L-citrulline and Metformin in Becker's Muscular Dystrophy

Study Overview
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Brief Summary
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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).

Detailed Description
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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.

Official Title
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Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy

Conditions
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Becker's Muscular Dystrophy (BMD)

Intervention / Treatment
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* Drug: Metformin and Metformin & L-Citrulline
* Drug: L-Citrulline and Metformin & L-Citrulline


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 years or older Molecular or immunohistochemical diagnosis of BMD ambulant at the time point of screening Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Metformin and Metformin & L-Citrulline<br>1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks | Drug: Metformin and Metformin & L-Citrulline<br> <br> |
| Experimental: L-Citrulline and Metformin & L-Citrulline<br>15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks | Drug: L-Citrulline and Metformin & L-Citrulline<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks |  | week 6 and week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| MFM total score and six minute walking distance (6MWD) |  | week 6 and week 12 | 
| Change of muscle fat content (MFC) (assessed by MRI) |  | week 6 and week 12 | 
| Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) |  | week 6 and week 12 | 
| Change of laboratory parameters (oxidative and nitrosative stress) |  | week 6 and week 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Becker's Muscular dystrophy, BMD, Metformin, L-citrulline