L-citrulline and Metformin in Duchenne's Muscular Dystrophy

Duchenne's Muscular Dystrophy (DMD) Clinical Trial

Official 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"

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01995032
• Other study ID #: DMD02
• Status: Completed
• Phase: Phase 3
• First received: November 20, 2013
• Last updated: April 17, 2018
• Start date: October 2013
• Est. completion date: March 2016

Study information

• Verified date: April 2018
• Source: University Hospital, Basel, Switzerland
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 47
• Est. completion date: March 2016
• Est. primary completion date: October 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 78 Months to 10 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne's Muscular Dystrophy (DMD)
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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

• Placebo

Locations

Country	Name	City	State
Switzerland	University Children's Hospital	Basel	BS

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Basel, Switzerland

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)		baseline to week 26
Secondary	Mean change of MFM total score, the D2, and D3 MFM subscores		baseline to week 26
Secondary	Mean change of six minute walking distance (6MWD)		baseline to week 26
Secondary	Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles		baseline to week 26
Secondary	Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA)		baseline to week 26
Secondary	Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD)		baseline to week 26