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

NCT ID: NCT02516085

Last Updated: 2015-08-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-31
• Study Completion Date: 2012-10-31

Brief Summary

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.

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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

Group Type: EXPERIMENTAL

Metformin

Intervention Type: DRUG

L-Arginine

Intervention Type: DRUG

Interventions

Metformin

Intervention Type: DRUG

L-Arginine

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Basel, Switzerland

OTHER

Sponsor Role: lead

Responsible Party

Dirk Fischer

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Dirk Fischer, MD

Role: PRINCIPAL_INVESTIGATOR

University of Basel, Children's Hospital

References

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.

Reference Type: DERIVED

PMID: 26799743 (View on PubMed)

Other Identifiers

DMD01

Identifier Type: -

Identifier Source: org_study_id