A Study to Explore the Effect of Sepranolone in Tourette Syndrome

Sponsor

Asarina Pharma (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05434546

Collaborator

Herlev Hospital (Other)

Enrollment

Location

Arms

13.4

Anticipated Duration (Months)

2.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

30 subjects aged 12 to 45 years of age diagnosed with Tourette Syndrome with a YGTSS score > 20 during 30 days of baseline observation to be randomized to

continued present stable treatment (10 subjects) or
continued present stable treatment plus 10 mg of Sepranolone injected s.c. twice weekly for 12 weeks (20 patients) followed by one month of observation

Primary objective: to investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome after 12 weeks

Condition or Disease	Intervention/Treatment	Phase
Tourette Syndrome in Children	Drug: Sepranolone	Phase 2

Detailed Description

30 subjects aged 12 to 45 years of age diagnosed with Tourette Syndrome with a YGTSS score > 20 during 30 days of baseline observation to be randomized to

continued present stable treatment (10 subjects) or
continued present stable treatment plus 10 mg of Sepranolone injected s.c. twice weekly for 12 weeks (20 patients) followed by one month of observation

Primary objective: to investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette Syndrome after 12 weeks of treatment with Sepranolone Change from baseline YGTSS total score at week 4, 8 and 12

Secondary objectives:

to investigate the efficacy of Sepranolone to reduce tic related impairment in patients with Tourette Syndrome after 12 weeks of treatment with Sepranolone Change from baseline YGTSS impairment score at week 4, 8 and 12
to investigate the efficacy of Sepranolone to reduce the urge to tic in patients with Tourette Syndrome at week 12 of treatment with Sepranolone Change from baseline PUTS score at weeks 4, 8 and 12
To evaluate the effect of Sepranolone on activities of daily living in patient with Tourette Syndrome at week 12 of treatment with Sepranolone Change Quality of Life (GTSQOL) physical/activities of daily living subscale at weeks 4, 8 and 12
To evaluate the effect on Global impression by Sepranolone at week 12 of treatment with Sepranolone Change from baseline in the Tourette Syndrome - Clinical Global Impression (TS-CGI) score at week 4, 8 and 12

Safety objectives:

to evaluate the safety and tolerability of Sepranolone in patients with Tourette Syndrome

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Interventional, open-lable, multi-center, randomized, parallel group studyInterventional, open-lable, multi-center, randomized, parallel group study

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Clinical Trial to Explore the Safety and Efficacy of Sepranolone in Pediatric and Adult Patients With Tourette Syndrome.

Actual Study Start Date :

Feb 16, 2022

Anticipated Primary Completion Date :

Jan 31, 2023

Anticipated Study Completion Date :

Mar 31, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Sepranolone Sepranolone 10 mg sc twice weekly for 12 weeks on top of patients stable therapy througout past 3 months	Drug: Sepranolone Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks on top of stable Tourette treatment during the past 3 months Other Names: Isoallopregnanolone
No Intervention: No Intervention Continuation on stable treatment of the past 3 months for 12 weeks

Arm

Intervention/Treatment

Experimental: Sepranolone

Sepranolone 10 mg sc twice weekly for 12 weeks on top of patients stable therapy througout past 3 months

Drug: Sepranolone

Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks on top of stable Tourette treatment during the past 3 months

Other Names:

Isoallopregnanolone

No Intervention: No Intervention

Continuation on stable treatment of the past 3 months for 12 weeks

Outcome Measures

Primary Outcome Measures

Reduction of tic score (YGTSS) at week 12 [20 weeks (4 baseline +12 treatment +4 follow up)]
change from baseline YGTSS Total score at week 4, 8 and 12

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Tic severity score > 20 (YGTSS) at baseline
The patient may have OCD as a comorbidity
All patients to continue stable cognitive or medical treatment (> 3 months)

Exclusion Criteria:

Patient has participated in a clinical study over the past 30 days
Evidence or history of neurological disease that may interfere with the study
Malignant disease
Unstable or clinical significant medical condition that could pose a risk
HIV/ongoing hepatitis
Clinical significant findings in vital signs
History of anaphylactic reactions

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Herlev Hospital	Copenhagen	Herlev	Denmark	2730

Sponsors and Collaborators

Asarina Pharma
Herlev Hospital

Investigators

Principal Investigator: Nanette Debes, MD, Copenhagen University Hospital at Herlev

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Asarina Pharma

ClinicalTrials.gov Identifier:

NCT05434546

Other Study ID Numbers:

APH205

First Posted:

Jun 28, 2022

Last Update Posted:

Jun 28, 2022

Last Verified:

Mar 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Tourette Syndrome

Syndrome

Pregnanolone

Study Results

No Results Posted as of Jun 28, 2022