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Safety and Efficacy Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation

Neuromuscular Disease Clinical Trial

Official title:
A Phase 2a, Double Blind, Randomized, Placebo-controlled, 28 Day, Two-arm, Parallel Group Study of A0001 in Patients With the A3243G Mitochondrial DNA Point Mutation and Evidence of Impaired Mitochondrial Function

Clinical Trial Summary

This is a phase 2a, double-blind, placebo-controlled, single-center study. Twenty-one patients who qualify for the study will be randomly assigned to either active drug or placebo. The study will take place at Newcastle University. Patients will have a 66% chance of getting active drug. Patients will be required to take study treatment orally twice a day for 28 days. A baseline visit will occur within 21 days of screening visit. All patients will be followed for 1 week after completion of study or early withdrawal from the study.

Clinical Trial Description

n/a

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01074359
Study type Interventional
Source Penwest Pharmaceuticals Co.
Contact
Status Terminated
Phase Phase 2
Start date February 2010
Completion date November 2010
View Details
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