Safety of MT-401-OTS in Patients With Relapsed AML or MDS

NCT06552416 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: MRKR-21-401-OTS5R01FD007272-02
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 3

Brief Summary

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) Those with intermediate or high-risk AML per 2022 ELN criteria who have evidence of MRD and/or \</= 10% blast following prior induction therapy or at least 4 cycles of nonintensive therapy and 2) those with high- or very-high-risk MDS per 2023 IWG criteria and who have residual disease with \</= 10% blasts following treatment with an HMA-based therapy.

Conditions

Acute Myeloid Leukemia, in Relapse; MDS

Outcome Measures

Primary Outcomes (2)

• Safety of MT-401-OTS

  Assess: * Incidence of DLTs to determine the RP2D * Safety (including but not limited to): TEAEs, SAEs, deaths, and clinical laboratory abnormalities per NCI CTCAE, Version 5.0 * Acute GVHD per 2016 MAGIC criteria * CRS and ICANS per ASTCT criteria

  Through study completion. Approximately 5 years

• Tolerability of MT-401-OTS

  cGVHD per 2014 NIH consensus criteria for cGVHD

  Through study completion. Approximately 5 years

Secondary Outcomes (2)

• To assess the efficacy of MT-401-OTS in participants with high-risk or very-high-risk MDS per IPSS-M who have evidence of disease following at least 4 cycles of a HMA

  Following conclusion of the disease assessment of last MDS subject treated. Approximately 2 years

• To assess the efficacy of MT-401-OTS in participants with intermediate or high-risk AML per 2022 ELN criteria who have evidence of disease following induction therapy or at least 4 cycles of nonintensive treatment

  Following conclusion of the disease assessment of last AML subject treated. Approximately 2 years

Study Arms (5)

Cohort -1

EXPERIMENTAL

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

Drug: MT-401-OTS

Cohort 1

EXPERIMENTAL

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

Drug: MT-401-OTS

Cohort 2

EXPERIMENTAL

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

Drug: MT-401-OTS

Cohort 3

EXPERIMENTAL

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

Drug: MT-401-OTS

Cohort 4 (optional)

EXPERIMENTAL

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

Drug: MT-401-OTS

Interventions

MT-401-OTS DRUG

MT-401-OTS is an off the shelf cellular therapy product given by IV infusion through either a peripheral or central line.

Cohort -1; Cohort 1; Cohort 2; Cohort 3; Cohort 4 (optional)

Eligibility Criteria

• Age: 65 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• General
• Must be ≥ 65 years of age and capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in the protocol, at the time of signing the ICF
• Must have a life expectancy ≥ 12 weeks
• Must have an ECOG performance status of 0-2
• Must have available MT-401-OTS product with a ≥ 2/8 HLA match Disease Characteristics
• For participants with AML:
• Must have a confirmed diagnosis of AML or MDS/AML per 2022 WHO Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms or 2022 International Consensus Criteria
• Must have intermediate or high-risk disease based on ELN 2022 criteria.
• If no targetable mutation is present, must have received 1 prior standard regimen with at least 4 cycles of standard therapy containing an HMA or a standard cytarabine-containing induction therapy
• If targetable mutation is present, must have received a regimen that includes commercially available targeted therapy unless unable to tolerate or the participant declines (must be documented in the informed consent). If targeted therapy was not administered as part of first-line of therapy, a second regimen is allowed.
• Must have either: ≤ 10% bone marrow blasts and ≤ 5% peripheral blasts during screening and not be considered to have hyperproliferating disease at diagnosis or after treatment OR Evidence of MRD based on evaluation at a local laboratory
• For participants with MDS:
• Must have confirmed diagnosis of MDS based on 2022 WHO Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms or 2022 ICC criteria
• Must have high-risk or very-high-risk disease based on IPSS-M (ie, not evolved to AML)
• Must have received standard treatment with at least 4 cycles of an HMA and have evidence of continued disease, including morphologic disease or MRD-positive

You may not qualify if:

• Disease-Related
• Have leukemic involvement in the CNS
• Have other extramedullary disease involvement (except hepatosplenic involvement)
• Have APL Medical Conditions
• Have primary immunodeficiency
• Have severe or uncontrolled autoimmune disorder
• Have a history or presence of clinically relevant CNS pathology, such as epilepsy, seizure, aphasia, stroke, severe brain injuries, dementia, Parkinson's disease, cerebellar disease, organic brain syndrome or psychosis
• Nonmelanoma skin cancer treated within the last 24 months that is considered completely cured
• Adequately treated breast lobular carcinoma in situ and breast ductal carcinoma in situ
• Adequately treated cervical carcinoma in situ without evidence of disease
• History of localized breast cancer and receiving antihormonal agents, or history of localized prostate cancer (N0M0) and receiving androgen-deprivation therapy
• A malignancy that is considered cured with minimal risk of recurrence
• Have any active systemic infection requiring therapy (viral, bacterial, or fungal), including HIV
• Have active hepatitis B or C infection or other clinically active liver diseases, as defined below:
• Seropositivity for hepatitis B as defined by a positive test for HbsAg Participants with resolved infection (ie, participants who are HbsAg-negative with antibodies to total anti-HBc with or without the presence of anti-HBs) must be screened using RT-PCR measurement of HBV DNA levels. Those who are RT PCR-positive will be excluded.

Sponsors & Collaborators

• Marker Therapeutics, Inc.: lead
• University of Kansas Medical Center: collaborator
• H. Lee Moffitt Cancer Center and Research Institute: collaborator
• City of Hope National Medical Center: collaborator
• FDA Office of Orphan Products Development: collaborator

Study Sites (3)

City of Hope Center (City of Hope National Medical Center, City of Hope Medical Center)

Duarte, California, 91006, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

KU Cancer Center

Kansas City, Kansas, 66160, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Patricia Allison, BS

CONTACT

713-400-6400; pallison@markertherapeutics.com

Beth Lepping, BSN

CONTACT

713-400-6400; elepping@markertherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Dose Escalation

Study Record Dates

• First Submitted: August 7, 2024
• First Posted: August 14, 2024
• Study Start: June 16, 2025
• Primary Completion (Estimated): September 1, 2029
• Study Completion (Estimated): September 1, 2029
• Last Updated: December 16, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)