L-citrulline and Metformin in Duchenne's Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT01995032 |
Recruitment Status :
Completed
First Posted : November 26, 2013
Last Update Posted : April 19, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne's Muscular Dystrophy (DMD) | Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o. Drug: Placebo | Phase 3 |
This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits.
Amendment 1: Amended eligibility criteria
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 47 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | "A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscular Dystrophy" |
Study Start Date : | October 2013 |
Actual Primary Completion Date : | October 2015 |
Actual Study Completion Date : | March 2016 |

Arm | Intervention/treatment |
---|---|
Experimental: 750 mg metformin and 7.5 g L-citrulline daily p.o.
7.5 g L-citrulline p.o. and 750 mg metformin daily p.o. (3x 2.5 g, respectively 3x 250 mg) for 26 weeks
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Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o. |
Placebo Comparator: Placebo
metformin placebo and L-citrulline placebo 3 times daily p.o. for 26 weeks
|
Drug: Placebo |
- Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [ Time Frame: baseline to week 26 ]
- Mean change of MFM total score, the D2, and D3 MFM subscores [ Time Frame: baseline to week 26 ]
- Mean change of six minute walking distance (6MWD) [ Time Frame: baseline to week 26 ]
- Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles [ Time Frame: baseline to week 26 ]
- Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA) [ Time Frame: baseline to week 26 ]
- Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) [ Time Frame: baseline to week 26 ]

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Ages Eligible for Study: | 78 Months to 10 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Molecular diagnosis of DMD
- Patients 6.5 - 10 years of age at time of screening
- Ambulant
- Ability to walk 150 m in the 6 min walking distance (6MWT)
- D1 subdomain of the MFM scale >40%
- stable treatment with steroids for >6 months or steroid naïve patients
Exclusion Criteria:
- Previous (3 months or less) or concomitant participation in another therapeutic trial
- Use of L-citrulline, L-arginine or metformin within the last 3 months
- Known individual hypersensitivity to L-citrulline or metformin
- known or suspected malignancy
- Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
- start of cortisone treatment or change in dosage <6 months prior to screening

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01995032
Switzerland | |
University Children's Hospital | |
Basel, BS, Switzerland, 4031 |
Principal Investigator: | Dirk Fischer, MD | University Children's Hospital Basel |
Responsible Party: | University Hospital, Basel, Switzerland |
ClinicalTrials.gov Identifier: | NCT01995032 |
Other Study ID Numbers: |
DMD02 |
First Posted: | November 26, 2013 Key Record Dates |
Last Update Posted: | April 19, 2018 |
Last Verified: | April 2018 |
Duchenne's muscular dystrophy DMD |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases |
Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Metformin Hypoglycemic Agents Physiological Effects of Drugs |