L-citrulline and Metformin in Becker's Muscular Dystrophy

This study is currently recruiting participants. (see Contacts and Locations)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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, Inborn
Genetic Diseases, X-Linked
Metformin
Hypoglycemic Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014