Trial of Cell Based Therapy for DMD

NCT ID: NCT06692426

Last Updated: 2025-03-24

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-20
• Study Completion Date: 2027-03-03

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)

Detailed Description

The University of Minnesota holds equity in, and has rights to potential royalties from, Myogenica, the company that has licensed this trial's stem cell therapy from the University. These interests have been reviewed and managed by the University of Minnesota in accordance with its conflict of interest policies. If you would like further information please contact Jon Guden, Associate Director, Conflict of Interest Program, at jguden@umn.edu.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm A: MyoPAXon 25 x 10^6

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

Group Type: EXPERIMENTAL

MyoPAXon

Intervention Type: DRUG

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Tacrolimus

Intervention Type: DRUG

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Arm B: MyoPAXon 50 x 10^6

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

Group Type: EXPERIMENTAL

MyoPAXon

Intervention Type: DRUG

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Tacrolimus

Intervention Type: DRUG

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Arm C: MyoPAXon 100 x 10^6

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

Group Type: EXPERIMENTAL

MyoPAXon

Intervention Type: DRUG

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Tacrolimus

Intervention Type: DRUG

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Arm D: MyoPAXon 200 x 10^6

MyoPAXon will be delivered via open intramuscular (IM) injection into a single EDB muscle for each participant as a one-time dose. The participants will be started on tacrolimus 1 week prior to dosing and stay on the maintenance dose on a BID schedule for 3 months after injection

Group Type: EXPERIMENTAL

MyoPAXon

Intervention Type: DRUG

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Tacrolimus

Intervention Type: DRUG

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Interventions

MyoPAXon

MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2

Intervention Type: DRUG

Tacrolimus

Tacrolimus (Prograf) is an immunosuppressant that inhibits calcineurin and T cell activation, and is commonly used to prevent solid organ transplant rejection1 and graft versus host disease (GVHD) as well as allograft rejection in the setting of allogeneic hematopoietic stem cell transplantation.

Intervention Type: DRUG

Other Intervention Names

Prograf

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Duchenne UK

UNKNOWN

Sponsor Role: collaborator

Parent Project Muscular Dystrophy

OTHER

Sponsor Role: collaborator

Masonic Cancer Center, University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Masonic Cancer Center

Minneapolis, Minnesota, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Peter Kang, MD

Role: CONTACT

mdstemcell@umn.edu

612-624-9452

Facility Contacts

Peter Kang, MD

Role: primary

Other Identifiers

2021LS140

Identifier Type: -

Identifier Source: org_study_id