Clinical trial NCT05434546: A Study to Explore the Effect of Sepranolone in Tourette Syndrome
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Tourette Syndrome
Tourette Syndrome in Adolescence
Keywords: Tourette Syndrome in Adults
Lead sponsor: Asarina Pharma
Sponsor class: INDUSTRY
First posted: 2022-06-28
Enrollment: 26
Sex: ALL
Minimum age (years): 12
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Interventional, open-label, multicenter, randomized, parallel group study
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: 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

Primary outcomes:
measure: 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.
timeFrame: Change from baseline at week 4, 8 and 12.
Secondary outcomes:
measure: Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome
timeFrame: From randomization (day 1) until the end of study visit (week 16).
measure: 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.
timeFrame: Change from baseline at week 4, 8 and 12.
measure: 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.
timeFrame: Change from baseline at week 4, 8 and 12.
measure: 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.
timeFrame: Change from baseline at week 4, 8 and 12.
measure: 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).
timeFrame: TS-CGI score at week 4, 8 and 12.
Other outcomes:
Locations:
city: Copenhagen
country: Denmark
facility: Herlev Hospital
state: Herlev
city: Copenhagen
country: Denmark
facility: Bispebjerg Hospital
Study officials:
affiliation: Copenhagen University Hospital at Herlev
name: Nanette Debes, MD
role: PRINCIPAL_INVESTIGATOR
References: