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

NCT05188274 | Unknown

• 1 other identifier: T92-US-01
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Tourette Syndrome in Children; Tourette Syndrome in Adolescence

Keywords

Tourette Syndrome

Outcome Measures

Primary Outcomes (1)

• The mean change from baseline to week 8 in total tic score (TTS) of Yale Global Tic Severity Scale (YGTSS).

  The Yale Global Tic Severity Scale (YGTSS) is a semi-structured clinical interview for assessing the severity of tics in children and adults. The YGTSS enables evaluations of number, frequency, intensity, complexity, and interference of motor and phonic tics, covering the past week. Each domain is scored on a 6-point scale (range 0-5) with a separate rating for "overall impairment" regarding the subject's daily life and activities. YGTSS-TTS is the sum of the total motor tic score plus the total phonic tic score ranging from 0-50. Higher scores indicate greater severity/worse outcome. Changes in TTS after 8 weeks of supportive care duration will be compared between the T92 and placebo groups.

  Baseline, Week 8

Secondary Outcomes (6)

• The change from baseline to Week 8 in total Yale Global Tic Severity Scale (YGTSS) scale YGTSS tic-related impairment (TRI) score at week 8

  Baseline, Week 8

• Mean change from baseline to TS-CGI severity and improvement at week 8

  Baseline, Week 2, 4, 6 and 8

• Clinical response rate, defined as a ≥ 30% reduction from baseline on TTS score at different check points.

  Baseline, Week 2, 4, 6 and 8

• Evaluation of T92 in reducing severity of the sensations before the emergence of tics by comparing the change in PUTS scores at week 8 from baseline.

  Baseline, Week 4 and 8

• Evaluation of T92 in reducing severity of obsessions and compulsion occurring by comparing the change in CY-BOCS scores at week 8 from baseline.

  Baseline, Week 4 and 8

Study Arms (2)

T92 group

EXPERIMENTAL

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

Dietary Supplement: T92

Placebo group

PLACEBO COMPARATOR

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

Dietary Supplement: Placebo

Interventions

T92 DIETARY_SUPPLEMENT

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

T92 group

Placebo DIETARY_SUPPLEMENT

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

Placebo group

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).
• TTS ≥ 20 on the YGTSS at screening and baseline.
• In the Investigator's opinion the presenting tic symptoms caused impairment in the subject's normal daily routines.
• Females of childbearing potential had a negative pregnancy test, practiced acceptable double-barrier methods of contraception (or abstinence), and were not pregnant or lactating.
• 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.
• 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.

You may not qualify if:

• 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)
• 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.
• Active major depression disorder.
• History of neuroleptic malignant syndrome.
• Subjects who have had treatment with:
• investigational medication within 3 months of starting study
• depot antipsychotics within 3 months of starting study
• Antipsychotics with possible effects on TS symptoms: i.e., topiramate within 1 week; levodopa or dopamine agonists within 2 weeks prior to baseline.
• VMAT2 inhibitors within 2 weeks prior to baseline.
• Atypical antipsychotics within 4 weeks: this class include risperidone (Risperdal), quetiapine (Seroquel), olanzapine (Zyprexa), ziprasidone (Zeldox), paliperidone (Invega), aripiprazole (Abilify) and clozapine (Clozaril).
• 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.
• 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.
• CBIT need to be started at least two months at screening. Could continue on existing therapy or stop it based on PI's opinion.
• Significant psychoactive substance use disorder within the past 3 months; or the urine drug screen was positive.
• Significant lab abnormality:

Sponsors & Collaborators

• Tasly Pharmaceuticals, Inc.: lead

Study Sites (1)

Yale Child Study Center

New Haven, Connecticut, 06520, United States

Location

MeSH Terms

Conditions

Tourette Syndrome

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

Study Officials

• Michael H Bloch, MD, PhD

  Yale University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Michael H Bloch, MD, PhD

CONTACT

203-974-7551; michael.bloch@yale.edu

James F Leckman, MD, PhD

CONTACT

203-785-7971; james.leckman@yale.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2021
• First Posted: January 12, 2022
• Study Start: May 1, 2022
• Primary Completion: February 1, 2024
• Study Completion: October 1, 2024
• Last Updated: January 12, 2022

Record last verified: 2022-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)