Back to resultsL-citrulline and Metformin in Becker's Muscular Dystrophy
Primary Purpose
Becker's Muscular Dystrophy (BMD)
Status
Completed
Phase
Phase 2
Locations
Switzerland
Study Type
Interventional
Intervention
Metformin and Metformin & L-Citrulline
L-Citrulline and Metformin & L-Citrulline
Sponsored by
About this trial
This is an interventional treatment trial for Becker's Muscular Dystrophy (BMD) focused on measuring Becker's Muscular dystrophy, BMD, Metformin, L-citrulline
Eligibility 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
Sites / Locations
- University Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Metformin and Metformin & L-Citrulline
L-Citrulline and Metformin & L-Citrulline
Arm Description
1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
Outcomes
Primary Outcome Measures
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
Secondary Outcome Measures
MFM total score and six minute walking distance (6MWD)
Change of muscle fat content (MFC) (assessed by MRI)
Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)
Change of laboratory parameters (oxidative and nitrosative stress)
Full Information
NCT ID
NCT02018731
First Posted
December 17, 2013
Last Updated
May 13, 2016
Sponsor
University Hospital, Basel, Switzerland
1. Study Identification
Unique Protocol Identification Number
NCT02018731
Brief Title
L-citrulline and Metformin in Becker's Muscular Dystrophy
Official Title
Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
May 2016
Overall Recruitment Status
Completed
Study Start Date
June 2013 (undefined)
Primary Completion Date
September 2014 (Actual)
Study Completion Date
December 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Basel, Switzerland
4. Oversight
Data Monitoring Committee
No
5. Study Description
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Becker's Muscular Dystrophy (BMD)
Keywords
Becker's Muscular dystrophy, BMD, Metformin, L-citrulline
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Metformin and Metformin & L-Citrulline
Arm Type
Experimental
Arm Description
1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
Arm Title
L-Citrulline and Metformin & L-Citrulline
Arm Type
Experimental
Arm Description
15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
Intervention Type
Drug
Intervention Name(s)
Metformin and Metformin & L-Citrulline
Intervention Type
Drug
Intervention Name(s)
L-Citrulline and Metformin & L-Citrulline
Primary Outcome Measure Information:
Title
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
Time Frame
week 6 and week 12
Secondary Outcome Measure Information:
Title
MFM total score and six minute walking distance (6MWD)
Time Frame
week 6 and week 12
Title
Change of muscle fat content (MFC) (assessed by MRI)
Time Frame
week 6 and week 12
Title
Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)
Time Frame
week 6 and week 12
Title
Change of laboratory parameters (oxidative and nitrosative stress)
Time Frame
week 6 and week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dirk Fischer, MD
Organizational Affiliation
University Children's Hospital Basel
Official's Role
Principal Investigator
Facility Information:
Facility Name
University Children's Hospital
City
Basel
State/Province
BS
ZIP/Postal Code
4031
Country
Switzerland
12. IPD Sharing Statement
Learn more about this trial
L-citrulline and Metformin in Becker's Muscular Dystrophy
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