Trial of Cell Based Therapy for DMD

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)

Conditions

Duchenne Muscular Dystrophy

Study Overview

Study Details

Study overview

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)

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

Trial of Cell Based Therapy for DMD

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Minneapolis

Masonic Cancer Center, Minneapolis, Minnesota, United States, 55455

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Masonic Cancer Center, University of Minnesota,

Study Record Dates

2027-03-03