Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.

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.
 
ClinicalTrials.gov Identifier: NCT01995032
Recruitment Status : Completed
First Posted : November 26, 2013
Last Update Posted : April 19, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland

Tracking Information
First Submitted Date  ICMJE November 20, 2013
First Posted Date  ICMJE November 26, 2013
Last Update Posted Date April 19, 2018
Study Start Date  ICMJE October 2013
Actual Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 20, 2013)
Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [ Time Frame: baseline to week 26 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01995032 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 20, 2013)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE L-citrulline and Metformin in Duchenne's Muscular Dystrophy
Official Title  ICMJE "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"
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.
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

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Duchenne's Muscular Dystrophy (DMD)
Intervention  ICMJE
  • Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.
  • Drug: Placebo
Study Arms  ICMJE
  • 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
    Intervention: 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
    Intervention: Drug: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 12, 2016)
47
Original Estimated Enrollment  ICMJE
 (submitted: November 20, 2013)
50
Actual Study Completion Date  ICMJE March 2016
Actual Primary Completion Date October 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 78 Months to 10 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Switzerland
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01995032
Other Study ID Numbers  ICMJE DMD02
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party University Hospital, Basel, Switzerland
Study Sponsor  ICMJE University Hospital, Basel, Switzerland
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Dirk Fischer, MD University Children's Hospital Basel
PRS Account University Hospital, Basel, Switzerland
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP