Duchenne Muscular DystrophyPhase 1NCT02516085
Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
A Phase 1 study testing Metformin and L-Arginine in people with Duchenne Muscular Dystrophy, run by University Hospital, Basel, Switzerland.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 to 10 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Metformin and L-Arginine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- Jan 2012to Oct 2012
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Molecular diagnosis of DMD
- Patients 7 - 10 years of age at time of screening
- Ambulant
Who cannot join
- 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
About this study
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.
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Metformin, L-Arginine
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetforminDrug
L-ArginineDrug
| Study group | Role | What they receive |
|---|---|---|
| L-arginine and metformin | Experimental | 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 Receives: Drug: Metformin; Drug: L-Arginine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change of muscle metabolism
mitochondrial protein expression analysis in muscular biopsies
Measured: baseline to week 16
Secondary outcomes (1)
In vivo change of muscle metabolism
indirect calorimetry, Dual-Energy X-Ray Absorptiometry, quantitative thigh muscle MRI, clinical score of muscle performance
Measured: baseline to week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2015 (estimated)
- Study startJan 2012
- Primary completionOct 2012
- Study completionOct 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dirk Fischer, University Hospital, Basel, Switzerland
- Investigators
- Dirk Fischer, MD, Principal investigator, University of Basel, Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02516085
- Sponsor's protocol ID
- DMD01
- Status last verified
- Aug 2015
- First submitted
- 3 Aug 2015
- Last updated
- 7 Aug 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 26799743
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
- ClinicalTrials.gov ID
- NCT02516085
- Acronym / sponsor ID
- DMD01
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- Oct 2012 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 5 Aug 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| L-arginine and metformin | Experimental | Drug: Metformin, Drug: L-Arginine7.5 g L-arginine p.o. and 500 mg metformin p.o. per day (3x 2.5 g, respectively 3x 250 mg) for 16 weeks |
Who may qualify
- Ages
- 7 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Molecular diagnosis of DMD
- Patients 7 - 10 years of age at time of screening
- Ambulant
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Metformin | Drug | L-arginine and metformin |
| L-Arginine | Drug | L-arginine and metformin |
Primary outcome
Mean change of muscle metabolism
Time frame: baseline to week 16
mitochondrial protein expression analysis in muscular biopsies
1 secondary outcome
In vivo change of muscle metabolism
Time frame: baseline to week 16
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2015.
- Derived from this study: PubMed 26799743Hafner 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dirk Fischer, MD principal investigator
University of Basel, Children's Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Metformin and L-Arginine in people with Duchenne Muscular Dystrophy, run by University Hospital, Basel, Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Aug 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change of muscle metabolism (time frame: baseline to week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02516085, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2015.