L-citrulline and Metformin in Duchenne's Muscular Dystrophy

University Hospital Basel

Status and phase: Completed; Phase 3

Conditions: Duchenne's Muscular Dystrophy (DMD)

Treatments: Drug: Placebo; Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.

Study type: Interventional

Funder types: Other

Identifiers

NCT01995032

DMD02

Details and patient eligibility

About

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.

Full description

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

Enrollment: 47 patients

Sex: All

Ages: 78 months to 10 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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47 participants in 2 patient groups, including a placebo group

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

Experimental group

Description:

7.5 g L-citrulline p.o. and 750 mg metformin daily p.o. (3x 2.5 g, respectively 3x 250 mg) for 26 weeks

Treatment:

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

Placebo

Placebo Comparator group

Description:

metformin placebo and L-citrulline placebo 3 times daily p.o. for 26 weeks

Treatment:

Drug: Placebo