Tourette's SyndromePhase 2TBZ-MRNCT01133353

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

A Phase 2 study testing Tetrabenazine MR in people with Tourette's Syndrome, run by Bausch Health Americas, Inc..

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial 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). Read more about this study

Who is it for?: Ages 5 to 16 years
Conditions the study is for: Tourette's Syndrome
What is being tested?: Tetrabenazine MR
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 0 participants
Where: No sites listed yet
Study dates: Mar 2011to Dec 2011
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 5 to 16 years
All sexes
No healthy volunteers
Child

Who may be able to join

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

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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

Who cannot join

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

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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

About this study

Official title: 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

Read the 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.

Conditions: Tourette's Syndrome
Being tested: Tetrabenazine MR, Placebo
Medical subject headings: Tourette Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

Study groups in NCT01133353
Study group	Role	What they receive
Tetrabenazine MR	Experimental	Not described Receives: Drug: Tetrabenazine MR
Placebo	Placebo comparator	Not described Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Improvement in total tic score as measured by the Yale Global Tic Severity Score (YGTSS)
Measured: Days 0 to 84

Secondary outcomes (6)

To evaluate the safety and tolerability of tetrabenazine using cognitive measures
Measured: Days 0 and 84
To evaluate the safety and tolerability of tetrabenazine using an assessment of depression
Measured: Days 0 to 84
To evaluate the safety and tolerability of tetrabenazine using an assessment of suicidality
Measured: Days 0 to 91
To explore the therapeutic effects of tetrabenazine using the full YGTSS
Measured: Days 0 to 84
To explore the therapeutic effects of tetrabenazine using the Patient Global Impression of Change scale (PGI-C)
Measured: Days 0 to 84
To explore the therapeutic effects of tetrabenazine using the Clinical Global Impression of Severity scale (CGI-S)
Measured: Days 0 to 84

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Mar 2011Dec 2011

First posted28 May 2010 (estimated)
Study startMar 2011
Primary completionNov 2011 (estimated)
Study completionDec 2011 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Tourette's SyndromeStudies of the same condition, open and under way.
More trials run by Bausch Health Americas, Inc.The sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01133353
Sponsor's protocol ID: BVF-018-201
Acronym: TBZ-MR
Status last verified: Nov 2019
First submitted: 24 May 2010
Last updated: 29 Nov 2019
Results posted: No
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Not yet recruiting
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
Not yet recruiting
Transcutaneous Auricular Vagus Nerve Stimulation for Chronic Tic Disorders/Tourette's Syndrome in Children and Adolescents
Tourette Syndrome, Chronic Tic Disorder
Recruiting
Deep Brain Stimulation Therapy in Movement Disorders
Parkinson's Disease
Recruiting
Exploring the Role of Neuroactive Steroids in Tourette Syndrome
Tourette Syndrome in Children
Active, not recruiting
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
Tourette Syndrome

All Tourette's Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: 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
ClinicalTrials.gov ID: NCT01133353
Acronym / sponsor ID: TBZ-MR / BVF-018-201
Conditions: Tourette's Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Withdrawn
Enrolment: 0 participants (actual)
Lead sponsor: Bausch Health Americas, Inc.Industry (pharma, biotech, devices)
Study start: Mar 2011
Primary completion: Nov 2011 (estimated)
Study completion: Dec 2011 (estimated)
First posted: 28 May 2010 (estimated)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Tetrabenazine MR	Experimental	Drug: Tetrabenazine MR
Placebo	Placebo comparator	Drug: Placebo

Who may qualify

Ages: 5 to 16 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (13 listed)

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

Key exclusion criteria (26 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Tetrabenazine MRModified-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	Drug	Tetrabenazine MR
Placebo1 to 5 tablets once per day depending on weight for 12 weeks	Drug	Placebo

Primary outcome

Improvement in total tic score as measured by the Yale Global Tic Severity Score (YGTSS)
Time frame: Days 0 to 84

6 secondary outcomes

To evaluate the safety and tolerability of tetrabenazine using cognitive measures
Time frame: Days 0 and 84
To evaluate the safety and tolerability of tetrabenazine using an assessment of depression
Time frame: Days 0 to 84
To evaluate the safety and tolerability of tetrabenazine using an assessment of suicidality
Time frame: Days 0 to 91
To explore the therapeutic effects of tetrabenazine using the full YGTSS
Time frame: 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
To explore the therapeutic effects of tetrabenazine using the Clinical Global Impression of Severity scale (CGI-S)
Time frame: Days 0 to 84

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2019.

Related trials for Tourette's Syndrome

Not yet recruiting
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
Not yet recruiting
Transcutaneous Auricular Vagus Nerve Stimulation for Chronic Tic Disorders/Tourette's Syndrome in Children and Adolescents
Tourette Syndrome, Chronic Tic Disorder
Recruiting
Deep Brain Stimulation Therapy in Movement Disorders
Parkinson's Disease
Recruiting
Exploring the Role of Neuroactive Steroids in Tourette Syndrome
Tourette Syndrome in Children
Active, not recruiting
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
Tourette Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tetrabenazine MR in people with Tourette's Syndrome, run by Bausch Health Americas, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 29 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Improvement in total tic score as measured by the Yale Global Tic Severity Score (YGTSS) (time frame: Days 0 to 84). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01133353.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2019.