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 - MEL01. A0001 in Patients with A3243G Mitochondrial DNA Mutatio

• Conditions: A3243G mitochondrial DNA point mutation and evidence of impaired mitochondrial function

• Registration Number: EUCTR2009-014263-40-GB
• Lead Sponsor: Penwest Pharmaceuticals Co.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-09-11
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. Patient has been diagnosed as having neuromuscular symptoms due to the A3243G mitochondrial DNA point mutation.
2. Male or female patients of any race within the age range of 18 to 70 years.
3. Sexually active participants and their partners must agree to use two forms of highly effective method of contraception or sterilisation from the time specified prior to screening through 30 days following the last dose of study drug:
- Intra-uterine device (IUD) in place for at least 2 months prior to screening; - Barrier method (condom or diaphragm) with spermicide for at least 14 days prior to screening; - Stable hormonal contraceptive for at least 3 months prior to screening; - Sexually active male patients, including sterilized men, must use a
condom for the duration of the trial.
4. Patient must have <1.9 PCR/ATP ratio following the Cardiac MRS at screening.
5. Willingness to abstain from Idebenone and CoQ10 for 14 days prior to study enrollment and for the duration of the study.
6. No other significant disease, medical history, clinically significant abnormal laboratory values or physical examination findings except for that attributable to the A3243G mitochondrial DNA point mutation and has normal end organ function, in the opinion of the Investigator.
7. Patient can comprehend the nature and purpose of the study and agree to comply with the requirements of the entire protocol.
8. Patient can swallow three size 0 capsules twice daily.
9. Patient has voluntarily signed a Research Ethics Committee approved informed consent form to participate in the study after all relevant aspects of the study have been explained and discussed with the patient.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. 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.
2. Use of any investigational product within the past 30 days.
3. Patients with Dementia as defined by Mini Mental State Exam (MMSE) < 26.
4. Clinically significant renal or liver impairment (ALT, AST > 2.0 x ULN), or prior evidence of a coagulation disorder.
5. History of or current gastro-intestinal diseases influencing drug absorption.
6. Patient is pregnant or lactating.
7. History, within 3 years, of drug abuse (including Benzodiazepines, opioids, amphetamine, cocaine, and THC).
8. History of alcoholism (within two years).
9. Patients taking warfarin or other anticoagulants.
10. Any currently known or expected increased risk of bleeding.
11. Uncontrolled diabetes.
12. Diagnosis of a coagulation disorder or a clinically significant abnormality in INR (>1.25x ULN).
13. Any subject considering or having scheduled any surgical procedure during the study or within a month after administration of the last dose of study.
14. Use of any new prescription drug therapy, or change in dose of existing therapy, within two weeks prior to receiving the first dose of study medication.
15. Known hypersensitivity or allergy to A0001, vitamin E, beta-carotene, or vehicle components or known allergies to drugs of the similar class which, in the opinion of the Investigator, suggests an increased potential for an adverse hypersensitivity to A0001.
16. Any patient judged by the Investigator or Sponsor (or designee) to be inappropriate for the study.
17. Presence of ACC/AHA Stage D cardiac failure at Screening defined as refractory heart failure requiring specialized interventions
18. Abnormal Troponin I level at Screening

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified