L-citrulline and Metformin in Duchenne's Muscular Dystrophy

Summary

The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.

Timeline

Start

2013-10

Primary completion

2015-10

Completion

2016-03

Outcome

Missed primary endpoint

paper subscore decrease was 5.5% greater than that of patients receiving placebo (95% CI, -1.0% to 12.1%; P = .09). PMID 31664444 ↗

paper Treatment with combination therapy was not associated with an overall reduction in motor function decline among ambulant patients with DMD; PMID 31664444 ↗

Publications

• Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D. Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Netw Open. 2019 Oct 2;2(10):e1914171. doi: 10.1001/jamanetworkopen.2019.14171.
• Hafner P, Bonati U, Rubino D, Gocheva V, Zumbrunn T, Gueven N, Fischer D. Treatment with L-citrulline and metformin in Duchenne muscular dystrophy: study protocol for a single-centre, randomised, placebo-controlled trial. Trials. 2016 Aug 3;17(1):389. doi: 10.1186/s13063-016-1503-1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	L-citrulline	Other / unclassified	2.5 g	Oral
Subject	Metformin	Small molecule	250 mg	Oral

Indications