A Clinical Study to Evaluate the Safety and Efficacy of T92 in Pediatric Patients With Tourette Syndrome

NCT ID: NCT05188274

Last Updated: 2022-01-12

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: NA
• Total Enrollment: 150 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-31
• Study Completion Date: 2024-10-31

Brief Summary

A 12-week clinical study to evaluate the safety and efficacy of T92 in pediatric patients with Tourette Syndrome.

Detailed Description

This is a multi-center, randomized, double-blind, placebo-controlled, outpatient clinical study designed to evaluate the efficacy and safety of T92 in Tourette Syndrome pediatric patients.

This trial consists of a screening/wash-out period of up to 6 weeks, an 8-week supportive care period and a 4-week follow-up period for all subjects who completed the study. For the first two weeks, the patients will continue to take T92 at half dose and the T92 administration will be stopped from week 3 of the follow-up period.

Subjects will be randomly assigned to receive T92 or matching placebo based on individual body weight. The calculated amount of investigational product (T92 or placebo) will be administrated orally twice daily. Morning dose and evening dose should be administrated at about the same time every day and irrelevant to meals.

Conditions

Tourette Syndrome in Children; Tourette Syndrome in Adolescence

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: QUADRUPLE

Study Groups

T92 group

The dose of T92 was calculated based on body weight, orally twice daily. Supportive care duration: 8 weeks

Group Type: EXPERIMENTAL

T92

Intervention Type: DIETARY_SUPPLEMENT

T92 granules (5g/sachet) will be taken BID for 8 weeks, the dosage will be calculated by individual's body weight.

Placebo group

The dose of placebo was calculated based on body weight, orally twice daily. Supportive care duration: 8 weeks

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DIETARY_SUPPLEMENT

The placebo matched to T92 granules will be taken BID for 8 weeks.

Interventions

T92

T92 granules (5g/sachet) will be taken BID for 8 weeks, the dosage will be calculated by individual's body weight.

Intervention Type: DIETARY_SUPPLEMENT

Placebo

The placebo matched to T92 granules will be taken BID for 8 weeks.

Intervention Type: DIETARY_SUPPLEMENT

Eligibility Criteria

Inclusion Criteria

1. Male or female children and adolescents aged 6 to 17 years upon screening with a Diagnosis of Tourette Syndrome according to Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V).

2. TTS ≥ 20 on the YGTSS at screening and baseline.

3. In the Investigator's opinion the presenting tic symptoms caused impairment in the subject's normal daily routines.

4. Females of childbearing potential had a negative pregnancy test, practiced acceptable double-barrier methods of contraception (or abstinence), and were not pregnant or lactating.

5. Written informed assent or consent provided by the subject, and written informed consent provided by the parent(s)/guardians(s), as appropriate per the IRB/EC.

6. In the opinion of the Investigator, the subject and designated guardian(s) and/or parent(s) must be considered likely to comply with the study protocol and to have a high probability of completing the study.

Exclusion Criteria

1. Medical history consistent with another neurologic condition that may have had accompanying abnormal movements (e.g., Huntington's disease, Parkinson's disease, Sydenham's chorea, Wilson's disease, Mental retardation, Traumatic brain injury, Stroke, Restless legs syndrome)

2. History of schizophrenia, bipolar disorder, or other psychotic disorder; Comorbid conditions such as: Obsessive Compulsive Disorder (OCD) and Attention Deficit Hyperactivity Disorder (ADHD) can be included.

3. Active major depression disorder.

4. History of neuroleptic malignant syndrome.

5. Subjects who have had treatment with:

1. investigational medication within 3 months of starting study

2. depot antipsychotics within 3 months of starting study

3. Antipsychotics with possible effects on TS symptoms: i.e., topiramate within 1 week; levodopa or dopamine agonists within 2 weeks prior to baseline.

4. VMAT2 inhibitors within 2 weeks prior to baseline.

5. Atypical antipsychotics within 4 weeks: this class include risperidone (Risperdal), quetiapine (Seroquel), olanzapine (Zyprexa), ziprasidone (Zeldox), paliperidone (Invega), aripiprazole (Abilify) and clozapine (Clozaril).

6. Selective serotonin reuptake inhibitors unless the dosage has been stable for a minimum of 4 weeks prior to study start and not prescribed to relieve the neurological signs of TS.

6. Sexually active males or females who would not commit to utilizing 2 of the approved birth control methods or who would not remain abstinent during the trial and for 90 days (males) or 30 days (females) following the last dose of investigational product.

7. CBIT need to be started at least two months at screening. Could continue on existing therapy or stop it based on PI's opinion.

8. Significant psychoactive substance use disorder within the past 3 months; or the urine drug screen was positive.

9. Significant lab abnormality:

1. Platelets ≤ 75,000/mm3

2. Hemoglobin ≤ 9 g/dl

3. Neutrophils, absolute ≤ 1000/mm3

4. Aspartate transaminase (AST) > 3×ULN (upper limit of normal)

5. Alanine aminotransferase (ALT) > 3×ULN

6. Creatinine ≥ 2 mg/dl

10. History or presence of any clinically important medical condition that, in the judgment of the investigator, is likely to deteriorate, could be detrimental to the subject, or could affect the subject's ability to complete the study.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tasly Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael H Bloch, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Locations

Yale Child Study Center

New Haven, Connecticut, United States

Countries

United States

Central Contacts

Michael H Bloch, MD, PhD

Role: CONTACT

michael.bloch@yale.edu

203-974-7551

James F Leckman, MD, PhD

Role: CONTACT

james.leckman@yale.edu

203-785-7971

Facility Contacts

Michael H Bloch, MD, PhD

Role: primary

michael.bloch@yale.edu

203-974-7551

James F Leckman, MD, PhD

Role: backup

james.leckman@yale.edu

203-785-7971

Other Identifiers

T92-US-01

Identifier Type: -

Identifier Source: org_study_id