Efficacy of MT-401 in Patients With AML Following Stem Cell Transplant

NCT04511130 | Terminated Phase 2 DMC FDA Drug

• 2 other identifiers: MRKR-19-401FD-R-7272
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 18

Brief Summary

This study is a Phase 2 multicenter study with a Safety Lead-in evaluating safety and efficacy of MT-401 administration to patients with AML, who have received their first allogeneic HSCT. The dose administered is 50 x 10\^6 cells (flat dosing).

Conditions

Acute Myeloid Leukemia; Stem Cell Transplantation

Outcome Measures

Primary Outcomes (4)

• Safety Lead-In

  Number of participants with MT-401 Dose Limiting Toxicities (DLTs)

  Baseline through Cycle 1 (28 Days)

• Phase 2 Adjuvant Group

  Relapse Free Survival (RFS), defined as the time from randomization to first disease recurrence or death from any cause.

  Up to 24 months after the first participant is randomized

• Phase 2 Active Disease Group

  Complete Remission (CR), per European LeukemiaNet (ELN) 2017 criteria

  Up to 12 months

• Phase 2 Active Disease Group

  Duration of CR (DOCR), defined as the time from the first observation of CR through disease recurrence or death from any cause

  Up to 24 months

Study Arms (3)

MT-401 following HSCT

EXPERIMENTAL

Treatment with MT-401 at 90 days following HSCT

Drug: MT-401

Standard of Care following HSCT

NO INTERVENTION

Standard of Care

MT-401 following relapse

EXPERIMENTAL

Treatment with MT-401 following relapse after first HSCT

Drug: MT-401

Interventions

MT-401 DRUG

MT-401 (zedenoleucel) is an allogeneic multi-tumor-associated antigen (MultiTAA)-specific T cell product manufactured under Good Manufacturing Practice (GMP) using donor-derived T cells obtained from apheresis.

Also known as: zedenoleucel

MT-401 following HSCT; MT-401 following relapse

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• First allogeneic HSCT, in ≤ CR2, and MRD negative prior to transplant (including matched sibling, MUD with at least 6 of 8 HLA markers, or haploidentical with at least 5 of 10 HLA markers) as:
• Adjuvant therapy for AML (Group 1) at 85-130 days post-HSCT defined as patients with CRMRD; or
• Treatment for refractory/relapsed AML (first relapse post-HSCT) when disease occurs after transplant (Group 2) defined as
• First relapse (MRD+ or frank relapse) post-HSCT
• Patients in Arm 1B (SOC) who experience first relapse (MRD+ or frank relapse) post HSCT
• Safety Lead-in defined as patients who fit all the criteria for Group 2 only
• Are ≥18 years of age
• Karnofsky/ Lansky score of ≥60
• Life expectancy ≥12 weeks
• Adequate blood, liver, and renal function
• Blood: Hemoglobin ≥7.0 g/dL (can be transfused)
• Liver: Bilirubin ≤2X upper limit of normal; aspartate aminotransferase ≤3X upper limit of normal
• Renal: Serum creatinine ≤2X upper limit of normal or measured or calculated creatinine clearance ≥45mL/min
• \. Patients are allowed to be on experimental conditioning regimens prior to transplant if no planned maintenance therapy post-transplant.
• \. In Group 2, patients may receive bridging therapy at the investigators' discretion in situations where MT-401 is not ready for administration or the treating physician believes the patient would benefit

You may not qualify if:

• Clinically significant or severely symptomatic intercurrent infection
• Pregnant or lactating
• For Group 1, anti-neoplastic therapy after HSCT and prior to or during dosing of MT-401
• For Group 2, concomitant anti-neoplastic therapy during or after dosing of MT-401
• Evidence of acute or chronic GVHD ≥Grade 2 (exception: acute or chronic Grade 2 GVHD of skin allowed if stable) within one week prior to receiving MT-401

Sponsors & Collaborators

• Marker Therapeutics, Inc.: lead

Study Sites (18)

University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States

Location

City of Hope National Medical Center

Duarte, California, 91010, United States

Location

Moores Cancer Center at University of Californa San Diego

La Jolla, California, 92093, United States

Location

UCLA Department of Medicine

Los Angeles, California, 90095, United States

Location

Yale Cancer Center

New Haven, Connecticut, 06519, United States

Location

Mayo Clinical Cancer Center-Florida

Jacksonville, Florida, 32224, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Winship Cancer Institute of Emory University

Atlanta, Georgia, 303222, United States

Location

University of Chicago

Chicago, Illinois, 77027, United States

Location

University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

Location

Mayo Clinic Cancer Center-Rochester

Rochester, Minnesota, 55905, United States

Location

John Theurer Cancer Center at Hackensack UMC

Hackensack, New Jersey, 07601, United States

Location

Weill Cornell Medicine | NewYork-Presbyterian

New York, New York, 10027, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Cleveland Clinic Taussig Cancer Center

Cleveland, Ohio, 44195, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

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

Study Officials

• Nishan Rajakumaraswamy, MD

  Marker Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2020
• First Posted: August 13, 2020
• Study Start: October 14, 2020
• Primary Completion: March 1, 2024
• Study Completion: March 1, 2024
• Last Updated: October 30, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (18)