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).
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
University Children's Hospital
Basel, Canton of Basel-City, Switzerland
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
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