Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
1 other identifier
interventional
5
0 countries
N/A
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Jan 2012
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2012
CompletedFirst Submitted
Initial submission to the registry
August 3, 2015
CompletedFirst Posted
Study publicly available on registry
August 5, 2015
CompletedAugust 7, 2015
August 1, 2015
9 months
August 3, 2015
August 5, 2015
Conditions
Outcome Measures
Primary Outcomes (1)
Mean change of muscle metabolism
mitochondrial protein expression analysis in muscular biopsies
baseline to week 16
Secondary Outcomes (1)
In vivo change of muscle metabolism
baseline to week 16
Study Arms (1)
L-arginine and metformin
EXPERIMENTAL7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks
Interventions
Eligibility Criteria
You may qualify if:
- Molecular diagnosis of DMD
- Patients 7 - 10 years of age at time of screening
- Ambulant
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Related Publications (1)
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.
PMID: 26799743DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Dirk Fischer, MD
University of Basel, Children's Hospital
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- MD
Study Record Dates
First Submitted
August 3, 2015
First Posted
August 5, 2015
Study Start
January 1, 2012
Primary Completion
October 1, 2012
Study Completion
October 1, 2012
Last Updated
August 7, 2015
Record last verified: 2015-08