L-citrulline and Metformin in Becker's Muscular Dystrophy | RxWiki

L-citrulline and Metformin in Becker's Muscular Dystrophy

Overview[ - collapse ][ - ]

Purpose	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).
Condition	Becker's Muscular Dystrophy (BMD)
Intervention	Drug: Metformin and Metformin & L-Citrulline Drug: L-Citrulline and Metformin & L-Citrulline
Phase	Phase 2
Sponsor	University Hospital, Basel, Switzerland
Responsible Party	University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier	NCT02018731
First Received	December 17, 2013
Last Updated	December 17, 2013
Last verified	December 2013

Tracking Information[ + expand ][ + ]

First Received Date	December 17, 2013
Last Updated Date	December 17, 2013
Start Date	June 2013
Estimated Primary Completion Date	December 2016
Current Primary Outcome Measures	Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks [Time Frame: week 6 and week 12] [Designated as safety issue: No]
Current Secondary Outcome Measures	MFM total score and six minute walking distance (6MWD) [Time Frame: week 6 and week 12] [Designated as safety issue: No] Change of muscle fat content (MFC) (assessed by MRI) [Time Frame: week 6 and week 12] [Designated as safety issue: No] Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) [Time Frame: week 6 and week 12] [Designated as safety issue: No] Change of laboratory parameters (oxidative and nitrosative stress) [Time Frame: week 6 and week 12] [Designated as safety issue: No]

Descriptive Information[ + expand ][ + ]

Brief Title	L-citrulline and Metformin in Becker's Muscular Dystrophy
Official Title	Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
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 Type	Interventional
Study Phase	Phase 2
Study Design	Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Condition	Becker's Muscular Dystrophy (BMD)
Intervention	Drug: Metformin and Metformin & L-Citrulline Drug: L-Citrulline and Metformin & L-Citrulline
Study Arm (s)	Experimental: 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 Experimental: 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

Recruitment Information[ + expand ][ + ]

Recruitment Status	Recruiting
Estimated Enrollment	20
Estimated Completion Date	December 2016
Estimated Primary Completion Date	December 2014
Eligibility Criteria	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
Gender	Both
Ages	18 Years
Accepts Healthy Volunteers	No
Contacts	Contact: Ulrike Bonati, MD +41617042243 ulrike.bonati@ukbb.ch
Location Countries	Switzerland

Administrative Information[ + expand ][ + ]

NCT Number	NCT02018731
Other Study ID Numbers	BMD01
Has Data Monitoring Committee	No
Information Provided By	University Hospital, Basel, Switzerland
Study Sponsor	University Hospital, Basel, Switzerland
Collaborators	Not Provided
Investigators	Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
Verification Date	December 2013

Locations[ + expand ][ + ]