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

Duchenne Muscular Dystrophy Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02516085
• Other study ID #: DMD01
• Status: Completed
• Phase: Phase 1
• First received: August 3, 2015
• Last updated: August 5, 2015
• Start date: January 2012
• Est. completion date: October 2012

Study information

• Verified date: August 2015
• Source: University Hospital, Basel, Switzerland
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: Swissmedic
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years to 10 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• Metformin

• L-Arginine

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Basel, Switzerland

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change of muscle metabolism	mitochondrial protein expression analysis in muscular biopsies	baseline to week 16	No
Secondary	In vivo change of muscle metabolism	indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance	baseline to week 16	No