Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin

• Sponsor: University Hospital, Basel, Switzerland (Other)
• Overall Status: Completed
• CT.gov ID: NCT02516085
• Collaborator: (none)
• Enrollment: 5
• Arm: 1
• Duration (Months): 9

Study Details

Study Description

Brief Summary

The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Drug: Metformin; Drug: L-Arginine	Phase 1

Detailed Description

This is an investigator-initiated, open-label, single-center, proof-of-concept-study. The study medication consists of L-arginine and metformin. The duration of the study is 16 weeks and comprehends one screening and four study visits.

Study Design

• Study Type: Interventional
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Study Start Date: Jan 1, 2012
• Actual Primary Completion Date: Oct 1, 2012
• Actual Study Completion Date: Oct 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: L-arginine and metformin 7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks	Drug: Metformin; Drug: L-Arginine

Outcome Measures

Primary Outcome Measures

1. Mean change of muscle metabolism [baseline to week 16]

   mitochondrial protein expression analysis in muscular biopsies

Secondary Outcome Measures

1. In vivo change of muscle metabolism [baseline to week 16]

   indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance

Eligibility Criteria

Criteria

• Ages Eligible for Study: 7 Years to 10 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Molecular diagnosis of DMD

• Patients 7 - 10 years of age at time of screening

• Ambulant

Exclusion Criteria:

• Previous (3 months or less) or concomitant participation in another therapeutic trial

• Use of L-arginine, L-citrulline or metformin within the last 3 months

• Known individual hypersensitivity to L-citrulline or metformin

• Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of the investigator

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• University Hospital, Basel, Switzerland

Investigators

• Principal Investigator: Dirk Fischer, MD, University of Basel, Children's Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Dirk Fischer, MD, University Hospital, Basel, Switzerland
• ClinicalTrials.gov Identifier: NCT02516085
• Other Study ID Numbers: DMD01
• First Posted: Aug 5, 2015
• Last Update Posted: Aug 7, 2015
• Last Verified: Aug 1, 2015

• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Metformin

Study Results

No Results Posted as of Aug 7, 2015