A Study to Explore the Effect of Sepranolone in Tourette Syndrome

NCT05434546 | Completed Phase 2

• 1 other identifier: APH205
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 2

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

Keywords

Tourette Syndrome in Adults

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (5)

• Safety and tolerability of Sepranolone in adolescent and adult patients with Tourette syndrome

  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.

  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.

  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.

  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).

  TS-CGI score at week 4, 8 and 12.

Study Arms (2)

Sepranolone

EXPERIMENTAL

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

Drug: Sepranolone

No Intervention

NO INTERVENTION

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

Interventions

Sepranolone DRUG

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

Also known as: Isoallopregnanolone

Sepranolone

Eligibility Criteria

• Age: 12 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Asarina Pharma: lead

Study Sites (2)

Herlev Hospital

Copenhagen, Herlev, 2730, Denmark

Location

Bispebjerg Hospital

Copenhagen, 2400, Denmark

Location

MeSH Terms

Conditions

Tourette Syndrome

Interventions

Pregnanolone

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Nanette Debes, MD

  Copenhagen University Hospital at Herlev

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Interventional, open-label, multicenter, randomized, parallel group study

Study Record Dates

• First Submitted: March 12, 2022
• First Posted: June 28, 2022
• Study Start: February 16, 2022
• Primary Completion: February 1, 2023
• Study Completion: February 1, 2023
• Last Updated: June 9, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

DK (2)