Clinical Trial: L-citrulline and Metformin in Duchenne's Muscular Dystrophy
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: "A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscul
Brief 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.
Detailed Summary: This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits.
Sponsor: University Hospital, Basel, Switzerland
Current Primary Outcome: Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [ Time Frame: baseline to week 26 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Mean change of MFM total score, the D2, and D3 MFM subscores [ Time Frame: baseline to week 26 ]
- Mean change of six minute walking distance (6MWD) [ Time Frame: baseline to week 26 ]
- Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles [ Time Frame: baseline to week 26 ]
- Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA) [ Time Frame: baseline to week 26 ]
- Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) [ Time Frame: baseline to week 26 ]
Original Secondary Outcome: Same as current
Information By: University Hospital, Basel, Switzerland
Dates:
Date Received: November 20, 2013
Date Started: October 2013
Date Completion:
Last Updated: August 12, 2016
Last Verified: August 2016