L-citrulline and Metformin in Duchenne's Muscular Dystrophy

NCT ID: NCT01995032

Last Updated: 2018-04-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 47 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-10-31
• Study Completion Date: 2016-03-31

Brief Summary

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.

Detailed Description

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

Conditions

Duchenne's Muscular Dystrophy (DMD)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

7.5 g L-citrulline p.o. and 750 mg metformin daily p.o. (3x 2.5 g, respectively 3x 250 mg) for 26 weeks

Group Type: EXPERIMENTAL

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

Intervention Type: DRUG

Placebo

metformin placebo and L-citrulline placebo 3 times daily p.o. for 26 weeks

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Interventions

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

Intervention Type: DRUG

Placebo

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 78 Months
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Basel, Switzerland

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dirk Fischer, MD

Role: PRINCIPAL_INVESTIGATOR

University Children's Hospital Basel

Locations

University Children's Hospital

Basel, Canton of Basel-City, Switzerland

Countries

Switzerland

References

Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D. Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Netw Open. 2019 Oct 2;2(10):e1914171. doi: 10.1001/jamanetworkopen.2019.14171.

Reference Type: DERIVED

PMID: 31664444 (View on PubMed)

Hafner P, Bonati U, Rubino D, Gocheva V, Zumbrunn T, Gueven N, Fischer D. Treatment with L-citrulline and metformin in Duchenne muscular dystrophy: study protocol for a single-centre, randomised, placebo-controlled trial. Trials. 2016 Aug 3;17(1):389. doi: 10.1186/s13063-016-1503-1.

Reference Type: DERIVED

PMID: 27488051 (View on PubMed)

Other Identifiers

DMD02

Identifier Type: -

Identifier Source: org_study_id