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New treatment option for Duchenne Muscular Dystrophy
Official title Trial of Cell Based Therapy for DMD
ClinicalTrials.gov ID: NCT06692426
What this study is testing
What is MyoPAXon?
MyoPAXon is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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)
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on...
- 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
You likely can't join if
- Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
- Active treatment with another investigational therapy
- Known allergy to MyoPAXon components
See the full eligibility 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...
- 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 treatment 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.
- Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
- Active treatment with another investigational therapy
- Known allergy to MyoPAXon components
The study team makes the final eligibility decision.
Where it's taking place
- Minneapolis, Minnesota, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Minneapolis, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.