A Study to Explore the Effect of Sepranolone in Tourette Syndrome

Study Overview
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Brief Summary
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The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. The total study duration from the Screening Visit to the final follow-up visit was approximately 26 weeks and included the following periods: A baseline period of 4 weeks between the screening visit (Visit 1) and randomization (4 weeks of baseline period were not needed in well-known adult subjects with stable Tourette syndrome history over the past at least 4 weeks). A school holiday/annual leave period of 2-6 weeks where no study-related activities were done. A randomized treatment period of 12 weeks A safety follow-up period of 4 weeks

Official Title
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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

Conditions
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Tourette Syndrome, Tourette Syndrome in Adolescence

Intervention / Treatment
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* Drug: Sepranolone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Tic severity score ≥ 20 Yale Global Tic Severity Scale (YGTSS) Total Score at baseline The patient may have Obsessive-Compulsive Disease (OCD) as a comorbidity 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

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Interventional, open-label, multicenter, randomized, parallel group study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sepranolone<br>Sepranolone 10 mg sc twice weekly for 12 weeks alongside the patient's standard of care Tourette treatment. | Drug: Sepranolone<br>* Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment.<br>* Other names: Isoallopregnanolone;|
| No Intervention: No Intervention<br>Continuation of the patient's standard of care Tourette treatment for 12 weeks. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To investigate the efficacy of Sepranolone to reduce tic severity in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the The Yale Global Tic Severity Scale (YGTSS) Total Score. | 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. | Change from baseline at week 4, 8 and 12. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome | 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. | From randomization (day 1) until the end of study visit (week 16). | 
| To evaluate the efficacy of Sepranolone to reduce tic related impairment in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Yale Global Tic Severity Scale (YGTSS) Impairment Score. | 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 secondary endpoint is the impairment score assessment. The score ranges from 0-50, where a higher score indicates a worse outcome. | Change from baseline at week 4, 8 and 12. | 
| To evaluate the efficacy of Sepranolone to reduce the urge to tic in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the Premonitory Urge for Tics Scale (PUTS) scale. | 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. | Change from baseline at week 4, 8 and 12. | 
| To evaluate the effect of Sepranolone on activities of daily living in patients with Tourette syndrome at 12 weeks, as measured by change in baseline in the GTS-QoL ADL subscale. | 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. | Change from baseline at week 4, 8 and 12. | 
| To evaluate the effect on Global impression by Sepranolone in patients with Tourette syndrome at 12 weeks, as measured by the Tourette Syndrome-Clinical Global Impression (TS-CGI). | 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. | TS-CGI score at week 4, 8 and 12. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Tourette Syndrome in Adults