Cell-based therapy for Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 1 trial evaluates the safety and tolerability of local injections of induced pluripotent stem cell-derived muscle progenitor cells in individuals with Duchenne muscular dystrophy (DMD). Conducted at the University of Minnesota, the study focuses on non-ambulatory patients with specific muscle function criteria. Participants will receive the MyoPAXon treatment along with Tacrolimus, and will be monitored for 15 years to assess outcomes. The trial aims to gather preliminary data on the effectiveness of this innovative therapy.

Why it matters

Who can join: the full rules

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

Where it's running

• Masonic Cancer Center
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Study coordinator: Peter Kang, MD
• Email: mdstemcell@umn.edu
• Phone: 612-624-9452

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

  Recruiting at 2 sites Phase 2 Keros Therapeutics, Inc. · Tbilisi and 3 other locations · NCT07704099

• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.