Skip to main content
OpenTrials
Completed

NCT Number: NCT02018731

L-citrulline and Metformin in 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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Becker's Muscular Dystrophy (BMD) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University Children's Hospital

Basel, Canton of Basel-City, 4031, Switzerland

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Metformin and Metformin & L-Citrulline

Drug

L-Citrulline and Metformin & L-Citrulline

Drug

Primary outcomes

  1. Mean change of motor function measure (MFM) D1 subscore after 6 and 12 weeks

    Time frame: week 6 and week 12

Secondary outcomes

  1. MFM total score and six minute walking distance (6MWD)

    Time frame: week 6 and week 12

  2. Change of muscle fat content (MFC) (assessed by MRI)

    Time frame: week 6 and week 12

  3. Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry)

    Time frame: week 6 and week 12

  4. Change of laboratory parameters (oxidative and nitrosative stress)

    Time frame: week 6 and week 12

Sponsors and collaborators

Lead sponsor

University Hospital, Basel, Switzerland

Other

Registry information

Official study title

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

Important dates

Study start
2013
Primary completion
2014
Study completion
2015
First posted
Dec 23, 2013
Registry last updated
May 16, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.