L-citrulline and Metformin in Becker's Muscular Dystrophy

May 13, 2016 updated by: University Hospital, Basel, Switzerland

Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy

The purpose of the study is to compare the effects of L-citrulline and metformin and their combination therapy on muscle function and force in patients with Becker muscular dystrophy (BMD).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a single center, not-randomized open study. The study medication consists of L-citrulline and metformin administered orally; 5g citrulline and 500mg metformin tablets will be given 3 times daily.

Half of the patients will be treated with metformin during the first 6 weeks of the study. The other patients will receive initially L-citrulline for 6 weeks, before all patients will be treated with the combination therapy (metformin and L-citrulline) for another 6 week period.

The study lasts 12 weeks and includes the screening visit and 3 further visits at baseline, week 6 and week 12.

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 2

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

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 18 years or older
  • Molecular or immunohistochemical diagnosis of BMD
  • ambulant at the time point of screening

Exclusion Criteria:

  • Participation in another therapeutic BMD study within the last 3 months
  • Use of L-Arginine, L-Citrulline or Metformin within the last 3 months
  • Other chronic disease or relevant limitation of renal, liver, heart function according to discretion of investigator
  • known hypersensitivity to L-citrulline or metformin

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: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Metformin and Metformin & L-Citrulline
1500 mg/d metformin for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
Drug: Metformin and Metformin & L-Citrulline
Experimental: L-Citrulline and Metformin & L-Citrulline
15 g/d L-citrulline for 6 weeks, followed by 1500 mg/d metformin and 15 g/d L-citrulline for 6 further weeks
Drug: L-Citrulline and Metformin & L-Citrulline

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks
Time Frame: week 6 and week 12
week 6 and week 12

Secondary Outcome Measures

Outcome Measure
Time Frame
MFM total score and six minute walking distance (6MWD)
Time Frame: week 6 and week 12
week 6 and week 12
Change of muscle fat content (MFC) (assessed by MRI)
Time Frame: week 6 and week 12
week 6 and week 12
Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)
Time Frame: week 6 and week 12
week 6 and week 12
Change of laboratory parameters (oxidative and nitrosative stress)
Time Frame: week 6 and week 12
week 6 and week 12

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

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

June 1, 2013

Primary Completion (Actual)

September 1, 2014

Study Completion (Actual)

December 1, 2015

Study Registration Dates

First Submitted

December 17, 2013

First Submitted That Met QC Criteria

December 17, 2013

First Posted (Estimate)

December 23, 2013

Study Record Updates

Last Update Posted (Estimate)

May 16, 2016

Last Update Submitted That Met QC Criteria

May 13, 2016

Last Verified

May 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BMD01

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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