Clinical trial NCT06692426: Trial of Cell Based Therapy for DMD
Official title: Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Duchenne Muscular Dystrophy
Lead sponsor: Masonic Cancer Center, University of Minnesota
Sponsor class: OTHER
Collaborators: name: Duchenne UK
name: Parent Project Muscular Dystrophy
First posted: 2024-11-18
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Maximal tolerable dose (MTD) of MyoPAXon
timeFrame: 3 months
Secondary outcomes:
measure: Proportion of patients developing humoral and cellular responses
timeFrame: 3 months
Other outcomes:
Locations:
city: Minneapolis
country: United States
facility: Masonic Cancer Center
state: Minnesota
Study officials:
References: