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L-citrulline and Metformin in Duchenne's Muscular Dystrophy

This study has been completed.
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland Identifier:
First received: November 20, 2013
Last updated: August 12, 2016
Last verified: August 2016
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 Intervention Phase
Duchenne's Muscular Dystrophy (DMD) Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o. Drug: Placebo Phase 3

Study Type: Interventional
Study Design: 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"

Resource links provided by NLM:

Further study details as provided by University Hospital, Basel, Switzerland:

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

Secondary Outcome Measures:
  • 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 ]

Enrollment: 47
Study Start Date: October 2013
Study Completion Date: March 2016
Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

Ages Eligible for Study:   7 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Molecular diagnosis of DMD
  • Patients 7 - 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01995032

University Children's Hospital
Basel, BS, Switzerland, 4031
Sponsors and Collaborators
University Hospital, Basel, Switzerland
Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: University Hospital, Basel, Switzerland Identifier: NCT01995032     History of Changes
Other Study ID Numbers: DMD02
Study First Received: November 20, 2013
Last Updated: August 12, 2016

Keywords provided by University Hospital, Basel, Switzerland:
Duchenne's muscular dystrophy

Additional relevant MeSH terms:
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 processed this record on August 17, 2017