L-citrulline and Metformin in Becker's Muscular Dystrophy

Becker's Muscular Dystrophy (BMD) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02018731
• Other study ID #: BMD01
• Status: Completed
• Phase: Phase 2
• First received: December 17, 2013
• Last updated: May 13, 2016
• Start date: June 2013
• Est. completion date: December 2015

Study information

• Verified date: May 2016
• Source: University Hospital, Basel, Switzerland
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: Swissmedic
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: December 2015
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Becker's Muscular Dystrophy (BMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Metformin and Metformin & L-Citrulline

• L-Citrulline and Metformin & L-Citrulline

Locations

Country	Name	City	State
Switzerland	University Children's Hospital	Basel	BS

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Basel, Switzerland

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks		week 6 and week 12	No
Secondary	MFM total score and six minute walking distance (6MWD)		week 6 and week 12	No
Secondary	Change of muscle fat content (MFC) (assessed by MRI)		week 6 and week 12	No
Secondary	Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)		week 6 and week 12	No
Secondary	Change of laboratory parameters (oxidative and nitrosative stress)		week 6 and week 12	No