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Pilot Efficacy Study of T2000 in Myoclonus Dystonia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00506012
Recruitment Status : Terminated (Slow Recruitment of eligible patients)
First Posted : July 25, 2007
Last Update Posted : December 23, 2013
Information provided by (Responsible Party):
Taro Pharmaceuticals USA

Brief Summary:
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 or disease Intervention/treatment Phase
Myoclonus Drug: T2000 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2007
Actual Primary Completion Date : August 2011
Actual Study Completion Date : October 2011

Arm Intervention/treatment
Experimental: 1
Drug: T2000
T2000 at doses of 200 mg a day to 1000 mg a day

Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00506012

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

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Responsible Party: Taro Pharmaceuticals USA Identifier: NCT00506012    
Other Study ID Numbers: T2000-0633
First Posted: July 25, 2007    Key Record Dates
Last Update Posted: December 23, 2013
Last Verified: November 2013
Keywords provided by Taro Pharmaceuticals USA:
Myoclonus Dystonia
Essential Myoclonus
Inherited Myoclonus
Movement Disorder
Additional relevant MeSH terms:
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Dystonic Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases