MEL01 v1.0: A0001 in Patients with A3243G Mitochondrial DNA Mutation

• Research type

  Research Study

• Full title

  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

• IRAS ID

  20203

• Contact name

  Patrick F Chinnery

• Sponsor organisation

  Penwest Pharmaceuticals Co.

• Eudract number

  2009-014263-40

• ISRCTN Number

  Not available

• Research summary

  This clinical study is being conducted by Penwest Pharmaceuticals Co. to determine the safety and efficacy of A0001 in patients with the A3243G mitochondrial DNA point mutation and evidence of impaired mitochondrial function. Patients with this mutation exhibit abnormalities in blood chemistry parameters related to oxidative metabolism (decreased level of adenosine-5'-triphosphate (ATP)) that's accentuated by exercise. There is currently no treatment. The research question being addressed is whether A0001 can improve the rate of ATP recovery in the cardiac muscle as measured by 31Phosphorous Magnetic Resonance Spectroscopy during exercise.This is a phase 2a, double-blind, placebo-controlled, single-centre study. Approximately 30 patients will be recruited into the study to ensure 21 are evaluable. 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. Due to the short duration of treatment, it is not expected to provide any sustained therapeutic benefit for participants, although they may benefit from gaining knowledge about their health status through study tests and physician assessments.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  09/H0904/50

• Date of REC Opinion

  14 Dec 2009

• REC opinion

  Further Information Favourable Opinion