Trial of Cell Based Therapy for DMD
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MyoPAXon, Tacrolimus.
- Who it may be relevant to
- Registry conditions: Duchenne Muscular Dystrophy. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
Overview
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.
Interventions
- Drug MyoPAXon
MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2 - 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.
Primary outcome measures
- Maximal tolerable dose (MTD) of MyoPAXon [Time frame: 3 months]
Secondary outcome measures (1)
- Proportion of patients developing humoral and cellular responses [Time frame: 3 months]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- Masonic Cancer Center — Minneapolis
Identifiers
NCT: NCT06692426 · 2021LS140