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

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Metformin
L-Arginine
Sponsored by
University Hospital, Basel, Switzerland
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

7 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    L-arginine and metformin

    Arm Description

    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

    Outcomes

    Primary Outcome Measures

    Mean change of muscle metabolism
    mitochondrial protein expression analysis in muscular biopsies

    Secondary Outcome Measures

    In vivo change of muscle metabolism
    indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance

    Full Information

    First Posted
    August 3, 2015
    Last Updated
    August 5, 2015
    Sponsor
    University Hospital, Basel, Switzerland
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02516085
    Brief Title
    Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2015
    Overall Recruitment Status
    Completed
    Study Start Date
    January 2012 (undefined)
    Primary Completion Date
    October 2012 (Actual)
    Study Completion Date
    October 2012 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    University Hospital, Basel, Switzerland

    4. Oversight

    Data Monitoring Committee
    Yes

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Duchenne Muscular Dystrophy

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    5 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    L-arginine and metformin
    Arm Type
    Experimental
    Arm Description
    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
    Intervention Type
    Drug
    Intervention Name(s)
    Metformin
    Intervention Type
    Drug
    Intervention Name(s)
    L-Arginine
    Primary Outcome Measure Information:
    Title
    Mean change of muscle metabolism
    Description
    mitochondrial protein expression analysis in muscular biopsies
    Time Frame
    baseline to week 16
    Secondary Outcome Measure Information:
    Title
    In vivo change of muscle metabolism
    Description
    indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance
    Time Frame
    baseline to week 16

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    7 Years
    Maximum Age & Unit of Time
    10 Years
    Accepts Healthy Volunteers
    No
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Dirk Fischer, MD
    Organizational Affiliation
    University of Basel, Children's Hospital
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    26799743
    Citation
    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.
    Results Reference
    derived

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

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