Myoclonus Clinical Trial
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
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.
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.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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