Duchenne Muscular Dystrophy
Conditions
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.
Interventions
DRUGMetformin
DRUGL-Arginine
Sponsors
University Hospital, Basel, Switzerland
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to 10 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean change of muscle metabolism | baseline to week 16 | mitochondrial protein expression analysis in muscular biopsies |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Outcome results
None listed