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NCT06692426 | NOT YET RECRUITING | Duchenne Muscular Dystrophy

Ph I Trial of Cell Based Therapy for DMD
Sponsor:

Masonic Cancer Center, University of Minnesota

Brief Summary:

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)

Condition or disease

Duchenne Muscular Dystrophy

Intervention/treatment

MyoPAXon

Tacrolimus

Phase

PHASE1

Study Type : INTERVENTIONAL
Estimated Enrollment : 8 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
Actual Study Start Date : 2024-12-16
Estimated Primary Completion Date : 2027-03-03
Estimated Study Completion Date : 2027-03-03

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion 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.
Exclusion Criteria
  • * Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
  • * Active treatment with another investigational therapy
  • * Known allergy to MyoPAXon components
Ph I Trial of Cell Based Therapy for DMD

Location Details

NCT06692426

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How to Participate

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Locations

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