Becker's Muscular Dystrophy (BMD)Phase 2NCT02018731
L-citrulline and Metformin in Becker's Muscular Dystrophy
A Phase 2 study testing Metformin and Metformin & L-Citrulline and L-Citrulline and Metformin & L-Citrulline in people with Becker's Muscular Dystrophy (BMD), run by University Hospital, Basel, Switzerland at 1 site in Switzerland.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Becker's Muscular Dystrophy (BMD)
- What is being tested?
- Metformin and Metformin & L-Citrulline and L-Citrulline and Metformin & L-Citrulline
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteSwitzerland
- Study dates
- Jun 2013to Dec 2015
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years or older
- Molecular or immunohistochemical diagnosis of BMD
- ambulant at the time point of screening
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Switzerland1 site
University Children's Hospital
Basel, Canton of Basel-City, 4031
About this study
Official title: Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
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).
Read the 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.
- Conditions
- Becker's Muscular Dystrophy (BMD)
- Being tested
- Metformin and Metformin & L-Citrulline, L-Citrulline and Metformin & L-Citrulline
- Keywords
- Becker's Muscular dystrophy, BMD, Metformin, L-citrulline
- 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.
Metformin and Metformin & L-CitrullineDrug
L-Citrulline and Metformin & L-CitrullineDrug
| Study group | Role | What they receive |
|---|---|---|
| Metformin and Metformin & L-Citrulline | Experimental | 1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks Receives: Drug: Metformin and Metformin & L-Citrulline |
| L-Citrulline and Metformin & L-Citrulline | Experimental | 15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks Receives: Drug: L-Citrulline and Metformin & L-Citrulline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
Measured: week 6 and week 12
Secondary outcomes (4)
MFM total score and six minute walking distance (6MWD)
Measured: week 6 and week 12
Change of muscle fat content (MFC) (assessed by MRI)
Measured: week 6 and week 12
Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)
Measured: week 6 and week 12
Change of laboratory parameters (oxidative and nitrosative stress)
Measured: week 6 and week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2013 (estimated)
- Study startJun 2013
- Primary completionSep 2014
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker's Muscular Dystrophy (BMD)Studies of the same condition, open and under way.
- More trials run by University Hospital, Basel, SwitzerlandThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dirk Fischer, MD, Principal investigator, University Children's Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02018731
- Sponsor's protocol ID
- BMD01
- Status last verified
- May 2016
- First submitted
- 17 Dec 2013
- Last updated
- 16 May 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02018731
- Acronym / sponsor ID
- BMD01
- Conditions
- Becker's Muscular Dystrophy (BMD)
- Keywords
- Becker's Muscular dystrophy, BMD, Metformin, L-citrulline
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Study start
- Jun 2013
- Primary completion
- Sep 2014 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 23 Dec 2013 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Metformin and Metformin & L-Citrulline | Experimental | Drug: Metformin and Metformin & L-Citrulline1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks |
| L-Citrulline and Metformin & L-Citrulline | Experimental | Drug: L-Citrulline and Metformin & L-Citrulline15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 18 years or older
- Molecular or immunohistochemical diagnosis of BMD
- ambulant at the time point of screening
Key exclusion criteria (4 listed)
- 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
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 and Metformin & L-Citrulline | Drug | Metformin and Metformin & L-Citrulline |
| L-Citrulline and Metformin & L-Citrulline | Drug | L-Citrulline and Metformin & L-Citrulline |
Primary outcome
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
Time frame: week 6 and week 12
4 secondary outcomes
MFM total score and six minute walking distance (6MWD)
Time frame: week 6 and week 12
Change of muscle fat content (MFC) (assessed by MRI)
Time frame: week 6 and week 12
Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)
Time frame: week 6 and week 12
Change of laboratory parameters (oxidative and nitrosative stress)
Time frame: week 6 and week 12
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 16 May 2016.
Related trials for Becker's Muscular Dystrophy (BMD)
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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 Children's Hospital Basel
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Metformin and Metformin & L-Citrulline and L-Citrulline and Metformin & L-Citrulline in people with Becker's Muscular Dystrophy (BMD), run by University Hospital, Basel, Switzerland at 1 site in 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 16 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 motor function measure (MFM) D1 subscore after 6 and 12 weeks (time frame: week 6 and week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 May 2016.