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L-citrulline and Metformin in Becker’s Muscular Dystrophy
study id #: NCT02018731
condition: Becker's Muscular Dystrophy (BMD)
The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).
intervention: Metformin and Metformin & L-Citrulline, L-Citrulline and Metformin & L-Citrulline
mechanism of action: Muscle cell engery regulators to prevent muscle loss
start date: June 2013
estimated completion: December 2015
phase of development: Phase 2
size / enrollment: 20
This is a single center, not-randomized open study. The study medication consists of L-citrulline and metformin administered orally; 5g citrulline and 500mg metformin tablets will be given 3 times daily.
Half of the patients will be treated with metformin during the first 6 weeks of the study. The other patients will receive initially L-citrulline for 6 weeks, before all patients will be treated with the combination therapy (metformin and L-citrulline) for another 6 week period.
The study lasts 12 weeks and includes the screening visit and 3 further visits at baseline, week 6 and week 12.
- Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks [ Time Frame: week 6 and week 12 ]
- MFM total score and six minute walking distance (6MWD) [ Time Frame: week 6 and week 12 ]
- Change of muscle fat content (MFC) (assessed by MRI) [ Time Frame: week 6 and week 12 ]
- Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) [ Time Frame: week 6 and week 12 ]
- Change of laboratory parameters (oxidative and nitrosative stress) [ Time Frame: week 6 and week 12 ]
• 18 years or older
• Molecular or immunohistochemical diagnosis of BMD
• ambulant at the time point of screening
• Participation in another therapeutic BMD study within the last 3 months
• Use of L-Arginine, L-Citrulline or Metformin within the last 3 months
• Other chronic disease or relevant limitation of renal, liver, heart function according to discretion of investigator
• known hypersensitivity to L-citrulline or metformin
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