L-citrulline and Metformin in Duchenne's Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01995032
• Date of registration: 20/11/2013
• Prospective Registration: No
• Primary sponsor: University Hospital, Basel, Switzerland
• Public title: L-citrulline and Metformin in Duchenne's Muscular Dystrophy
• Scientific 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"
• Date of first enrolment: October 2013
• Target sample size: 47
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01995032
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Switzerland

Contacts

• Name: Dirk Fischer, MD
• Affiliation: University Children's Hospital Basel

Key inclusion & exclusion 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



Age minimum: 78 Months
Age maximum: 10 Years
Gender: All

Health Condition(s) or Problem(s) studied

Duchenne's Muscular Dystrophy (DMD)

Intervention(s)

Drug: Placebo

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

Primary Outcome(s)

Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers) [Time Frame: baseline to week 26]

Secondary Outcome(s)

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]

Mean change of six minute walking distance (6MWD) [Time Frame: baseline to week 26]

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]

Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD) [Time Frame: baseline to week 26]

Mean change of MFM total score, the D2, and D3 MFM subscores [Time Frame: baseline to week 26]

Secondary ID(s)

DMD02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors