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Sponsored by: |
Taro Pharmaceuticals USA |
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Information provided by: | Taro Pharmaceuticals USA |
ClinicalTrials.gov Identifier: | NCT00506012 |
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 II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Dose Comparison, Single Group Assignment, Safety/Efficacy Study |
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 |
Estimated Enrollment: | 5 |
Study Start Date: | August 2007 |
Estimated Study Completion Date: | March 2010 |
Estimated Primary Completion Date: | December 2009 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
---|---|
1: Experimental
T2000
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Drug: T2000
T2000 at doses of 200 mg a day to 1000 mg a day
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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.
Ages Eligible for Study: | 18 Years to 75 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients should meet diagnostic criteria for M-D based on the following criteria:
Exclusion Criteria:
Contact: Julie So, RN | 416-603-5875 ext 3 | jso@uhnres.utoronto.ca |
Canada, Ontario | |
Movement Disorder Clinic: University Health Network; Toronto Western Hospital | Recruiting |
Toronto, Ontario, Canada, M5R 2N5 | |
Contact: Julie So, RN 416-603-5875 ext 3 jso@uhnres.utoronto.ca | |
Principal Investigator: Anthony E Lang, MD FRCPC |
Principal Investigator: | Anthony E Lang, MD FRCPC | University Health Network - Toronto Western Hospital |
Responsible Party: | Taro Pharmaceuticals USA ( Medical Director ) |
Study ID Numbers: | T2000-0633 |
Study First Received: | July 20, 2007 |
Last Updated: | January 8, 2009 |
ClinicalTrials.gov Identifier: | NCT00506012 |
Health Authority: | Canada: Health Canada |
Myoclonus Dystonia Essential Myoclonus Inherited Myoclonus Dyskinesia Movement Disorder |
Dystonic Disorders Signs and Symptoms Myoclonus Promethazine Movement Disorders |
Neurologic Manifestations Dystonia Dyskinesias Diphenhydramine |
Nervous System Diseases |