Trial of Cell Based Therapy for DMD

NCT06692426 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 2021LS140
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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)

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• Maximal tolerable dose (MTD) of MyoPAXon

  Maximal tolerable dose (MTD) of MyoPAXon

  3 months

Secondary Outcomes (1)

• Proportion of patients developing humoral and cellular responses

  3 months

Study Arms (4)

Arm A: MyoPAXon 25 x 10^6

EXPERIMENTAL

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

Drug: MyoPAXon; Drug: Tacrolimus

Arm B: MyoPAXon 50 x 10^6

EXPERIMENTAL

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

Drug: MyoPAXon; Drug: Tacrolimus

Arm C: MyoPAXon 100 x 10^6

EXPERIMENTAL

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

Drug: MyoPAXon; Drug: Tacrolimus

Arm D: MyoPAXon 200 x 10^6

EXPERIMENTAL

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

Drug: MyoPAXon; Drug: Tacrolimus

Interventions

MyoPAXon DRUG

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

Arm A: MyoPAXon 25 x 10^6; Arm B: MyoPAXon 50 x 10^6; Arm C: MyoPAXon 100 x 10^6; Arm D: MyoPAXon 200 x 10^6

Tacrolimus 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.

Also known as: Prograf

Arm A: MyoPAXon 25 x 10^6; Arm B: MyoPAXon 50 x 10^6; Arm C: MyoPAXon 100 x 10^6; Arm D: MyoPAXon 200 x 10^6

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Parent Project Muscular Dystrophy: collaborator
• Masonic Cancer Center, University of Minnesota: lead
• Duchenne UK: collaborator

Study Sites (1)

Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Tacrolimus

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Central Study Contacts

Peter Kang, MD

CONTACT

612-624-9452; mdstemcell@umn.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2024
• First Posted: November 18, 2024
• Study Start: March 20, 2025
• Primary Completion (Estimated): March 3, 2027
• Study Completion (Estimated): March 3, 2027
• Last Updated: March 4, 2026

Record last verified: 2026-03

Locations

US (1)