Cell-Based Therapy for Duchenne Muscular Dystrophy

Overseen By Peter Kang, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Masonic Cancer Center, University of Minnesota

Disqualifiers: HLA antibodies, Investigational therapy, Allergy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

8 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, MyoPAXon (a cell-based therapy), to determine its safety and tolerability in people with Duchenne muscular dystrophy (DMD). Researchers aim to understand how injections of muscle-building cells might benefit those who are non-ambulatory, meaning they cannot walk. Participants will receive one of four different doses of MyoPAXon injected into a specific muscle and will take tacrolimus for a few months to support the treatment. Individuals diagnosed with DMD who are not currently on other experimental treatments might be suitable for this study. As a Phase 1 trial, this research focuses on understanding the treatment's effects in people, offering participants the opportunity to be among the first to receive this new therapy.

Do I need to stop my current medications for the trial?

The trial protocol does not specify if you need to stop your current medications, but you must be off investigational therapies for more than 30 days before joining.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that MyoPAXon has been tested in animal studies with monkeys. In these studies, between 37 to 60 million cells were transplanted, aiding in the formation of muscle fibers and repair cells. Importantly, no major side effects were reported.

As this treatment is in a phase 1 trial, the main goal is to assess its safety and tolerability in humans. Although detailed results in humans aren't available yet, phase 1 trials typically involve careful monitoring to ensure safety. This trial will test different doses of MyoPAXon, ranging from 25 to 200 million cells, all injected into a specific muscle. Participants will also take tacrolimus to help their bodies accept the new cells, a common practice in such studies.

While MyoPAXon has shown promise in early animal studies, this trial aims to provide more information about its safety in humans. Researchers will closely monitor participants for any side effects or issues. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne muscular dystrophy?

Researchers are excited about MyoPAXon because it offers a novel approach to treating Duchenne Muscular Dystrophy (DMD) by using cell-based therapy to potentially regenerate muscle tissue. Unlike traditional treatments such as corticosteroids, which mainly aim to slow muscle damage, MyoPAXon involves delivering muscle progenitor cells directly into a muscle to promote repair and growth. This targeted method, combined with the supportive use of tacrolimus to enhance cell survival and integration, could lead to more effective and lasting improvements in muscle function for individuals with DMD.

What evidence suggests that MyoPAXon might be an effective treatment for Duchenne muscular dystrophy?

Research has shown that MyoPAXon, a treatment using special cells, could help with Duchenne muscular dystrophy (DMD). In animal studies, these cells have helped build muscle fibers, suggesting they might also repair muscles in people. In this trial, participants will receive MyoPAXon injections into muscles, potentially strengthening them by creating new muscle cells. This method uses special stem cells that can transform into different cell types needed for muscle repair. Although human data is limited, early animal results are promising for this treatment. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Duchenne muscular dystrophy, confirmed by gene mutations or muscle biopsy. Participants must be non-ambulatory but have intact EDB muscles and off other investigational therapies for over 30 days. They should commit to birth control methods if applicable and consent to a long-term follow-up of 15 years.

Inclusion Criteria

Off investigational therapies for more than 30 days

Voluntary written consent from the subject or parent(s)/guardian(s) and assent from participant prior to the performance of any research related activity.

I cannot walk by myself.

Exclusion Criteria

Active treatment with another investigational therapy

Known allergy to MyoPAXon components

My body has antibodies against specific antigens on MyoPAXon.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Treatment

Participants are started on tacrolimus 1 week prior to dosing

1 week

Treatment

MyoPAXon is delivered via open intramuscular injection into a single EDB muscle as a one-time dose

1 day

Post-Treatment

Participants stay on the maintenance dose of tacrolimus on a BID schedule

3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• MyoPAXon

Trial Overview The trial tests the safety of injecting stem cell-derived muscle progenitor cells into patients with DMD. It's an early-stage study (phase 1) focusing on how well participants tolerate this new cell-based therapy called MyoPAXon, alongside Tacrolimus to prevent rejection.

How Is the Trial Designed?

Group I: Arm D: MyoPAXon 200 x 10^6 Experimental Treatment 2 Interventions

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

Group II: Arm C: MyoPAXon 100 x 10^6 Experimental Treatment 2 Interventions

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

Group III: Arm B: MyoPAXon 50 x 10^6 Experimental Treatment 2 Interventions

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

Group IV: Arm A: MyoPAXon 25 x 10^6 Experimental Treatment 2 Interventions

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

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota

Lead Sponsor

Trials

285

Recruited

15,700+

Parent Project Muscular Dystrophy

Collaborator

Trials

7

Recruited

11,000+

Duchenne UK

Collaborator

Trials

2

Recruited

40+

Published Research Related to This Trial

The first-in-human study of DEC cell therapy for Duchenne Muscular Dystrophy (DMD) showed no adverse events in the first 3 patients over 14 months, indicating a strong safety profile for this novel treatment.

