L-citrulline and Metformin in Becker's Muscular Dystrophy

This study is currently recruiting participants.
Verified December 2013 by University Hospital, Basel, Switzerland
Sponsor:
Information provided by (Responsible Party):
University Hospital, Basel, Switzerland
ClinicalTrials.gov Identifier:
NCT02018731
First received: December 17, 2013
Last updated: NA
Last verified: December 2013
History: No changes posted
  Purpose

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).


Condition Intervention Phase
Becker's Muscular Dystrophy (BMD)
Drug: Metformin and Metformin & L-Citrulline
Drug: L-Citrulline and Metformin & L-Citrulline
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker'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 after 6 and 12 weeks [ Time Frame: week 6 and week 12 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • MFM total score and six minute walking distance (6MWD) [ Time Frame: week 6 and week 12 ] [ Designated as safety issue: No ]
  • Change of muscle fat content (MFC) (assessed by MRI) [ Time Frame: week 6 and week 12 ] [ Designated as safety issue: No ]
  • Change of muscle metabolism (assessed by dual energy x-ray absorptiometry (DEXA) and indirect calorimetry) [ Time Frame: week 6 and week 12 ] [ Designated as safety issue: No ]
  • Change of laboratory parameters (oxidative and nitrosative stress) [ Time Frame: week 6 and week 12 ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: June 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT02018731

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    +41617042243    ulrike.bonati@ukbb.ch   
Contact: Daniela Rubino       daniela.rubino@ukbb.ch   
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: NCT02018731     History of Changes
Other Study ID Numbers: BMD01
Study First Received: December 17, 2013
Last Updated: December 17, 2013
Health Authority: Switzerland: Swissmedic

Keywords provided by University Hospital, Basel, Switzerland:
Becker's Muscular dystrophy
BMD
Metformin
L-citrulline

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 April 17, 2014