Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2

• Register: ClinicalTrials.gov
• Last refreshed on: 3 August 2015
• Main ID: NCT02484560
• Date of registration: 16/06/2015
• Prospective Registration: No
• Primary sponsor: University of Gaziantep
• Public title: Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
• Scientific title: Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
• Date of first enrolment: June 2015
• Target sample size: 10
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02484560
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Turkey

Key inclusion & exclusion criteria

Inclusion Criteria:

- Ambulatory and Non-ambulatory patients diagnosed with DMD that is proven both
clinically and genetically and are between 5-20 years old who need partial
respiratory support daily. Patients with less than or equal to stage 1 NIH, cardiac,
liver, and renal function. Patients must also not present any indication of cancer,
allergic disease, nor bleeding diathesis.

Exclusion Criteria:

- Patients who require full respiratory support. Patients have stage II NIH or greater,
cardiac, liver, and renal function. Patients present with signs of symptoms of
cancer, allergic disease, or bleeding diathesis.



Age minimum: 8 Years
Age maximum: 14 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Primary Outcome(s)

Degree of improvement in patients with Duchenne Muscular Dystrophy after stem cell therapy treatment administered using Northstar Ambulatory Assessment, Magnetic Resonance Imaging & Spectroscopy, muscle strength assessment equipment, and a questionnaire. [Time Frame: 12 Months]

Secondary ID(s)

56733164/203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Istinye University, Cukurova University, Yildirim Beyazit University, Gaziantep Deva Hospital, Gaziantep Public Hospital