NCT01074359

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

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Penwest Pharmaceuticals Co. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Neuromuscular Disease

Interventions

Type	Name	Description
DRUG	A0001 (alpha-tocopherolquinone)	28 days (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.
DRUG	Placebo	28 days of placebo oral capsules. Treatment taken twice daily with meals.

Timeline

• Start date: 2010-02-01
• Primary completion: 2010-11-01
• Completion: 2010-11-01
• First posted: 2010-02-24
• Last updated: 2011-04-22

Locations

1 site across 1 country: United Kingdom