Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
- Registration Number
- NCT02516085
- Lead Sponsor
- University Hospital, Basel, Switzerland
- Brief Summary
The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.
- Detailed Description
This is an investigator-initiated, open-label, single-center, proof-of-concept-study. The study medication consists of L-arginine and metformin. The duration of the study is 16 weeks and comprehends one screening and four study visits.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 5
Inclusion Criteria
- Molecular diagnosis of DMD
- Patients 7 - 10 years of age at time of screening
- Ambulant
Exclusion Criteria
- Previous (3 months or less) or concomitant participation in another therapeutic trial
- Use of L-arginine, L-citrulline or metformin within the last 3 months
- Known individual hypersensitivity to L-citrulline or metformin
- Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of the investigator
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description L-arginine and metformin Metformin 7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks L-arginine and metformin L-Arginine 7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks
- Primary Outcome Measures
Name Time Method Mean change of muscle metabolism baseline to week 16 mitochondrial protein expression analysis in muscular biopsies
- Secondary Outcome Measures
Name Time Method In vivo change of muscle metabolism baseline to week 16 indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance