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Clinical Trial Results:
A randomized, open-label, multicenter, phase 2 clinical trial to explore the safety and efficacy of sepranolone in pediatric and adult patients with Tourette Syndrome.

Summary
EudraCT number	2021-001045-12
Trial protocol	DK
Global end of trial date	01 Feb 2023

Results information
Results version number	v1(current)
This version publication date	07 Jul 2023
First version publication date	07 Jul 2023
Other versions

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

APH205

ISRCTN number

US NCT number

NCT05434546

WHO universal trial number (UTN)

Sponsor organisation name

Asarina Pharma ApS

Sponsor organisation address

Ole Maaloes vej 3 , Copenhagen, Denmark, 2200

Public contact

Peter Nordkild, Asarina Pharma ApS, +45 2547 1646, peter.nordkild@asarinapharma.com

Scientific contact

Peter Nordkild, Asarina Pharma ApS, +45 2547 1646, peter.nordkild@asarinapharma.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?

Analysis stage

Final

Date of interim/final analysis

25 May 2023

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

01 Feb 2023

Was the trial ended prematurely?

Main objective of the trial

To investigate the efficacy of sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks

Protection of trial subjects

Subjects taking part in this clinical study were insured by the Sponsor against any injury caused by the clinical study, in accordance with the local regulatory requirements. A copy of the insurance certificate was provided to each Investigator and was filed in the investigator’s file at the sites and in the clinical trial’s Trial Master File (TMF). The Investigator had to notify the Sponsor immediately upon notice of any claims or lawsuits brought by the patients or their relatives.

Background therapy

Patient's standard of care Tourette treatment for 12 weeks.

Evidence for comparator

Actual start date of recruitment

16 Feb 2022

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Denmark: 26

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

4 pediatric patients and 24 adults were screened. All screened patients were randomized except one pediatric patient and one adult who did not fulfill inclusion criteria. Among the pediatric patients, two were randomized to the Sepranolone group and one to the SoC group, while 15 adults were randomized to Sepranolone group and 8 to the SoC group.

Screening details

All subjects who signed the informed consent were enrolled in the study and all clinical data related to these subjects were documented.

Number of subjects started

Number of subjects completed

Period 1 title

Overall study (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Sepranolone + Standard of Care (SoC)

Arm description

Sepranolone 10 mg subcutaneously twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment.

Arm type

Experimental

Investigational medicinal product name

Sepranolone

Investigational medicinal product code

Other name

Pharmaceutical forms

Injection

Routes of administration

Subcutaneous use

Dosage and administration details

Sepranolone 10 mg sc twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment.

Standard of Care (SoC)

Arm description

Continuation of the patient's standard of care Tourette treatment for 12 weeks.

Arm type

No intervention

Investigational medicinal product name

No investigational medicinal product assigned in this arm

Number of subjects in period 1	Sepranolone + Standard of Care (SoC)	Standard of Care (SoC)
Started	17	9
Completed	16	8
Not completed	1	1
Consent withdrawn by subject	-	1
Adverse event, non-fatal	1	-

Baseline characteristics

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Reporting group title

Sepranolone + Standard of Care (SoC)

Reporting group description

Sepranolone 10 mg subcutaneously twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment.

Reporting group title

Standard of Care (SoC)

Reporting group description

Continuation of the patient's standard of care Tourette treatment for 12 weeks.

Reporting group values	Sepranolone + Standard of Care (SoC)	Standard of Care (SoC)	Total
Number of subjects	17	9	26
Age categorical
Units: Subjects
In utero	0	0	0
Preterm newborn infants (gestational age < 37 wks)	0	0	0
Newborns (0-27 days)	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0
Children (2-11 years)	0	0	0
Adolescents (12-17 years)	2	1	3
Adults (18-64 years)	15	8	23
From 65-84 years	0	0	0
85 years and over	0	0	0
Age continuous
Units: years
arithmetic mean (standard deviation)	35 ( 8.3 )	33 ( 10 )	-
Gender categorical
All randomized pediatric patients (3/3) were male whereas 5/15 (33.3%) of the adults in the Sepranolone group and 3/8 (37.5%) in the SoC group were female.
Units: Subjects
Female	5	3	8
Male	12	6	18

