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L-citrulline and Metformin in Becker's Muscular Dystrophy

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: NCT02018731
Recruitment Status : Completed
First Posted : December 23, 2013
Last Update Posted : May 16, 2016
Sponsor:
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland

Study Description
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Brief Summary:
The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).

Condition or disease Intervention/treatment Phase
Becker's Muscular Dystrophy (BMD) Drug: Metformin and Metformin & L-Citrulline Drug: L-Citrulline and Metformin & L-Citrulline Phase 2

Detailed Description:

This is a single center, not-randomized open study. The study medication consists of L-citrulline and metformin administered orally; 5g citrulline and 500mg metformin tablets will be given 3 times daily.

Half of the patients will be treated with metformin during the first 6 weeks of the study. The other patients will receive initially L-citrulline for 6 weeks, before all patients will be treated with the combination therapy (metformin and L-citrulline) for another 6 week period.

The study lasts 12 weeks and includes the screening visit and 3 further visits at baseline, week 6 and week 12.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
Study Start Date : June 2013
Actual Primary Completion Date : September 2014
Actual Study Completion Date : December 2015

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Metformin and Metformin & L-Citrulline
1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
 Drug: Metformin and Metformin & L-Citrulline
Experimental: L-Citrulline and Metformin & L-Citrulline
15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
 Drug: L-Citrulline and Metformin & L-Citrulline


Outcome Measures
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Primary Outcome Measures :
  1. Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks [ Time Frame: week 6 and week 12 ]

Secondary Outcome Measures :
  1. MFM total score and six minute walking distance (6MWD) [ Time Frame: week 6 and week 12 ]
  2. Change of muscle fat content (MFC) (assessed by MRI) [ Time Frame: week 6 and week 12 ]
  3. Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) [ Time Frame: week 6 and week 12 ]
  4. Change of laboratory parameters (oxidative and nitrosative stress) [ Time Frame: week 6 and week 12 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 18 years or older
  • Molecular or immunohistochemical diagnosis of BMD
  • ambulant at the time point of screening

Exclusion Criteria:

  • Participation in another therapeutic BMD study within the last 3 months
  • Use of L-Arginine, L-Citrulline or Metformin within the last 3 months
  • Other chronic disease or relevant limitation of renal, liver, heart function according to discretion of investigator
  • known hypersensitivity to L-citrulline or metformin
Contacts and Locations
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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): NCT02018731


Locations
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Switzerland
University Children's Hospital
Basel, BS, Switzerland, 4031
Sponsors and Collaborators
University Hospital, Basel, Switzerland
Investigators
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Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
More Information
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Responsible Party: University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier: NCT02018731    
Other Study ID Numbers: BMD01
First Posted: December 23, 2013    Key Record Dates
Last Update Posted: May 16, 2016
Last Verified: May 2016
Keywords provided by University Hospital, Basel, Switzerland:
Becker's Muscular dystrophy
BMD
Metformin
L-citrulline
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