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L-citrulline and Metformin in Duchenne’s Muscular Dystrophy
study id #: NCT01995032
condition: Duchenne's Muscular Dystrophy (DMD)
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.
intervention: 750 mg metformin and 7.5 g L-citrulline daily p.o., Placebo
mechanism of action: Muscle cell engery regulators to prevent muscle loss
last updated: November 22, 2018
start date: October 2013
estimated completion: March 2016
phase of development: Phase 3
size / enrollment: 47
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.
Amendment 1: Amended eligibility criteria
- Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [ Time Frame: baseline to week 26 ]
- 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 ]
• Molecular diagnosis of DMD
• Patients 6.5 - 10 years of age at time of screening
• Ability to walk 150 m in the 6 min walking distance (6MWT)
• D1 subdomain of the MFM scale >40%
• stable treatment with steroids for >6 months or steroid naïve patients
• Previous (3 months or less) or concomitant participation in another therapeutic trial
• Use of L-citrulline, L-arginine or metformin within the last 3 months
• Known individual hypersensitivity to L-citrulline or metformin
• known or suspected malignancy
• Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
• start of cortisone treatment or change in dosage <6 months prior to screening
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