Overview

This trial is active, not recruiting.

Condition duchenne muscular dystrophy
Treatment biological: umbilical cord based allogenic mesenchymal stem cell
Phase phase 1
Sponsor University of Gaziantep
Collaborator Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital
Start date June 2015
End date December 2015
Trial size 10 participants
Trial identifier NCT02484560, 56733164/203

Summary

Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Ambulatory patients receiving stem cell therapy
biological: umbilical cord based allogenic mesenchymal stem cell
(Experimental)
non-ambulatory patients receiving stem cell therapy
biological: umbilical cord based allogenic mesenchymal stem cell

Primary Outcomes

Measure
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

Eligibility Criteria

Male participants from 8 years up to 14 years old.

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.

Additional Information

Official title Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
Description Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently. The purpose of this study is to investigate the effects of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children with Duchenne Muscular Dystrophy and determine its suitability as a form of treatment.
Trial information was received from ClinicalTrials.gov and was last updated in June 2015.
Information provided to ClinicalTrials.gov by University of Gaziantep.