High Dose Ascorbic Acid Treatment of CMT1A

Phase 2/3 Completed 110 enrolled Wayne State University Charcot-Marie-Tooth Association · collab Muscular Dystrophy Association · collab

RandomizedParallel-groupTriple-blindTreatment

Summary

This study will look at the impact of ascorbic acid (Vitamin C) on the progression of disease in people with CMT1A as compared to volunteers receiving a placebo. This study will assess whether is it futile to proceed with a larger, longer-term, placebo-controlled study.

Timeline

Start: 2007-04
Primary completion: 2012-12
Completion: 2012-12

Outcome

Missed primary endpoint

paper This was well below 50% reduction of CMTNS worsening from natural history, so futility could not be declared (P > .99). PMID 23797954 ↗

paper It seems unlikely that our results support undertaking a larger trial of 4-g/d AA treatment in subjects with CMT1A. PMID 23797954 ↗

Publications

Lewis RA, McDermott MP, Herrmann DN, Hoke A, Clawson LL, Siskind C, Feely SM, Miller LJ, Barohn RJ, Smith P, Luebbe E, Wu X, Shy ME; Muscle Study Group. High-dosage ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A: results of a randomized, double-masked, controlled trial. JAMA Neurol. 2013 Aug;70(8):981-7. doi: 10.1001/jamaneurol.2013.3178.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Vitamin C	Other / unclassified	4 g	Oral