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

• ClinicalTrials.gov Identifier: NCT02484560
• Recruitment Status: Unknown
• First Posted: June 29, 2015
• Last Update Posted: July 1, 2015
• Sponsor: University of Gaziantep
• Collaborator: Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital
• Information provided by (Responsible Party): Alper Dai, MD, University of Gaziantep

Study Description

Brief 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.

Condition or disease	Intervention/treatment	Phase
Duchenne Muscular Dystrophy	Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell	Phase 1

Detailed 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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 10 participants
• Allocation: Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
• Study Start Date: June 2015
• Estimated Primary Completion Date: December 2015
• Estimated Study Completion Date: December 2015

Arms and Interventions

Arm	Intervention/treatment
Experimental: Ambulatory Patients; Ambulatory patients receiving stem cell therapy	Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell
Experimental: Non-Ambulatory Patients; non-ambulatory patients receiving stem cell therapy	Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Outcome Measures

Primary Outcome Measures :

1. 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 ]

   Tests Used in Assement:

   Northstar ambulatory assessment CHAQ (Child Health Assessment Questionnaire) MRI/MRS Muscle Strength Assessment - Myogrip, Myopinch, and Moviplate

Eligibility Criteria

• Ages Eligible for Study: 8 Years to 14 Years (Child)
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

Turkey

Gaziantep University Hospital

Gaziantep, Turkey, 27310

Sponsors and Collaborators

University of Gaziantep

Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital

More Information

• Responsible Party: Alper Dai, MD, Lead Investigator, University of Gaziantep
• ClinicalTrials.gov Identifier: NCT02484560
• Other Study ID Numbers: 56733164/203
• First Posted: June 29, 2015
• Last Update Posted: July 1, 2015
• Last Verified: June 2015

Keywords provided by Alper Dai, MD, University of Gaziantep:

Duchenne Muscular Dystrophy; Neuromuscular Diseases; Muscular Dystrophy; Genetic Diseases, X-linked; Musculoskeletal Diseases; Nervous System Diseases

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked