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L-citrulline and Metformin in Duchenne's Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01995032
Recruitment Status : Completed
First Posted : November 26, 2013
Last Update Posted : April 19, 2018
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland

Brief Summary:
The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.

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

Detailed Description:

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

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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
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

Primary Outcome Measures :
  1. Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [ Time Frame: baseline to week 26 ]

Secondary Outcome Measures :
  1. Mean change of MFM total score, the D2, and D3 MFM subscores [ Time Frame: baseline to week 26 ]
  2. Mean change of six minute walking distance (6MWD) [ Time Frame: baseline to week 26 ]
  3. 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 ]
  4. 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 ]
  5. Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) [ Time Frame: baseline to week 26 ]

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

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 identifier (NCT number): NCT01995032

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University Children's Hospital
Basel, BS, Switzerland, 4031
Sponsors and Collaborators
University Hospital, Basel, Switzerland
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Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: University Hospital, Basel, Switzerland 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
Keywords provided by University Hospital, Basel, Switzerland:
Duchenne's muscular dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hypoglycemic Agents
Physiological Effects of Drugs