RECRUITING

Trial of Cell Based Therapy for DMD

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is a single-center, single-arm, interventional phase 1 trial to evaluate the safety and tolerability of local injection of induced pluripotent stem cell (iPSC)- derived CD54+ allogeneic muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD)

Official Title

Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy

Quick Facts

Study Start:2025-03-20

Study Completion:2027-03-03

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06692426

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy * Non-ambulatory * Intact extensor digitorum brevis (EDB) muscles bilaterally * Off investigational therapies for \> 30 days * Age 18 years of age or older at the time of consent * Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function): * Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy. * Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period * Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified * Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.	* Presence of HLA antibodies directed toward HLA antigens on MyoPAXon * Active treatment with another investigational therapy * Known allergy to MyoPAXon components

Inclusion Criteria

Exclusion Criteria

* Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy
* Non-ambulatory
* Intact extensor digitorum brevis (EDB) muscles bilaterally
* Off investigational therapies for \> 30 days
* Age 18 years of age or older at the time of consent
* Have adequate organ function confirmed by the following laboratory values obtained within 14 days prior to enrollment (28 days for cardiac and pulmonary function):
* Participants with partners of childbearing potential must be willing to use at least two forms of effective birth control (one form must be a barrier method) while receiving the study product and for 3 months after stopping tacrolimus therapy.
* Ability to follow commands sufficiently to perform voluntary aspects of outcome measures throughout the study period
* Willing to consent to monitoring for 15 years, including an extension period, as required for all interventional studies involving the transplantation of cells that have been genetically modified
* Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.

* Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
* Active treatment with another investigational therapy
* Known allergy to MyoPAXon components

Contacts and Locations

Study Contact

Peter Kang, MD

CONTACT

612-624-9452

mdstemcell@umn.edu

Study Locations (Sites)

Masonic Cancer Center

Minneapolis, Minnesota, 55455

United States

Collaborators and Investigators

Sponsor: Masonic Cancer Center, University of Minnesota

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-03-20

Study Completion Date2027-03-03

Study Record Updates

Study Start Date2025-03-20

Study Completion Date2027-03-03

Terms related to this study

Additional Relevant MeSH Terms

Duchenne Muscular Dystrophy