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

NCT ID: NCT04511130

Last Updated: 2025-10-30

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 92 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-10-14
• Study Completion Date: 2024-03-01

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).

Detailed Description

This study is in patients aged ≥18 years old undergoing or having relapsed after their first allogeneic HSCT (matched sibling, matched unrelated donor, or haploidentical transplants) for AML.

Potential patients for the study may be screened/enrolled:

• Prior to their first allogeneic HSCT.

or

• Patients experiencing their first relapse post-allogeneic transplant.

Patients eligible for the study will be placed into one of two groups:

• Adjuvant (Group 1): Patients screened prior to their HSCT with CR without minimal residual disease (CRMRD-) at 85-130 days post transplant will be randomized (1:1) in an unblinded fashion to:

• MT-401 (Arm A)
• SOC (Arm B)
• Active Disease: (Group 2): Patients meeting the following criteria will be assigned to Group 2 and will receive MT 401:

• Patients who experience relapse (patients with MRD [MRD+] or frank relapse) at or prior to post-transplant Day 85-130
• Patients in Arm B of Group 1 (SOC) who develop relapse (MRD+ or frank relapse) post-HSCT (crossover patients)
• Patients who do not consent prior to HSCT but are experiencing their first relapse (MRD+ or frank relapse) and have the same donor available for manufacturing

Conditions

Acute Myeloid Leukemia; Stem Cell Transplantation

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

MT-401 following HSCT

Treatment with MT-401 at 90 days following HSCT

Group Type: EXPERIMENTAL

MT-401

Intervention Type: 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.

Standard of Care following HSCT

Standard of Care

Group Type: NO_INTERVENTION

No interventions assigned to this group

MT-401 following relapse

Treatment with MT-401 following relapse after first HSCT

Group Type: EXPERIMENTAL

MT-401

Intervention Type: 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.

Interventions

MT-401

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.

Intervention Type: DRUG

Other Intervention Names

zedenoleucel

Eligibility Criteria

Inclusion Criteria

1. 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

2. Are ≥18 years of age

3. Karnofsky/ Lansky score of ≥60

4. Life expectancy ≥12 weeks

5. 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

7. Patients are allowed to be on experimental conditioning regimens prior to transplant if no planned maintenance therapy post-transplant.

8. 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

Exclusion Criteria

1. Clinically significant or severely symptomatic intercurrent infection

2. Pregnant or lactating

3. For Group 1, anti-neoplastic therapy after HSCT and prior to or during dosing of MT-401

4. For Group 2, concomitant anti-neoplastic therapy during or after dosing of MT-401

5. 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Marker Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nishan Rajakumaraswamy, MD

Role: STUDY_DIRECTOR

Marker Therapeutics

Locations

University of Alabama at Birmingham

Birmingham, Alabama, United States

City of Hope National Medical Center

Duarte, California, United States

Moores Cancer Center at University of Californa San Diego

La Jolla, California, United States

UCLA Department of Medicine

Los Angeles, California, United States

Yale Cancer Center

New Haven, Connecticut, United States

Mayo Clinical Cancer Center-Florida

Jacksonville, Florida, United States

Moffitt Cancer Center

Tampa, Florida, United States

Winship Cancer Institute of Emory University

Atlanta, Georgia, United States

University of Chicago

Chicago, Illinois, United States

University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Mayo Clinic Cancer Center-Rochester

Rochester, Minnesota, United States

John Theurer Cancer Center at Hackensack UMC

Hackensack, New Jersey, United States

Weill Cornell Medicine | NewYork-Presbyterian

New York, New York, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

Cleveland Clinic Taussig Cancer Center

Cleveland, Ohio, United States

Baylor College of Medicine

Houston, Texas, United States

MD Anderson Cancer Center

Houston, Texas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

FD-R-7272

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

MRKR-19-401

Identifier Type: -

Identifier Source: org_study_id