L-citrulline and Metformin in Duchenne's Muscular Dystrophy

Phase 3CompletedNCT01995032

University Hospital, Basel, Switzerland47 enrolled

Overview

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

This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits. Amendment 1: Amended eligibility criteria

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Duchenne's Muscular Dystrophy (DMD)

Interventions

750 mg metformin and 7.5 g L-citrulline daily p.o.DRUG

PlaceboDRUG

Eligibility

Sex: ALLMin age: 78 MonthsMax age: 10 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Molecular diagnosis of DMD * Patients 6.5 - 10 years of age at time of screening * Ambulant * Ability to walk 150 m in the 6 min walking distance (6MWT) * D1 subdomain of the MFM scale \>40% * stable treatment with steroids for \>6 months or steroid naïve patients Exclusion Criteria: * Previous (3 months or less) or concomitant participation in another therapeutic trial * Use of L-citrulline, L-arginine or metformin within the last 3 months * Known individual hypersensitivity to L-citrulline or metformin * known or suspected malignancy * Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator * start of cortisone treatment or change in dosage \<6 months prior to screening

Locations (1)

University Children's Hospital

Basel, Canton of Basel-City, Switzerland