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

Phase 2TerminatedNCT01074359

Penwest Pharmaceuticals Co.30 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Neuromuscular Disease

Interventions

A0001 (alpha-tocopherolquinone)DRUG

28 days (1.5 g total daily dose) oral A0001 capsules. Treatment taken twice daily with meals.

PlaceboDRUG

28 days of placebo oral capsules. Treatment taken twice daily with meals.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation * PCR/ATP ratio of \<1.9 following the Cardiac MRS at screening Exclusion Criteria: * Any major illness not due to the A3243G mitochondrial DNA point mutation in the past three months or any significant ongoing chronic medical illness, especially significant central nervous neurological disease limiting capacity to carry out the study * Use of any investigational product within the past 30 days

Locations (1)

University of Newcastle upon Tyne

Newcastle, Framlington Place, United Kingdom

Outcomes

Primary Outcomes

Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS)