A Study of the Effectiveness and Safety of Tetrabenazine MR in Pediatric Subjects With Tourette's Syndrome

NCT01133353 | Withdrawn Phase 2 DMC

• 1 other identifier: BVF-018-201
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this clinical trial is to study the therapeutic effect of tetrabenazine MR in children with Tourette's Syndrome, as measured by the improvement in total tic score of the Yale Global Tic Severity Scale (YGTSS).

Conditions

Tourette's Syndrome

Outcome Measures

Primary Outcomes (1)

• Improvement in total tic score as measured by the Yale Global Tic Severity Score (YGTSS)

  Days 0 to 84

Secondary Outcomes (6)

• To evaluate the safety and tolerability of tetrabenazine using cognitive measures

  Days 0 and 84

• To evaluate the safety and tolerability of tetrabenazine using an assessment of depression

  Days 0 to 84

• To evaluate the safety and tolerability of tetrabenazine using an assessment of suicidality

  Days 0 to 91

• To explore the therapeutic effects of tetrabenazine using the full YGTSS

  Days 0 to 84

• To explore the therapeutic effects of tetrabenazine using the Patient Global Impression of Change scale (PGI-C)

  Days 0 to 84

Study Arms (2)

Tetrabenazine MR

EXPERIMENTAL

Drug: Tetrabenazine MR

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Tetrabenazine MR DRUG

Modified-Release (MR) tablets containing 15 mg or 30 mg tetrabenazine, dose-escalating up to a maximum of 150 mg depending on weight, once per day for 12 weeks

Tetrabenazine MR

Placebo DRUG

1 to 5 tablets once per day depending on weight for 12 weeks

Placebo

Eligibility Criteria

• Age: 5 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects must meet all of the following criteria:
• Children aged 5 to up to 17 years
• Subjects must be capable of providing informed consent or assent, if applicable, and parents/legal guardian must be capable of providing informed consent/permission and complying with study procedures
• Subject and parents/legal guardian must be able to communicate effectively with Investigator and study coordinator
• Diagnosed with Tourette's Syndrome, according to DSM-IV-TR and using K-SADS-PL to aid diagnosis, and requiring drug therapy
• Total tic score ≥ 22 as measured by YGTSS at screening and baseline
• Total body weight ≥ 15 kg (33 lbs.)
• Tics are causing distress or impairment, as determined by parent/legal guardian or subject and by the Investigator, despite current treatment regimen
• Able to swallow whole tablets without difficulty
• Non-pregnant status:
• All female subjects must be non-pregnant (as demonstrated by negative serum β-HCG test), non-breastfeeding, and must avoid pregnancy from at least 10 days before signing the informed consent/assent and up until 1 month after the end of the study by abstaining from sexual activity or using two (2) medically acceptable methods of contraception, such as: a non-hormonal IUD with spermicide, female condom with spermicide, contraceptive sponge with spermicide, an intravaginal system, diaphragm with spermicide, cervical cap with spermicide, a male sexual partner who agrees to use a male condom with spermicide, a sterile sexual partner.
• Male subjects must abstain from sexual activity from the time they sign the informed consent/assent and up until 1 month after the end of the study or use a condom with spermicide.
• For all females of child bearing potential, a serum pregnancy test result must be negative at Screening and urine pregnancy test must be negative at Baseline

You may not qualify if:

• Subjects are not eligible if any of the following criteria are met:
• Subjects with history or current major depressive disorder
• Prior treatment with \> 7 doses of tetrabenazine
• Prior treatment with reserpine
• Subjects with impaired renal function (defined as having a creatinine level of ≥ 1.5 times the upper limit of age-appropriate normal value)
• Subjects with current or any history of suicidal ideation
• Participation in an investigational medication trial within the 3 months prior to the Screening visit, except for study BVF-018-102
• Use of Botox® (botulinum toxin) within the 4 months prior to the Screening visit
• Immediate families of site Investigators or sponsor employees
• Excluding conditions related directly to the disease under study, subject has a history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, or oncologic or any other condition that, in the opinion of the Investigator, would jeopardize the safety of the subject or the validity of the study results.
• Subjects with clinically important thyroid dysfunction requiring medication
• Subjects with impaired hepatic function (defined as having SGOT/SGPT levels ≥ 1.5 times the upper limit of age-appropriate normal values)
• Subjects with a Children's Depression Rating Scale-Revised (CDRS-R) total score of \> 65
• Male subjects with QTc \> 450 msec; female subjects with QTc \> 470 msec based on Bazzett's correction formula
• Organic brain disease, for example, traumatic brain injury residua or toxic delirium

Sponsors & Collaborators

• Bausch Health Americas, Inc.: lead

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 Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2010
• First Posted: May 28, 2010
• Study Start: March 1, 2011
• Primary Completion: November 1, 2011
• Study Completion: December 1, 2011
• Last Updated: November 29, 2019

Record last verified: 2019-11