A Study to Explore the Effect of Sepranolone in Tourette Syndrome

NCT ID: NCT05434546

Last Updated: 2023-06-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-02-16
• Study Completion Date: 2023-02-01

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

Conditions

Tourette Syndrome; Tourette Syndrome in Adolescence

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Sepranolone

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

Group Type: EXPERIMENTAL

Sepranolone

Intervention Type: DRUG

Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment.

No Intervention

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

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Sepranolone

Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment.

Intervention Type: DRUG

Other Intervention Names

Isoallopregnanolone

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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Asarina Pharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nanette Debes, MD

Role: PRINCIPAL_INVESTIGATOR

Copenhagen University Hospital at Herlev

Locations

Herlev Hospital

Copenhagen, Herlev, Denmark

Bispebjerg Hospital

Copenhagen, , Denmark

Countries

Denmark

Other Identifiers

APH205

Identifier Type: -

Identifier Source: org_study_id