NCT02018731

Brief Summary

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2013

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

December 17, 2013

Completed
6 days until next milestone

First Posted

Study publicly available on registry

December 23, 2013

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2014

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
Last Updated

May 16, 2016

Status Verified

May 1, 2016

Enrollment Period

1.3 years

First QC Date

December 17, 2013

Last Update Submit

May 13, 2016

Conditions

Becker's Muscular Dystrophy (BMD)

Keywords

Becker's Muscular dystrophyBMDMetforminL-citrulline

Outcome Measures

Primary Outcomes (1)

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

    week 6 and week 12

Secondary Outcomes (4)

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

    week 6 and week 12

  • Change of muscle fat content (MFC) (assessed by MRI)

    week 6 and week 12

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

    week 6 and week 12

  • Change of laboratory parameters (oxidative and nitrosative stress)

    week 6 and week 12

Study Arms (2)

Metformin and Metformin & L-Citrulline

EXPERIMENTAL

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

L-Citrulline and Metformin & L-Citrulline

EXPERIMENTAL

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

Interventions

Metformin and Metformin & L-CitrullineDRUG
Metformin and Metformin & L-Citrulline
L-Citrulline and Metformin & L-CitrullineDRUG
L-Citrulline and Metformin & L-Citrulline

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Children's Hospital

Basel, Canton of Basel-City, 4031, Switzerland

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

MetforminCitrulline

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

BiguanidesGuanidinesAmidinesOrganic ChemicalsAmino Acids, DiaminoAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Dirk Fischer, MD

    University Children's Hospital Basel

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 17, 2013

First Posted

December 23, 2013

Study Start

June 1, 2013

Primary Completion

September 1, 2014

Study Completion

December 1, 2015

Last Updated

May 16, 2016

Record last verified: 2016-05

Locations

CH(1)