Duchenne Muscular DystrophyPhase 1NCT06692426
Trial of Cell Based Therapy for DMD
A Phase 1 study testing MyoPAXon and Tacrolimus in people with Duchenne Muscular Dystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- MyoPAXon and Tacrolimus
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Mar 2025to 3 Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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):
Show all 10Show fewer
- 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.
Who cannot join
- Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
- Active treatment with another investigational therapy
- Known allergy to MyoPAXon components
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Masonic Cancer Center
Minneapolis, Minnesota, 55455
Peter Kang, MD
About this study
Official title: Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
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)
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- MyoPAXon, Tacrolimus
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MyoPAXonDrug
MyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2
TacrolimusDrug
Also called Prograf
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm A: MyoPAXon 25 x 10^6 | Experimental | 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 Receives: Drug: MyoPAXon; Drug: Tacrolimus |
| Arm B: MyoPAXon 50 x 10^6 | Experimental | 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 Receives: Drug: MyoPAXon; Drug: Tacrolimus |
| Arm C: MyoPAXon 100 x 10^6 | Experimental | 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 Receives: Drug: MyoPAXon; Drug: Tacrolimus |
| Arm D: MyoPAXon 200 x 10^6 | Experimental | 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 Receives: Drug: MyoPAXon; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximal tolerable dose (MTD) of MyoPAXon
Maximal tolerable dose (MTD) of MyoPAXon
Measured: 3 months
Secondary outcomes (1)
Proportion of patients developing humoral and cellular responses
Proportion of patients developing humoral (donor-specific anti-HLA antibodies) and cellular responses within 3 months
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2024
- Study start20 Mar 2025
- Primary completion3 Mar 2027 (estimated)
- Study completion3 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- Duchenne UK, Parent Project Muscular Dystrophy
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06692426
- Sponsor's protocol ID
- 2021LS140
- Status last verified
- Mar 2026
- First submitted
- 14 Nov 2024
- Last updated
- 4 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT06692426
- Acronym / sponsor ID
- 2021LS140
- Conditions
- Duchenne Muscular Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Collaborators
- Duchenne UK, Parent Project Muscular Dystrophy
- Study start
- 20 Mar 2025 (actual)
- Primary completion
- 3 Mar 2027 (estimated)
- Study completion
- 3 Mar 2027 (estimated)
- First posted
- 18 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Arm A: MyoPAXon 25 x 10^6 | Experimental | Drug: MyoPAXon, Drug: TacrolimusMyoPAXon 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 | Experimental | Drug: MyoPAXon, Drug: TacrolimusMyoPAXon 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 | Experimental | Drug: MyoPAXon, Drug: TacrolimusMyoPAXon 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 | Experimental | Drug: MyoPAXon, Drug: TacrolimusMyoPAXon 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (3 listed)
- Presence of HLA antibodies directed toward HLA antigens on MyoPAXon
- Active treatment with another investigational therapy
- Known allergy to MyoPAXon components
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MyoPAXonMyoPAXon is a CD54+ allogeneic muscle progenitor cell product derived from the iPSC line LiPSC-ER2.2 | Drug | Arm A: MyoPAXon 25 x 10^6, Arm B: MyoPAXon 50 x 10^6, Arm C: MyoPAXon 100 x 10^6, Arm D: MyoPAXon 200 x 10^6 |
| TacrolimusAlso: PrografTacrolimus (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. | Drug | Arm A: MyoPAXon 25 x 10^6, Arm B: MyoPAXon 50 x 10^6, Arm C: MyoPAXon 100 x 10^6, Arm D: MyoPAXon 200 x 10^6 |
Primary outcome
Maximal tolerable dose (MTD) of MyoPAXon
Time frame: 3 months
Maximal tolerable dose (MTD) of MyoPAXon
1 secondary outcome
Proportion of patients developing humoral and cellular responses
Time frame: 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MyoPAXon and Tacrolimus in people with Duchenne Muscular Dystrophy, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Peter Kang, MD (612-624-9452, mdstemcell@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximal tolerable dose (MTD) of MyoPAXon (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2026.