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completed

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

key information

study id #: NCT02516085

condition: Duchenne Muscular Dystrophy

status: completed

purpose:

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.

intervention: Metformin, L-Arginine

mechanism of action: Muscle cell engery regulators to prevent muscle loss

results: https://clinicaltrials.gov/ct2/show/results/NCT02516085

last updated: November 22, 2018

study details

start date: January 2012

estimated completion: October 2012

phase of development: Phase 1

size / enrollment: 5

study 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.

primary outcomes:

  • Mean change of muscle metabolism [ Time Frame: baseline to week 16 ]
    mitochondrial protein expression analysis in muscular biopsies

secondary outcomes:

  • In vivo change of muscle metabolism [ Time Frame: baseline to week 16 ]
    indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance

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 contacts

sponsor: University Hospital, Basel, Switzerland

investigators: Dirk Fischer, MD

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