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

NCT02484560 | Unknown Phase 1 DMC

• 1 other identifier: 56733164/203
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

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

Outcome Measures

Primary Outcomes (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.

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

  12 Months

Study Arms (2)

Ambulatory Patients

EXPERIMENTAL

Ambulatory patients receiving stem cell therapy

Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Non-Ambulatory Patients

EXPERIMENTAL

non-ambulatory patients receiving stem cell therapy

Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Interventions

Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell DRUG

Ambulatory Patients; Non-Ambulatory Patients

Eligibility Criteria

• Age: 8 Years - 14 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of Gaziantep: lead
• Istinye University: collaborator

Study Sites (1)

Gaziantep University Hospital

Gaziantep, 27310, Turkey (Türkiye)

Location

MeSH Terms

Conditions

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

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Lead Investigator

Study Record Dates

• First Submitted: June 16, 2015
• First Posted: June 29, 2015
• Study Start: June 1, 2015
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: July 1, 2015

Record last verified: 2015-06

Locations

TU (1)