1(current)
This version publication date	23 Mar 2017
First version publication date	23 Mar 2017
Other versions

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

Collapse all Expand all

Trial information

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Sponsor protocol code

BMN-051-302

ISRCTN number

-

US NCT number

NCT02636686

WHO universal trial number (UTN)

-

Other trial identifiers

Duchenne Muscular Dystrophy: DMD

Sponsor organisation name

BioMarin Pharmaceutical Inc.

Sponsor organisation address

105 Digital Drive, Novato, United States, CA 94949

Public contact

Clinical Trials Information, BioMarin Pharmaceutical Inc, clinicaltrials@bmrn.com

Scientific contact

Clinical Trials Information, BioMarin Pharmaceutical Inc, clinicaltrials@bmrn.com

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

No

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

No

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

No

Analysis stage

Interim

Date of interim/final analysis

17 Oct 2016

Is this the analysis of the primary completion data?

No

Global end of trial reached?

Yes

Global end of trial date

07 Sep 2016

Was the trial ended prematurely?

Yes

Main objective of the trial

To evaluate the long-term safety and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study.

Protection of trial subjects

Not applicable

Background therapy

-

Evidence for comparator

-

Actual start date of recruitment

15 Sep 2015

Long term follow-up planned

No

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Belgium: 4

Country: Number of subjects enrolled

France: 1

Country: Number of subjects enrolled

Germany: 3

Country: Number of subjects enrolled

Netherlands: 8

Country: Number of subjects enrolled

Spain: 1

Country: Number of subjects enrolled

United States: 2

Worldwide total number of subjects

19

EEA total number of subjects

17

Number of subjects enrolled per age group

In utero

0

Preterm newborn - gestational age < 37 wk

0

Newborns (0-27 days)

0

Infants and toddlers (28 days-23 months)

0

Children (2-11 years)

5

Adolescents (12-17 years)

11

Adults (18-64 years)

3

From 65 to 84 years

0

85 years and over

0

Subject disposition

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Recruitment details

-

Screening details

All subjects entering the treatment phase are required to provide written informed consent/assent (along with parental/guardian consent if applicable) and have confirmation that the inclusion and exclusion criteria have been evaluated by a medically qualified designee, including medical history and concurrent medication review.

Period 1 title

BMN051-302 (overall period)

Is this the baseline period?

Yes

Allocation method

Non-randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

6 mg/kg/wk SC

Arm description

6 mg/kg/wk SC

Arm type

Experimental

Investigational medicinal product name

drisapersen

Investigational medicinal product code

BMN051

Other name

GSK2402968, PRO051

Pharmaceutical forms

Solution for injection/infusion

Routes of administration

Intravenous use

Dosage and administration details

IV dosing of 3mg/kg/week over a 1 hour infusion time

3 mg/kg/wk IV

Arm description

3 mg/kg/wk IV

Arm type

Experimental

Investigational medicinal product name

drisapersen

Investigational medicinal product code

BMN051

Other name

GSK2402968, PRO051

Pharmaceutical forms

Solution for injection/infusion

Routes of administration

Intravenous use

Dosage and administration details

IV dosing of 3mg/kg/week over a 1 hour infusion time

Number of subjects in period 1	6 mg/kg/wk SC	3 mg/kg/wk IV
Started	11	8
Completed	0	0
Not completed	11	8
Consent withdrawn by subject	1	-
Death	1	-
Study Terminated by Sponsor	9	7
Missing	-	1

Baseline characteristics

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Reporting group title

6 mg/kg/wk SC

Reporting group description

6 mg/kg/wk SC

Reporting group title

3 mg/kg/wk IV

Reporting group description

3 mg/kg/wk IV

Reporting group values	6 mg/kg/wk SC	3 mg/kg/wk IV	Total
Number of subjects	11	8	19
Age categorical
Units: Subjects
9 - 11	4	1	5
12 - 17	4	7	11
>= 18	3	0	3
Age continuous
Units: Years
arithmetic mean (standard deviation)	13.8 ( 4.31 )	13.3 ( 1.98 )	-
Gender categorical
Units: Subjects
Female	0	0	0
Male	11	8	19
Ethnicity
Units: Subjects
Hispanic or Latino	0	1	1
Not Hispanic or Latino	10	7	17
Unknown or Not Reported	1	0	1

End points

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Reporting group title

6 mg/kg/wk SC

Reporting group description

6 mg/kg/wk SC

Reporting group title

3 mg/kg/wk IV

Reporting group description

3 mg/kg/wk IV

Primary: Safety

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End point title

Safety ^[1]

End point description

To evaluate the long-term safety and tolerability of subcutaneous or intravenous drisapersen in subjects with DMD correctable by drisapersen-induced DMD exon 51 skipping who have previously participated in an eligible study.

End point type

Primary

End point timeframe

Long term

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: Study is terminated early and only safety data is presented.

End point values	6 mg/kg/wk SC	3 mg/kg/wk IV
Number of subjects analysed	11	8
Units: Safety	0	0

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Study Period

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

18.1

Reporting group title

3 mg/kg/wk IV

Reporting group description

-

Reporting group title

6 mg/kg/wk SC

Reporting group description

-

Serious adverse events	3 mg/kg/wk IV	6 mg/kg/wk SC
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
number of deaths (all causes)	0	1
number of deaths resulting from adverse events
Congenital, familial and genetic disorders
Duchenne muscular dystrophy
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	3 mg/kg/wk IV	6 mg/kg/wk SC
Total subjects affected by non serious adverse events
subjects affected / exposed	6 / 8 (75.00%)	11 / 11 (100.00%)
Investigations
Complement factor C3 decreased
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	2	0
Urine protein/creatinine ratio increased
subjects affected / exposed	1 / 8 (12.50%)	1 / 11 (9.09%)
occurrences all number	4	1
Injury, poisoning and procedural complications
Fall
subjects affected / exposed	0 / 8 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	4
Lower limb fracture
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Tibia fracture
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Cardiac disorders
Arrhythmia
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Cyanosis
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Nervous system disorders
Dizziness
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Headache
subjects affected / exposed	1 / 8 (12.50%)	1 / 11 (9.09%)
occurrences all number	1	1
General disorders and administration site conditions
Application site hypersensitivity
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Chills
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Influenza like illness
subjects affected / exposed	1 / 8 (12.50%)	2 / 11 (18.18%)
occurrences all number	2	2
Injection site discolouration
subjects affected / exposed	0 / 8 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	3
Injection site dryness
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	4
Injection site erythema
subjects affected / exposed	0 / 8 (0.00%)	7 / 11 (63.64%)
occurrences all number	0	35
Injection site haematoma
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	2
Injection site haemorrhage
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Injection site hypoaesthesia
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Injection site induration
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	5
Injection site vesicles
subjects affected / exposed	0 / 8 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	5
Non-cardiac chest pain
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Pyrexia
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Eye disorders
Eye irritation
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Gastrointestinal disorders
Abdominal pain
subjects affected / exposed	2 / 8 (25.00%)	0 / 11 (0.00%)
occurrences all number	2	0
Nausea
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Respiratory, thoracic and mediastinal disorders
Cough
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Dyspnoea
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Hypoventilation
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Skin and subcutaneous tissue disorders
Acne
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Rash erythematous
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Skin hyperpigmentation
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Renal and urinary disorders
Proteinuria
subjects affected / exposed	3 / 8 (37.50%)	1 / 11 (9.09%)
occurrences all number	6	2
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Back pain
subjects affected / exposed	1 / 8 (12.50%)	1 / 11 (9.09%)
occurrences all number	1	1
Joint swelling
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Muscle contracture
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	2	0
Myalgia
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Pain in extremity
subjects affected / exposed	1 / 8 (12.50%)	1 / 11 (9.09%)
occurrences all number	2	1
Infections and infestations
Influenza
subjects affected / exposed	0 / 8 (0.00%)	1 / 11 (9.09%)
occurrences all number	0	1
Nasopharyngitis
subjects affected / exposed	0 / 8 (0.00%)	2 / 11 (18.18%)
occurrences all number	0	2
Rhinitis
subjects affected / exposed	1 / 8 (12.50%)	0 / 11 (0.00%)
occurrences all number	1	0
Upper respiratory tract infection
subjects affected / exposed	2 / 8 (25.00%)	0 / 11 (0.00%)
occurrences all number	2	0

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

06 Jul 2015

Protocol Amendment 1

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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