Patients receiving DEC therapy demonstrated significant improvements in muscle function and strength, as measured by various functional tests, suggesting that this therapy could effectively enhance quality of life for DMD patients without the need for immunosuppression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dystrophin Expressing Chimeric (DEC) Cell Therapy for Duchenne Muscular Dystrophy: A First-in-Human Study with Minimum 6 Months Follow-up. Heydemann, A., Bieganski, G., Wachowiak, J., et al.[2023]

Transplantation of dystrophin-expressing chimeric (DEC) cells derived from myoblasts significantly increased dystrophin expression in the skeletal, cardiac, and respiratory muscles of a mouse model for Duchenne muscular dystrophy (DMD), leading to improved muscle function and reduced pathology.

The study demonstrated that DEC transplantation not only preserved heart function and respiratory capacity but also improved muscle strength and reduced inflammation and fibrosis, suggesting a promising new therapeutic approach for enhancing the overall health of DMD patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human dystrophin expressing chimeric (DEC) cell therapy ameliorates cardiac, respiratory, and skeletal muscle's function in Duchenne muscular dystrophy. Siemionow, M., Langa, P., Harasymczuk, M., et al.[2022]

The DT-DEC01 cell therapy demonstrated a strong safety profile with no adverse events reported up to 21 months after administration, indicating it is a safe treatment option for patients with Duchenne Muscular Dystrophy (DMD).

Functional improvements were observed in patients, including better performance in the 6-Minute Walk Test and other assessments, suggesting that DT-DEC01 may effectively enhance muscle function and overall health in DMD patients over a 12-month period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Efficacy of DT-DEC01 Therapy in Duchenne Muscular Dystrophy Patients: A 12 - Month Follow-Up Study After Systemic Intraosseous Administration. Siemionow, M., Biegański, G., Niezgoda, A., et al.[2023]

The novel DT-DEC01 therapy, which uses Dystrophin Expressing Chimeric (DEC) cells, was found to be safe in a small study of 3 boys aged 6-15 years, with no therapy-related adverse events reported up to 22 months after treatment.

Preliminary efficacy was observed through electromyography (EMG) assessments, showing increased motor unit potentials in treated muscles at 12 months, indicating potential benefits for muscle function in boys with Duchenne muscular dystrophy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration. Niezgoda, A., Biegański, G., Wachowiak, J., et al.[2023]

A new immunodeficient rat model of Duchenne muscular dystrophy (DMD) was developed, showing similar disease characteristics to human patients, which is crucial for studying potential treatments.

Successful engraftment of human myoblasts in this rat model indicates its potential for preclinical studies on cell transplantation therapies for DMD, paving the way for future research in this area.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation. Sato, M., Goto, M., Yamanouchi, K., et al.[2023]

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S152500162500543X

Preclinical quality, safety, and efficacy of a CGMP iPSC- ... Transplantation of 37–60 million MyoPAXon cells into immunosuppressed non-human primates showed human contribution to muscle fibers and satellite cells, with no ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06692426

NCT06692426 | Trial of Cell Based Therapy for DMD Duchenne muscular dystrophy, diagnosed by mutations in the DMD (dystrophin) gene and/or absence of immunohistochemical staining for dystrophin on muscle biopsy ...

3. reporter.nih.gov reporter.nih.gov/project-details/10822639

Systemic Transplantation of MyoPAXon Systemic MyoPAXon delivery will allow for the targeting of multiple skeletal muscles with a single injection, making it a compelling therapy for DMD patients ...

4. clinicaltrial.be clinicaltrial.be/en/details/459094?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

Trial of Cell Based Therapy for DMD Phase I Clinical Trial of Cell Based Therapy for Duchenne Muscular Dystrophy ... MyoPAXon 50 x 10^6, Arm C: MyoPAXon 100 x 10^6, Arm D ...

5. rarediseaseadvisor.com rarediseaseadvisor.com/hcp-resource/duchenne-muscular-dystrophy-clinical-trials/

Duchenne Muscular Dystrophy Clinical Trials As of November 2024, approximately 156 clinical trials for Duchenne muscular dystrophy (DMD) are ongoing or pending.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06692426

NCT06692426 | Trial of Cell Based Therapy for DMD 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 ( ...

7. parentprojectmd.org parentprojectmd.org/duchenne-uk-and-ppmd-award-500000-to-evaluate-safety-and-tolerability-of-muscle-progenitor-cells-in-phase-1-trial/

Duchenne UK and PPMD Award $500000 to Evaluate ... Duchenne UK and PPMD Award $500,000 to Evaluate Safety, Tolerability of Muscle Progenitor Cells in Phase 1 Trial. Parent Project Muscular ...

8. neurologylive.com neurologylive.com/view/fda-clears-ind-application-myogenica-myopaxon-muscular-dystrophy

FDA Clears IND Application for Myogenica's MyoPAXon in ... The trial will focus on assessing safety, tolerability, and engraftment of MyoPAXon in adult patients with nonambulatory Duchenne muscular ...