L-citrulline and Metformin in Duchenne's Muscular Dystrophy

April 17, 2018 updated by: University Hospital, Basel, Switzerland

"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"

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Actual)

47

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Switzerland
    • BS
      • Basel, BS, Switzerland, 4031
        • University Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 10 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)
Time Frame: baseline to week 26
baseline to week 26

Secondary Outcome Measures

Outcome Measure
Time Frame
Mean change of MFM total score, the D2, and D3 MFM subscores
Time Frame: baseline to week 26
baseline to week 26
Mean change of six minute walking distance (6MWD)
Time Frame: baseline to week 26
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
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
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
baseline to week 26

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Basel, Switzerland

Investigators

  • Principal Investigator: Dirk Fischer, MD, University Children's Hospital Basel

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2013

Primary Completion (Actual)

October 1, 2015

Study Completion (Actual)

March 1, 2016

Study Registration Dates

First Submitted

November 20, 2013

First Submitted That Met QC Criteria

November 20, 2013

First Posted (Estimate)

November 26, 2013

Study Record Updates

Last Update Posted (Actual)

April 19, 2018

Last Update Submitted That Met QC Criteria

April 17, 2018

Last Verified

April 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DMD02

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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