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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-02-28

Study Completion Date

2010-11-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Neuromuscular Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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A0001

A0001 (0.75 g BID)

Group Type EXPERIMENTAL

A0001 (alpha-tocopherolquinone)

Intervention Type DRUG

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

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

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A0001 (alpha-tocopherolquinone)

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

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No