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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02516085
Recruitment Status : Completed
First Posted : August 5, 2015
Last Update Posted : August 7, 2015
Sponsor:
Information provided by (Responsible Party):
Dirk Fischer, University Hospital, Basel, Switzerland

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Metformin Drug: L-Arginine Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : January 2012
Actual Primary Completion Date : October 2012
Actual Study Completion Date : October 2012


Arm Intervention/treatment
Experimental: 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
Drug: Metformin
Drug: L-Arginine



Primary Outcome Measures :
  1. Mean change of muscle metabolism [ Time Frame: baseline to week 16 ]
    mitochondrial protein expression analysis in muscular biopsies


Secondary Outcome Measures :
  1. 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



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Ages Eligible for Study:   7 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02516085


Sponsors and Collaborators
University Hospital, Basel, Switzerland
Investigators
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Principal Investigator: Dirk Fischer, MD University of Basel, Children's Hospital
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Dirk Fischer, MD, University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier: NCT02516085    
Other Study ID Numbers: DMD01
First Posted: August 5, 2015    Key Record Dates
Last Update Posted: August 7, 2015
Last Verified: August 2015
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Metformin
Hypoglycemic Agents
Physiological Effects of Drugs