Skip to main content
OpenTrials
Completed

NCT Number: NCT01995032

L-citrulline and Metformin in Duchenne's Muscular Dystrophy

The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Duchenne's Muscular Dystrophy (DMD) 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

78 month–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University Children's Hospital

Basel, Canton of Basel-City, 4031, Switzerland

About this study

This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits.

Amendment 1: Amended eligibility criteria

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Molecular diagnosis of DMD
  • Patients 6.5 - 10 years of age at time of screening
  • Ambulant
  • Ability to walk 150 m in the 6 min walking distance (6MWT)
  • D1 subdomain of the MFM scale >40%
  • stable treatment with steroids for >6 months or steroid naïve patients

Exclusion criteria

  • Previous (3 months or less) or concomitant participation in another therapeutic trial
  • Use of L-citrulline, L-arginine or metformin within the last 3 months
  • Known individual hypersensitivity to L-citrulline or metformin
  • known or suspected malignancy
  • Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator
  • start of cortisone treatment or change in dosage <6 months prior to screening

Treatment and study plan

750 mg metformin and 7.5 g L-citrulline daily p.o.

Drug

Placebo

Drug

Primary outcomes

  1. Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)

    Time frame: baseline to week 26

Secondary outcomes

  1. Mean change of MFM total score, the D2, and D3 MFM subscores

    Time frame: baseline to week 26

  2. Mean change of six minute walking distance (6MWD)

    Time frame: baseline to week 26

  3. Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles

    Time frame: baseline to week 26

  4. Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA)

    Time frame: baseline to week 26

  5. Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD)

    Time frame: baseline to week 26

Sponsors and collaborators

Lead sponsor

University Hospital, Basel, Switzerland

Other

Registry information

Official study title

"A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscular Dystrophy"

Important dates

Study start
2013
Primary completion
2015
Study completion
2016
First posted
Nov 26, 2013
Registry last updated
Apr 19, 2018

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.