L-citrulline and Metformin in Becker's Muscular Dystrophy

Phase 2

Completed

• Conditions: Becker's Muscular Dystrophy (BMD)
• Interventions: Drug: Metformin and Metformin & L-Citrulline; Drug: L-Citrulline and Metformin & L-Citrulline

• Registration Number: NCT02018731
• Lead Sponsor: University Hospital, Basel, Switzerland

Brief Summary

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

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 Timeline

• Study Start: 2013-06
• Study First Submitted: 2013-12-17
• Study First Submitted QC: 2013-12-17
• Study First Posted: 2013-12-23
• Primary Completion: 2014-09
• Study Completion: 2015-12
• Status Verified: 2016-05
• Last Update Submitted: 2016-05-13
• Last Update Posted: 2016-05-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Metformin and Metformin & L-Citrulline	Metformin and Metformin & L-Citrulline	1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
L-Citrulline and Metformin & L-Citrulline	L-Citrulline and Metformin & L-Citrulline	15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks

Primary Outcome Measures

Name	Time	Method
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks	week 6 and week 12

Secondary Outcome Measures

Name	Time	Method
Change of laboratory parameters (oxidative and nitrosative stress)	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
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

Trial Locations

Locations (1)

University Children's Hospital; 🇨🇭 Basel, BS, Switzerland