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Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

Primary Purpose

Duchenne's Muscular Dystrophy

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Umbilical Cord Mesenchymal Stem Cells

About this trial

This is an interventional treatment trial for Duchenne's Muscular Dystrophy focused on measuring Mesenchymal, Stem Cells, Umbilical Cord, Duchenne's, Muscular Dystrophy

Eligibility Criteria

28 Years - 31 Years (Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

Duchenne's Muscular Dystrophy

Exclusion Criteria:

None

Sites / Locations

Asthma and Allergy Consultants

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Mesenchymal Stem Cells

Arm Description

Umbilical Cord Mesenchymal Stem Cells

Outcomes

Primary Outcome Measures

Adverse Events

No occurrence of adverse events

Secondary Outcome Measures

Change from baseline of weight

Change of muscle diameter (circumferential measurements) from baseline

Change from baseline of Pulmonary Maximum Expiratory Pressure

Change from baseline of Pulmonary Forced Vital Capacity

Maximum Change from baseline of Predicted Inspiratory Pressure %

Change from baseline of Predicted Maximum Expiratory Pressure %

Change from baseline of Predicted Forced Vital Capacity %

Full Information

NCT ID

NCT02235844

First Posted

September 8, 2014

Last Updated

September 12, 2019

Sponsor

Allergy and Asthma Consultants, Wichita, Kansas

Collaborators

Aidan Foundation, Neil H. Riordan PhD

1. Study Identification

Unique Protocol Identification Number

NCT02235844

Brief Title

Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

Official Title

Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

Study Type

Interventional

2. Study Status

Record Verification Date

September 2019

Overall Recruitment Status

Completed

Study Start Date

September 2014 (Actual)

Primary Completion Date

September 30, 2017 (Actual)

Study Completion Date

September 30, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Allergy and Asthma Consultants, Wichita, Kansas

Collaborators

Aidan Foundation, Neil H. Riordan PhD

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Data Monitoring Committee

5. Study Description

Brief Summary

This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Both of these properties could lead to potential benefits for a DMD patient.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Duchenne's Muscular Dystrophy

Keywords

Mesenchymal, Stem Cells, Umbilical Cord, Duchenne's, Muscular Dystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

1 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Mesenchymal Stem Cells

Arm Type

Experimental

Arm Description

Umbilical Cord Mesenchymal Stem Cells

Intervention Type

Biological

Intervention Name(s)

Umbilical Cord Mesenchymal Stem Cells

Primary Outcome Measure Information:

Title

Adverse Events

Description

No occurrence of adverse events

Time Frame