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

University Hospital Basel

Status and phase: Completed; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: L-Arginine; Drug: Metformin

Study type: Interventional

Funder types: Other

Identifiers

NCT02516085

DMD01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 5 patients

Sex: All

Ages: 7 to 10 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

L-arginine and metformin

Experimental group

Description:

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

Treatment:

Drug: Metformin

Drug: L-Arginine