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

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.

Study Overview

• Status: Completed
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: Metformin; Drug: L-Arginine

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 Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change of muscle metabolism	mitochondrial protein expression analysis in muscular biopsies	baseline to week 16

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
In vivo change of muscle metabolism	indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance	baseline to week 16

Collaborators and Investigators

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

Publications and helpful links

General Publications

• Hafner P, Bonati U, Erne B, Schmid M, Rubino D, Pohlman U, Peters T, Rutz E, Frank S, Neuhaus C, Deuster S, Gloor M, Bieri O, Fischmann A, Sinnreich M, Gueven N, Fischer D. Improved Muscle Function in Duchenne Muscular Dystrophy through L-Arginine and Metformin: An Investigator-Initiated, Open-Label, Single-Center, Proof-Of-Concept-Study. PLoS One. 2016 Jan 22;11(1):e0147634. doi: 10.1371/journal.pone.0147634. eCollection 2016.

Study record dates

Study Major Dates

• Study Start: January 1, 2012
• Primary Completion (Actual): October 1, 2012
• Study Completion (Actual): October 1, 2012

Study Registration Dates

• First Submitted: August 3, 2015
• First Submitted That Met QC Criteria: August 4, 2015
• First Posted (Estimate): August 5, 2015

Study Record Updates

• Last Update Posted (Estimate): August 7, 2015
• Last Update Submitted That Met QC Criteria: August 5, 2015
• Last Verified: August 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne; Hypoglycemic Agents; Physiological Effects of Drugs; Metformin
• Other Study ID Numbers: DMD01