MT-401-OTS for Acute Myeloid Leukemia and Myelodysplastic Syndrome

(RAPID Trial)

Recruiting at 3 trial locations

Overseen By Beth Lepping, BSN

Age: 65+

Sex: Any

Trial Phase: Phase 1

Sponsor: Marker Therapeutics, Inc.

Must be taking: HMA

Must not be taking: Systemic steroids

Disqualifiers: CNS pathology, Active infections, Autoimmune, others

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

Compensation: Varies

Number of Visits: 4

Duration: 1 day

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This clinical trial tests the safety and effectiveness of a new treatment called MT-401-OTS for certain blood cancers, specifically Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS). The trial explores different doses to determine which works best with minimal side effects. Suitable participants have been diagnosed with AML or high-risk MDS and have received some treatment but still show signs of the disease. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but it mentions that participants should not be receiving concurrent therapies other than HMA (a type of treatment for certain blood disorders). It's best to discuss your current medications with the trial team to get a clear answer.

Is there any evidence suggesting that MT-401-OTS is likely to be safe for humans?

Research has shown that MT-401-OTS was generally well-tolerated in previous studies. Patients experienced no side effects related to the therapy. This aligns with earlier findings for similar treatments, which also showed few side effects. So far, the data suggests that MT-401-OTS is safe for humans, with no significant treatment-related issues reported. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for acute myeloid leukemia and myelodysplastic syndrome, which often rely on chemotherapy and bone marrow transplants, MT-401-OTS offers a novel cell-based therapy approach. Researchers are excited about MT-401-OTS because it uses a specific number of therapeutic cells tailored to potentially enhance the immune response against cancer cells. This treatment can be administered in varying doses, allowing for flexibility and personalization that could improve outcomes. Additionally, its ability to potentially deliver results more rapidly and with fewer side effects than traditional therapies is a promising advancement in treating these conditions.

What evidence suggests that MT-401-OTS could be an effective treatment for acute myeloid leukemia and myelodysplastic syndrome?

Research shows that MT-401-OTS could be a promising treatment for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). This trial will evaluate different dosages of MT-401-OTS, which uses special immune cells called MAR T cells to find and attack cancer cells by targeting four specific markers. Early studies have shown that these cells can help reduce the number of cancer cells in patients with these conditions. This method aims to strengthen the body's natural ability to fight cancer. Although more research is needed, these early results offer hope for people with AML and MDS. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for people aged 65 or older with specific blood cancers (AML or MDS) who have had some treatment but still show signs of disease. They should be able to consent, expect to live at least 12 weeks, and be fairly active (ECOG status 0-2). They can't join if they haven't tried certain therapies when appropriate unless there's a good reason.

Inclusion Criteria

I can take care of myself and am up and about more than 50% of my waking hours.

I have taken or was offered targeted therapy for my cancer's specific mutation.

This criterion is too general and needs specific details.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lymphodepleting Conditioning

Participants receive a lymphodepleting conditioning regimen consisting of fludarabine and cyclophosphamide, with bendamustine as an alternative

3 days

Daily visits for 3 days

Treatment

MT-401-OTS is administered intravenously over 10 minutes on Day 0

1 day

1 visit (in-person)

Safety Evaluation

Safety evaluations are performed throughout the study, including assessments of DLTs, TEAEs, SAEs, and other safety measures

Approximately 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment, including disease assessments at regular intervals

Approximately 2 years

What Are the Treatments Tested in This Trial?

Interventions

• MT-401-OTS

Trial Overview The study tests different doses of MT-401-OTS in patients with AML or high-risk MDS who've been treated before but still have evidence of disease. It's an early-phase trial to see how safe the drug is and how well it works.

How Is the Trial Designed?

Group I: Cohort 4 (optional) Experimental Treatment 1 Intervention

up to 400 × 106 cells flat dose TBD based on data from Cohorts 1-3; may include split dosing, dosing with or without lymphodepleting conditioning regimen, or dosing with or without HMA

Group II: Cohort 3 Experimental Treatment 1 Intervention

400 × 106 cells flat dose, 1 dose infused on Day 0

Group III: Cohort 2 Experimental Treatment 1 Intervention

200 × 106 cells flat dose, 1 dose infused on Day 0

Group IV: Cohort 1 Experimental Treatment 1 Intervention

100 × 106 cells flat dose, 1 dose infused on Day 0

Group V: Cohort -1 Experimental Treatment 1 Intervention

50 × 106 cells flat dose, 1 dose infused on Day 0

Who Is Running the Clinical Trial?

Marker Therapeutics, Inc.

Lead Sponsor

Trials

8

Recruited

450+

City of Hope National Medical Center

Collaborator

Trials

17

Recruited

8,900+

University of Kansas Medical Center

Collaborator

Trials

527

Recruited

181,000+

The University of Kansas Medical Center

Collaborator

Trials

3

Recruited

870+

H. Lee Moffitt Cancer Center and Research Institute

Collaborator

Trials

576

Recruited

145,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06552416

Study Details | NCT06552416 | Safety of MT-401-OTS in ... This study is a Phase 1 multicenter, open-label study evaluating the safety and efficacy of escalating doses of MT-401-OTS in 2 participant populations: 1) ...

2. ir.markertherapeutics.com ir.markertherapeutics.com/news-releases/news-release-details/marker-therapeutics-announces-first-patient-treated-shelf

Marker Therapeutics Announces First Patient Treated in Off ... Marker is evaluating the safety and efficacy of escalating doses of MT-401, a multi-antigen recognizing (MAR) T cell product targeting four ...

3. sec.gov sec.gov/Archives/edgar/data/1094038/000110465925096935/tm2527906d1_ex99-1.htm

Marker Therapeutics Announces First Patient Treated in Off- ... Marker is evaluating the safety and efficacy of escalating doses of MT-401, a multi-antigen recognizing (MAR) T cell product targeting four antigens, as an OTS ...

4. app.trialscreen.org app.trialscreen.org/trials/phase-1-safety-mt-401-ots-patients-relapsed-aml-or-mds-trial-nct06552416

Safety of MT-401-OTS in Patients With Relapsed AML or ... A Study to Evaluate the Safety and Efficacy of MT-401-OTS in Patients with Relapsed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome. Check your ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8793782/

PMC - PubMed Central APVO436 showed promising tolerability and single-agent activity in relapsed or refractory (R/R) acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

6. sciencedirect.com sciencedirect.com/science/article/pii/S2152265022012228

AML-109 Preliminary Results of MT-401 in Post-Transplant ... A multicenter Phase 2 study (ARTEMIS) evaluating the safety and efficacy of MT-401 in patients with AML post-transplant is ongoing and includes patients with ...

7. pharmanow.live pharmanow.live/latest-news/marker-therapeutics-mt401-ots-phase1

Marker Begins OTS MAR-T Clinical Trial Treatment was well tolerated with no therapy-related adverse events, consistent with previously reported favorable safety data for MAR-T cells.

8. sahmcapital.com sahmcapital.com/news/content/marker-therapeutics-announces-first-patient-treated-in-ots-program-with-encouraging-preliminary-safety-data-2025-10-06

Marker Therapeutics Announces First Patient Treated In ... The therapy was well tolerated with no treatment-related adverse events. This observation is consistent with the favorable safety profile and ...