Subject analysis set title

Sepranolone + SoC. Full analysis set (FAS)

Subject analysis set type

Full analysis

Subject analysis set description

Sepranolone Full analysis set (FAS) consisted of all patients in the Sepranolone group who were randomized into the study. The FAS followed the intention-to-treat (ITT) principle, i.e., patients were analyzed in the treatment group to which they were randomized, regardless of whether treatment was received as planned.

Subject analysis set title

Sepranolone + SoC. mITT analysis set

Subject analysis set type

Modified intention-to-treat

Subject analysis set description

Modified intention-to-treat (mITT) analysis set included patients in the FAS who had a valid baseline assessment and had taken at least 6 doses of study drug per 4-week period. The mITT was the primary analysis set for efficacy analyses.

Subject analysis set title

Sepranolone + SoC. Safety analysis set (SAS)

Subject analysis set type

Safety analysis

Subject analysis set description

SAS consisted of all patients who received at least one (1) dose of Sepranolone. Patients were analyzed according to the treatment they actually received. The SAS was the primary analysis set for safety analyses.

Subject analysis set title

SoC. Full analysis set (FAS)

Subject analysis set type

Full analysis

Subject analysis set description

FAS consisted of all patients who were randomized into the study. The FAS followed the intention-to-treat (ITT) principle, i.e., patients were analyzed in the treatment group to which they were randomized, regardless of whether treatment was received as planned.

Subject analysis set title

SoC. mITT analysis set

Subject analysis set type

Modified intention-to-treat

Subject analysis set description

mITT analysis set included patients in the FAS who had a valid baseline assessment and had taken at least 6 doses of study drug per 4-week period. The mITT was the primary analysis set for efficacy analyses.

Subject analysis set title

SoC. Safety analysis set (SAS)

Subject analysis set type

Safety analysis

Subject analysis set description

SAS for the SoC group consisted of all patients randomized to the SoC group. Patients were analyzed according to the treatment they actually received. The SAS was the primary analysis set for safety analyses.

Subject analysis sets values	Sepranolone + SoC. Full analysis set (FAS)	Sepranolone + SoC. mITT analysis set	Sepranolone + SoC. Safety analysis set (SAS)	SoC. Full analysis set (FAS)	SoC. mITT analysis set	SoC. Safety analysis set (SAS)
Number of subjects	17	17	17	9	9	9
Age categorical
Units: Subjects
In utero	0	0	0	0	0	0
Preterm newborn infants (gestational age < 37 wks)	0	0	0	0	0	0
Newborns (0-27 days)	0	0	0	0	0	0
Infants and toddlers (28 days-23 months)	0	0	0	0	0	0
Children (2-11 years)	0	0	0	0	0	0
Adolescents (12-17 years)	2	2	2	1	1	1
Adults (18-64 years)	15	15	15	8	8	8
From 65-84 years	0	0	0	0	0	0
85 years and over	0	0	0	0	0	0
Age continuous
Units: years
arithmetic mean (standard deviation)	( )	( )	( )	( )	( )	( )
Gender categorical
All randomized pediatric patients (3/3) were male whereas 5/15 (33.3%) of the adults in the Sepranolone group and 3/8 (37.5%) in the SoC group were female.
Units: Subjects
Female
Male

End points

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Reporting group title

Sepranolone + Standard of Care (SoC)

Reporting group description

Sepranolone 10 mg subcutaneously twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment.

Reporting group title

Standard of Care (SoC)

Reporting group description

Continuation of the patient's standard of care Tourette treatment for 12 weeks.

Subject analysis set title

Sepranolone + SoC. Full analysis set (FAS)

Subject analysis set type

Full analysis

Subject analysis set description

Subject analysis set title

Sepranolone + SoC. mITT analysis set

Subject analysis set type

Modified intention-to-treat

Subject analysis set description

Subject analysis set title

Sepranolone + SoC. Safety analysis set (SAS)

Subject analysis set type

Safety analysis

Subject analysis set description

Subject analysis set title

SoC. Full analysis set (FAS)

Subject analysis set type

Full analysis

Subject analysis set description

Subject analysis set title

SoC. mITT analysis set

Subject analysis set type

Modified intention-to-treat

Subject analysis set description

Subject analysis set title

SoC. Safety analysis set (SAS)

Subject analysis set type

Safety analysis

Subject analysis set description

Primary: Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

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End point title

Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

End point description

The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semi-structured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The primary endpoint is the total tic score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.

End point type

Primary

End point timeframe

Change from baseline at week 4, 8 and 12.

End point values	Sepranolone + SoC. mITT analysis set	SoC. mITT analysis set
Number of subjects analysed	17	9
Units: Change in total score from baseline
least squares mean (confidence interval 90%)	-8.57 (-11.22 to -5.92)	-3.94 (-7.76 to -0.11)

Change from baseline in YGTSS total tic score

Statistical analysis description

The change from baseline in Yale Global Tic Severity Scale (YGTSS) total tic score was modelled using mixed model repeated measures (MMRM) analysis with treatment group, visit and treatment by visit interaction as fixed effects, patient as random effect and baseline YGTSS total tic score as covariate. In this model, the statistical hypothesis for the primary objective is evaluated using a one-sided t-test at significance level 5%.

Comparison groups

Sepranolone + SoC. mITT analysis set v SoC. mITT analysis set

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.051

Method

t-test, 1-sided

Parameter type

Mean difference (net)

Point estimate

-4.63

Confidence interval

level

90%

sides

2-sided

lower limit

-9.31

upper limit

0.04

Secondary: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

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End point title

Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

End point description

Collection of adverse events (AEs) including spontaneous reporting, number of subjects with clinically significant changes in clinical safety laboratory blood and urine test values, vital signs, weight, and injection related events.

End point type

Secondary

End point timeframe

From randomization (day 1) until the end of study visit (week 16).

End point values	Sepranolone + SoC. Safety analysis set (SAS)	SoC. Safety analysis set (SAS)
Number of subjects analysed
Units: Percentage of participants
number (not applicable)
Any AEs	94	11
SAEs	0	0
AEs leading to death	0	0
AEs leading to study withdrawal	6	0
AEs leading to treatment withdrawal	6	0

No statistical analyses for this end point

Secondary: Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

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End point title

Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

End point description

The Yale Global Tic Severity Scale (YGTSS) is a validated scale for assessing the severity of motor and vocal tics in both children and adults with Tourette syndrome. Tics are scored based on a semistructured interview, including scoring of the number, frequency, intensity, complexity, and interference of tics. The secondary endpoint is the impairment score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome.

End point type

Secondary

End point timeframe

Change from baseline at week 4, 8 and 12.

End point values	Sepranolone + SoC. mITT analysis set	SoC. mITT analysis set
Number of subjects analysed	17	9
Units: Change in impairment score from baseline
least squares mean (confidence interval 90%)	-4.19 (-8.86 to 0.47)	-0.87 (-7.58 to 5.84)

Change from baseline in the YGTSS impairment score

Comparison groups

Sepranolone + SoC. mITT analysis set v SoC. mITT analysis set

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other ^[1]

P-value

= 0.246

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-3.32

Confidence interval

level

90%

sides

2-sided

lower limit

-11.5

upper limit

4.85

Notes
[1] - The change from baseline YGTSS impairment score at week 4, 8, and 12 was modelled using the same Mixed Model Repeated Measures (MMRM) as YGTSS total score but with baseline YGTSS impairment score as baseline. The Least Squares (LS) means for each treatment as well as the treatment difference from the model at each time point was tabulated and visualised along with the 90% two-sided confidence interval.

