A Study to Explore the Effect of Sepranolone in Tourette Syndrome

Asarina Pharma

Status and phase: Completed; Phase 2

Conditions: Tourette Syndrome; Tourette Syndrome in Adolescence

Treatments: Drug: Sepranolone

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05434546

APH205

Details and patient eligibility

About

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

Enrollment: 26 patients

Sex: All

Ages: 12 to 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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26 participants in 2 patient groups

Sepranolone

Experimental group

Description:

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

Treatment:

Drug: Sepranolone

No Intervention

No Intervention group

Description:

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