L-citrulline and Metformin in Duchenne's Muscular Dystrophy

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2013 by University Hospital, Basel, Switzerland
Sponsor:
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier:
NCT01995032
First received: November 20, 2013
Last updated: November 26, 2013
Last verified: November 2013
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Mean change of MFM total score, the D2, and D3 MFM subscores [ Time Frame: baseline to week 26 ] [ Designated as safety issue: No ]
  • Mean change of six minute walking distance (6MWD) [ Time Frame: baseline to week 26 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) [ Time Frame: baseline to week 26 ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: October 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: metformin and L-citrulline
7.5 g L-citrulline p.o. and 750 mg metformin p.o. per day (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.

  Eligibility

Ages Eligible for Study:   7 Years to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT01995032

Contacts
Contact: Ulrike Bonati, MD +41617042243 ulrike.bonati@ukbb.ch
Contact: Daniela Rubino daniela.rubino@ukbb.ch

Locations
Switzerland
University Children's Hospital Recruiting
Basel, BS, Switzerland, 4031
Contact: Ulrike Bonati, MD       ulrike.bonati@ukbb.ch   
Principal Investigator: Dirk Fischer, MD         
Sub-Investigator: Ulrike Bonati, MD         
Sponsors and Collaborators
University Hospital, Basel, Switzerland
Investigators
Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
  More Information

No publications provided

Responsible Party: University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier: NCT01995032     History of Changes
Other Study ID Numbers: DMD02
Study First Received: November 20, 2013
Last Updated: November 26, 2013
Health Authority: Switzerland: Swissmedic

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

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metformin
Hypoglycemic Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 19, 2014