Pilot Efficacy Study of T2000 in Myoclonus Dystonia

NCT00506012 | Terminated Phase 2

• 1 other identifier: T2000-0633
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Myoclonus

Keywords

Myoclonus Dystonia; Essential Myoclonus; Inherited Myoclonus; Dyskinesia; Movement Disorder

Outcome Measures

Primary Outcomes (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.

  Up to 12 weeks

Secondary Outcomes (1)

• Safety parameters including neurological examination, blood tests and EKG will be monitored throughout the treatment period and during withdrawal of the medication.

  Up to 16 weeks

Study Arms (1)

1

EXPERIMENTAL

T2000

Drug: T2000

Interventions

T2000 DRUG

T2000 at doses of 200 mg a day to 1000 mg a day

1

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Sun Pharmaceutical Industries, Inc.: lead

Study Sites (1)

Investigator Site

Toronto, Ontario, Canada

Location

Related Publications (1)

• Melmed C, Moros D, Rutman H. Treatment of essential tremor with the barbiturate T2000 (1,3-dimethoxymethyl-5,5-diphenyl-barbituric acid). Mov Disord. 2007 Apr 15;22(5):723-7. doi: 10.1002/mds.21321.

  PMID: 17265458 BACKGROUND

MeSH Terms

Conditions

Myoclonus; Myoclonic dystonia; Dyskinesias; Movement Disorders

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Central Nervous System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 20, 2007
• First Posted: July 25, 2007
• Study Start: August 1, 2007
• Primary Completion: August 1, 2011
• Study Completion: October 1, 2011
• Last Updated: December 23, 2013

Record last verified: 2013-11

Locations

CA (1)