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

University of Gaziantep

Status and phase: Unknown; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Study type: Interventional

Funder types: Other

Identifiers

NCT02484560

56733164/203

Details and patient eligibility

About

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.

Full 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.

Enrollment: 10 patients

Sex: Male

Ages: 8 to 14 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 2 patient groups

Ambulatory Patients

Experimental group

Description:

Ambulatory patients receiving stem cell therapy

Treatment:

Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell

Non-Ambulatory Patients

Experimental group

Description:

non-ambulatory patients receiving stem cell therapy

Treatment:

Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell