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

NCT ID: NCT02484560

Last Updated: 2015-07-01

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-06-30
• Study Completion Date: 2015-12-31

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.

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ambulatory Patients

Ambulatory patients receiving stem cell therapy

Group Type: EXPERIMENTAL

Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Intervention Type: DRUG

Non-Ambulatory Patients

non-ambulatory patients receiving stem cell therapy

Group Type: EXPERIMENTAL

Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Intervention Type: DRUG

Interventions

Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 8 Years
• Maximum Eligible Age: 14 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Istinye University

OTHER

Sponsor Role: collaborator

University of Gaziantep

OTHER

Sponsor Role: lead

Responsible Party

Alper Dai, MD

Lead Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Gaziantep University Hospital

Gaziantep, , Turkey (Türkiye)

Countries

Turkey (Türkiye)

Other Identifiers

56733164/203

Identifier Type: -

Identifier Source: org_study_id