Secondary: Change from baseline PUTS score at week 4, 8 and 12

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End point title

Change from baseline PUTS score at week 4, 8 and 12

End point description

The Premonitory Urge for Tics Scale (PUTS) scale is an assessment aiming to quantify the premonitory urge to tic. The scale is reliable and valid instrument for children from above the age of 10 and for adults. This scale is used as a self-report assessment instrument, where the Investigator asks 10 questions, out of which the score for the first 9 questions add up to the total score (i.e., the 9-Item Total). The respondent has 4 alternatives, "not at all true", "a little true", "pretty much true" and "very much true," represented by a score or 1-4, respectively. The total score ranges from 9-36, where a higher score indicates a worse outcome.

End point type

Secondary

End point timeframe

Change from baseline at week 4, 8 and 12.

End point values	Sepranolone + SoC. mITT analysis set	SoC. mITT analysis set
Number of subjects analysed	17	9
Units: Change in score from baseline
least squares mean (confidence interval 90%)	-2.60 (-4.36 to -0.84)	-1.80 (-4.33 to 0.74)

Change in baseline in PUTS score

Statistical analysis description

The change from baseline in 9-item total PUTS score at week 4, 8, and 12 was modelled using mixed model repeated measures (MMRM) with baseline total PUTS score as covariate. The Least Squares (LS) means for each treatment as well as the treatment difference from the model at each time point was tabulated and visualised along with the 90% twosided confidence interval.

Comparison groups

Sepranolone + SoC. mITT analysis set v SoC. mITT analysis set

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.333

Method

Mixed models analysis

Parameter type

Mean difference (net)

Point estimate

-0.8

Confidence interval

level

90%

sides

2-sided

lower limit

-3.94

upper limit

2.33

Secondary: Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

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End point title

Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

End point description

The Gilles de la Tourette Syndrome - Quality of Life (GTS-QoL) physical/activities of daily living (ADL) subscale assesses the impact of the symptoms of Tourette syndrome on the subject's quality of life. The instrument consists of 27 questions, asked by an interviewer, where subjects score the extent of impact on a 5-point verbal scale ranging from "no problems" to "extreme problems." The score ranges from 27-135, where a higher score indicates a worse outcome.

End point type

Secondary

End point timeframe

Change from baseline at week 4, 8 and 12.

End point values	Sepranolone + SoC. mITT analysis set	SoC. mITT analysis set
Number of subjects analysed	17	9
Units: Change in score from baseline
least squares mean (confidence interval 90%)	-2.91 (-4.34 to 1.49)	-2.21 (-4.25 to -0.16)

Change in Baseline in the GTS-QoL ADL Subscale.

Statistical analysis description

The change from baseline in the Gilles de la Tourette Syndrome - Quality of Life (GTS-QOL) total score and physical/activities of daily living (ADL) subscale at week 4, 8, and 12 will be modelled using the same MMRM as YGTSS but with baseline total GTS-QoL score/baseline of the ADL subscale as covariate. The LS means for each treatment as well as the treatment difference from the model at each time point will be tabulated and visualised along with the 90% two-sided confidence.

Comparison groups

Sepranolone + SoC. mITT analysis set v SoC. mITT analysis set

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

other

P-value

= 0.316

Method

Mixed models analysis

Parameter type

Median difference (net)

Point estimate

-0.71

Confidence interval

level

90%

sides

2-sided

lower limit

-3.2

upper limit

1.79

Secondary: TS-CGI score at week 4, 8 and 12

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End point title

TS-CGI score at week 4, 8 and 12

End point description

The Tourette Syndrome-Clinical Global Impression (TS-CGI) is a clinician rating of the change in severity of the symptoms of Tourette syndrome. The scale is a 7-step Likert scale, where the following alternatives are represented by a score of 1-7, respectively: "very much worsened," "much worsened," "minimally worsened," "no change," "minimally improved," "much improved," or "very much improved." The score ranges from 1-7, where a higher score indicates a better outcome.

End point type

Secondary

End point timeframe

TS-CGI score at week 4, 8 and 12.

End point values	Sepranolone + SoC. mITT analysis set	SoC. mITT analysis set
Number of subjects analysed	17	9
Units: Percentage of improved participants
number (not applicable)	50.0	37.5

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

The reporting of AEs started after randomization and continued until visit 7, i.e. the Follow-up visit.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

24.1

Reporting group title

Sepranolone

Reporting group description

Reporting group title

No Intervention

Reporting group description

Serious adverse events	Sepranolone	No Intervention
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 17 (0.00%)	0 / 9 (0.00%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0

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

Non-serious adverse events	Sepranolone	No Intervention
Total subjects affected by non serious adverse events
subjects affected / exposed	16 / 17 (94.12%)	1 / 9 (11.11%)
Investigations
Blood bilirubin increased
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
General disorders and administration site conditions
Fatigue
subjects affected / exposed	2 / 17 (11.76%)	0 / 9 (0.00%)
occurrences all number	2	0
Illness
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Injection site bruising
subjects affected / exposed	5 / 17 (29.41%)	0 / 9 (0.00%)
occurrences all number	7	0
Injection site erythema
subjects affected / exposed	3 / 17 (17.65%)	0 / 9 (0.00%)
occurrences all number	3	0
Injection site induration
subjects affected / exposed	3 / 17 (17.65%)	0 / 9 (0.00%)
occurrences all number	4	0
Injection site pain
subjects affected / exposed	9 / 17 (52.94%)	0 / 9 (0.00%)
occurrences all number	12	0
Injection site pruritus
subjects affected / exposed	6 / 17 (35.29%)	0 / 9 (0.00%)
occurrences all number	6	0
Injection site swelling
subjects affected / exposed	9 / 17 (52.94%)	0 / 9 (0.00%)
occurrences all number	16	0
Psychiatric disorders
Mood altered
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Groin pain
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Infections and infestations
COVID-19
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Conjunctivitis
subjects affected / exposed	1 / 17 (5.88%)	0 / 9 (0.00%)
occurrences all number	1	0
Influenza
subjects affected / exposed	0 / 17 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	2
Nasopharyngitis
subjects affected / exposed	2 / 17 (11.76%)	1 / 9 (11.11%)
occurrences all number	2	2
Pneumonia
subjects affected / exposed	0 / 17 (0.00%)	1 / 9 (11.11%)
occurrences all number	0	1

More information

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Were there any global substantial amendments to the protocol? No

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:
A randomized, open-label, multicenter, phase 2 clinical trial to explore the safety and efficacy of sepranolone in pediatric and adult patients with Tourette Syndrome.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

Primary: Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

Secondary: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

Secondary: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

Secondary: Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

Secondary: Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

Secondary: Change from baseline PUTS score at week 4, 8 and 12

Secondary: Change from baseline PUTS score at week 4, 8 and 12

Secondary: Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

Secondary: Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

Secondary: TS-CGI score at week 4, 8 and 12

Secondary: TS-CGI score at week 4, 8 and 12

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Clinical trials

Clinical Trial Results: A randomized, open-label, multicenter, phase 2 clinical trial to explore the safety and efficacy of sepranolone in pediatric and adult patients with Tourette Syndrome.

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

Primary: Change from baseline YGTSS Total Tic Score at week 4, 8 and 12.

Secondary: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

Secondary: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

Secondary: Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

Secondary: Change from baseline YGTSS Impairment Score at week 4, 8 and 12.

Secondary: Change from baseline PUTS score at week 4, 8 and 12

Secondary: Change from baseline PUTS score at week 4, 8 and 12

Secondary: Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

Secondary: Change from baseline in GTS-QOL physical/ADL subscale at week 4, 8 and 12

Secondary: TS-CGI score at week 4, 8 and 12

Secondary: TS-CGI score at week 4, 8 and 12

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A randomized, open-label, multicenter, phase 2 clinical trial to explore the safety and efficacy of sepranolone in pediatric and adult patients with Tourette Syndrome.