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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT01074359
Other study ID # MEL01
Secondary ID
Status Terminated
Phase Phase 2
First received February 22, 2010
Last updated April 21, 2011
Start date February 2010
Est. completion date November 2010

Study information

Verified date April 2011
Source Penwest Pharmaceuticals Co.
Contact n/a
Is FDA regulated No
Health authority United Kingdom: Medicines and Healthcare Products Regulatory Agency
Study type Interventional

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.


Recruitment information / eligibility

Status Terminated
Enrollment 30
Est. completion date November 2010
Est. primary completion date November 2010
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 70 Years
Eligibility 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

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


Intervention

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

Locations

Country Name City State
United Kingdom University of Newcastle upon Tyne Newcastle Framlington Place

Sponsors (1)

Lead Sponsor Collaborator
Penwest Pharmaceuticals Co.

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Improvement in the rate of ATP recovery ("Vmax") in cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS) Baseline and Day 28 No
Secondary Improvement in cardiac structure and function as measured by Magnetic Resonance Imaging (MRI) Baseline and Day 28 No
Secondary Exercise tolerance as measured by a 6 minute walk test Baseline, Day 14 and Day 28 No
Secondary Improvement in the rate of Maximal ATP recovery (Vmax) as measured by 31Phosphorous Magnetic Resonance Spectroscopy (31P-MRS) MRI of calf muscle during a standardized isolated calf muscle procedure of 2 bouts of plantar flexion exercise Baseline and Day 28 No
Secondary Fasting blood lactate, fasting blood glucose, fasting blood insulin , fasting blood HbA1c levels Baseline, Day 14 and Day 28 No
Secondary Mitochondrial disease severity (NMDAS) Baseline and Day 28 No
Secondary Quality of life (SF-36® Health Survey Questionnaire) Baseline and Day 28 No
Secondary Global impression of clinical severity Baseline, Day 14 and Day 28 No
Secondary Modified fatigue impact scale Baseline, Day 14 and Day 28 No
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