Pilot Efficacy Study of T2000 in Myoclonus Dystonia

This study has been terminated.
(Slow Recruitment of eligible patients)
Sponsor:
Information provided by (Responsible Party):
Taro Pharmaceuticals USA
ClinicalTrials.gov Identifier:
NCT00506012
First received: July 20, 2007
Last updated: December 19, 2013
Last verified: November 2013
  Purpose

This pilot study will evaluate the safety and efficacy of once daily T2000 when used to treat patients with Myoclonus Dystonia over a 12 week period.


Condition Intervention Phase
Myoclonus
Drug: T2000
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Efficacy and Safety of Taro Pharmaceuticals' Pro-Drug T2000 (1,3-Dimethoxymethyl-5,5-Diphenyl-Barbituric Acid) In Patients With Myoclonus Dystonia: An Open Label Sequential Dose Escalation Study

Resource links provided by NLM:


Further study details as provided by Taro Pharmaceuticals USA:

Primary Outcome Measures:
  • Effect of treatment on the movement disorder will be measured by a myoclonus scale and a dystonia scale as well as by assessment of overall functional status. Response at various dosages will be compared to baseline for all patients. [ Time Frame: Up to 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety parameters including neurological examination, blood tests and EKG will be monitored throughout the treatment period and during withdrawal of the medication. [ Time Frame: Up to 16 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 5
Study Start Date: August 2007
Study Completion Date: October 2011
Primary Completion Date: August 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
T2000
Drug: T2000
T2000 at doses of 200 mg a day to 1000 mg a day

Detailed Description:

Myoclonus Dystonia (M-D) is a rare, inherited movement disorder in which patients experience myoclonus - sudden, brief, jerky involuntary motions, often in association with dystonia - involuntary sustained contractions causing twisting or abnormal posture. While most M-D patients respond significantly to alcohol, there are no approved medications for M-D. A variety of medications are currently used to treat M-D, but these treatments work in a small proportion of patients and provide only partial improvement in symptoms; their use is also limited by side-effects in many patients.

T2000 is a medication currently under development for the treatment of movement disorders, including essential tremor (ET). Although T2000 is a new medication, it belongs to a class of medications that has been used for many years for the treatment of a variety of medical conditions. In previous studies, T2000 appeared to be effective in controlling symptoms of ET and some patients with severe ET had major improvements in tremor. As would be expected for medications in this class, T2000 can cause sedation at high blood levels, such as may be seen when large doses are given to older individuals. In younger patients, T2000 caused only minimal side effects even when administered at high doses and for periods of several weeks to several months.

The current study will evaluate the safety and efficacy of T2000 in patients with M-D. Patients will receive doses of T2000 beginning at 200 mg a day and increasing every other week by an additional 200 mg a day up to a maximal dose of 1000 mg a day. The total duration of treatment will be 12 weeks. Patient's symptoms of myoclonus and dystonia, as well as overall neurological examination, will be monitored throughout the study. The response to T2000 will be determined by comparing the severity of myoclonus and dystonia while patients are receiving T2000 compared to the symptoms observed without active medication.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients should meet diagnostic criteria for M-D based on the following criteria:

    • myoclonus is the primary feature; focal or segmental dystonia of any severity may also be present
    • symptoms began by age 20
    • a familial pattern should be present
    • neurological history should not be suggestive of a different neurological condition
    • investigations such as imaging, EEG and evoked potential tests should be normal
  • Patients will be eligible for this study if they are symptomatic on their current treatment, cannot tolerate current therapies, or are treatment naïve patients who have been explained treatment alternatives.

Exclusion Criteria:

  • Patients adequately controlled without side effects on a current M-D treatment
  • Current treatment with a barbiturate such as phenobarbital or primidone
  • Pregnant patients or patients who may become pregnant during the study
  • Patients who must take medications that alter liver metabolism as well as patients with liver disease or coagulation disorders
  • Patients with seizure disorders
  • Patients with a history of allergy or hypersensitivity reaction to barbiturates or other related medications, such as phenobarbital or phenytoin
  • Patient with significant general medical or clinical laboratory abnormalities
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00506012

Locations
Canada, Ontario
Investigator Site
Toronto, Ontario, Canada
Sponsors and Collaborators
Taro Pharmaceuticals USA
  More Information

Publications:
Responsible Party: Taro Pharmaceuticals USA
ClinicalTrials.gov Identifier: NCT00506012     History of Changes
Other Study ID Numbers: T2000-0633
Study First Received: July 20, 2007
Last Updated: December 19, 2013
Health Authority: Canada: Health Canada

Keywords provided by Taro Pharmaceuticals USA:
Myoclonus Dystonia
Essential Myoclonus
Inherited Myoclonus
Dyskinesia
Movement Disorder

Additional relevant MeSH terms:
Dystonia
Dystonic Disorders
Myoclonus
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases

ClinicalTrials.gov processed this record on April 14, 2014