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

University Hospital, Basel, Switzerland5 enrolled

Overview

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.

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

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

MetforminDRUG

L-ArginineDRUG

Eligibility

Sex: ALLMin age: 7 YearsMax age: 10 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Mean change of muscle metabolism

mitochondrial protein expression analysis in muscular biopsies

Time frame: baseline to week 16

Secondary Outcomes

In vivo change of muscle metabolism

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

Time frame: baseline to week 16

Data from ClinicalTrials.gov

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