- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01133353
A Study of the Effectiveness and Safety of Tetrabenazine MR in Pediatric Subjects With Tourette's Syndrome (TBZ-MR)
November 26, 2019 updated by: Bausch Health Americas, Inc.
A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Tetrabenazine MR in Subjects Aged 5 up to 17 Years With Tourette's Syndrome
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).
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
Detailed Description
Tourette's Syndrome (TS) is typically diagnosed in early childhood (6-7 years) or before the age of 21 years and is characterized by chronic, intermittent motor and phonic tics.
There is a body of evidence, mostly from the clinical experience data, suggesting the efficacy of tetrabenazine in TS patients with tics, including patients who were unresponsive to other treatment options.
These publications consistently report the usefulness of tetrabenazine as an alternative to conventional neuroleptics for the treatment of TS.
Study Type
Interventional
Phase
- Phase 2
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years to 14 years (Child)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
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
Exclusion Criteria:
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
- Any subject at immediate risk of requiring hospitalization
- Physical examination, electrocardiography, or laboratory values that are clinically important in the opinion of the Investigator
- Autistic spectrum disorder
- Schizophrenia
- Other psychotic disorder
- Subjects with Bipolar I Disorder
- Subjects with one or more first-degree relatives with Bipolar I Disorder
- Subjects who are unable to complete a washout period of 4 weeks for fluoxetine and 3 weeks for MAOIs, respectively, prior to Baseline visit and who require fluoxetine and MAOIs during the Treatment Period
- Inability to washout of strong inhibitors or inducers of CYP2D6 within 7 days of the Baseline visit
- Positive findings on urine drug screen at the Screening Visit
- Allergies to tetrabenazine or its excipients
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
|
1 to 5 tablets once per day depending on weight for 12 weeks
|
Experimental: Tetrabenazine MR
|
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
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Improvement in total tic score as measured by the Yale Global Tic Severity Score (YGTSS)
Time Frame: Days 0 to 84
|
Days 0 to 84
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To evaluate the safety and tolerability of tetrabenazine using cognitive measures
Time Frame: Days 0 and 84
|
Days 0 and 84
|
To evaluate the safety and tolerability of tetrabenazine using an assessment of depression
Time Frame: Days 0 to 84
|
Days 0 to 84
|
To evaluate the safety and tolerability of tetrabenazine using an assessment of suicidality
Time Frame: Days 0 to 91
|
Days 0 to 91
|
To explore the therapeutic effects of tetrabenazine using the full YGTSS
Time Frame: Days 0 to 84
|
Days 0 to 84
|
To explore the therapeutic effects of tetrabenazine using the Patient Global Impression of Change scale (PGI-C)
Time Frame: Days 0 to 84
|
Days 0 to 84
|
To explore the therapeutic effects of tetrabenazine using the Clinical Global Impression of Severity scale (CGI-S)
Time Frame: Days 0 to 84
|
Days 0 to 84
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
March 1, 2011
Primary Completion (Anticipated)
November 1, 2011
Study Completion (Anticipated)
December 1, 2011
Study Registration Dates
First Submitted
May 24, 2010
First Submitted That Met QC Criteria
May 27, 2010
First Posted (Estimate)
May 28, 2010
Study Record Updates
Last Update Posted (Actual)
November 29, 2019
Last Update Submitted That Met QC Criteria
November 26, 2019
Last Verified
November 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Mental Disorders
- Pathologic Processes
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Disease
- Genetic Diseases, Inborn
- Basal Ganglia Diseases
- Movement Disorders
- Neurodegenerative Diseases
- Heredodegenerative Disorders, Nervous System
- Neurodevelopmental Disorders
- Tic Disorders
- Syndrome
- Tourette Syndrome
- Physiological Effects of Drugs
- Adrenergic Agents
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Neurotransmitter Uptake Inhibitors
- Membrane Transport Modulators
- Adrenergic Uptake Inhibitors
- Tetrabenazine
Other Study ID Numbers
- BVF-018-201
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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