Ph I Trial of Cell Based Therapy for DMD

Phase 1

Not yet recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: MyoPAXon; Drug: Tacrolimus

• Registration Number: NCT06692426
• Lead 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)

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design

Study Timeline

• Status Verified: 2024-11
• Study First Submitted: 2024-11-14
• Study First Submitted QC: 2024-11-14
• Last Update Submitted: 2024-11-14
• Study First Posted: 2024-11-18
• Last Update Posted: 2024-11-18
• Study Start: 2024-12-16
• Primary Completion: 2027-03-03
• Study Completion: 2027-03-03

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 8

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.

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm A: MyoPAXon 25 x 10^6	MyoPAXon	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
Arm A: MyoPAXon 25 x 10^6	Tacrolimus	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
Arm B: MyoPAXon 50 x 10^6	MyoPAXon	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
Arm B: MyoPAXon 50 x 10^6	Tacrolimus	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
Arm C: MyoPAXon 100 x 10^6	MyoPAXon	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
Arm C: MyoPAXon 100 x 10^6	Tacrolimus	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
Arm D: MyoPAXon 200 x 10^6	MyoPAXon	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
Arm D: MyoPAXon 200 x 10^6	Tacrolimus	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

Primary Outcome Measures

Name	Time	Method
Maximal tolerable dose (MTD) of MyoPAXon	3 months	Maximal tolerable dose (MTD) of MyoPAXon

Secondary Outcome Measures

Name	Time	Method
Proportion of patients developing humoral and cellular responses	3 months	Proportion of patients developing humoral (donor-specific anti-HLA antibodies) and cellular responses within 3 months