Duchenne's Muscular Dystrophy (DMD)
Conditions
Keywords
Duchenne's muscular dystrophy, DMD
Brief summary
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.
Detailed description
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
Interventions
DRUG750 mg metformin and 7.5 g L-citrulline daily p.o.
DRUGPlacebo
Sponsors
University Hospital, Basel, Switzerland
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
78 Months to 10 Years
Healthy volunteers
No
Inclusion criteria
* Molecular diagnosis of DMD * Patients 6.5 - 10 years of age at time of screening * Ambulant * 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) | baseline to week 26 |
Secondary
| Measure | Time frame |
|---|---|
| Mean change of MFM total score, the D2, and D3 MFM subscores | baseline to week 26 |
| Mean change of six minute walking distance (6MWD) | baseline to week 26 |
| Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles | baseline to week 26 |
| Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA) | baseline to week 26 |
| Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) | baseline to week 26 |
Countries
Switzerland
Outcome results
None listed