Study of MGTA-117 in Patients With Adult Acute Myeloid Leukemia (AML) and Myelodysplasia-Excess Blasts (MDS-EB)

NCT05223699 | Terminated Phase 1 FDA Drug

• 1 other identifier: 117-HEM-101
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 8

Brief Summary

This research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB.

Conditions

Acute Myeloid Leukemia; Myelodysplasia

Keywords

Acute Myeloid Leukemia; Myelodysplasia-Excess Blasts; MGTA-117; Hematopoietic stem cell transplant

Outcome Measures

Primary Outcomes (10)

• Incidence rate of treatment emergent adverse events (TEAEs) leading to study drug discontinuation

  21 days

• Incidence rate of treatment emergent >= Grade 3 clinical laboratory abnormalities as assessed by CTCAE v5.0

  21 days

• Assess the clinically significant changes from baseline in vital signs, ECGs and laboratory parameters

  21 days

• Pharmacokinetics profile of MGTA-117

  Investigate area under the curve (AUC)

  21 days

• Pharmacokinetics profile of MGTA-117

  Investigate maximum plasma concentration (Cmax)

  21 days

• Pharmacokinetics profile of MGTA-117

  Investigate time of maximum concentration (Tmax)

  21 days

• Pharmacokinetics profile of MGTA-117

  Investigate the half-life (t1/2)

  21 days

• Pharmacokinetics profile of MGTA-117

  Investigate the plasma concentration

  21 days

• To establish a minimum safe and biologically effective dose

  Assess the CD117 receptor occupancy in circulating leukemic blasts

  7 days

• To establish a minimum safe and biologically effective dose

  The incidence of qualifying protocol-defined dose-limiting toxicities

  21 days

Study Arms (1)

Single dose MGTA-117

EXPERIMENTAL

Dosing of MGTA-117 prepared and administered by IV infusion.

Biological: MGTA-117

Interventions

MGTA-117 BIOLOGICAL

MGTA-117 will be administered as an IV infusion

Single dose MGTA-117

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must have a World Health Organization (WHO)-defined diagnosis of R/R AML and meet one of the following criteria:
• \- The participant has experienced primary AML induction failure or R/R AML
• \- The participant has a WHO-defined diagnosis of MDS-EB and has failed/is refractory to HMA
• \- Presence of MRD in morphologic CR
• CD117+ based on IHC or flow cytometry
• Participant must have an identified HSC donor (related donor or unrelated donor), haplo-identical transplant donor, or umbilical blood donor.
• Participant's Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
• Participant must have adequate baseline hepatic function. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP) ≤2 x upper limit of normal (ULN), and serum bilirubin ≤1.5 x ULN.
• Estimated creatinine clearance ≥60 mL/min
• Adequate cardiac function as demonstrated by cardiac left ventricular ejection fraction ≥40% or perform New York Heart Association (NYHA) classification I and II

You may not qualify if:

• Acute promyelocytic leukemia (APL).
• Known active central nervous system (CNS) leukemia or chloroma (granulocyte sarcoma).
• Received HSCT within 6 months prior to dosing
• Received chimeric antigen-receptor cell therapies within 6 months prior to dosing
• Has active graft-versus-host disease (GVHD).
• Active hepatitis B (Hep-B) or hepatitis C (Hep-C) infection or history of human immunodeficiency virus (HIV).
• Participant with a QTc value \>470 msec
• Participant has received another investigational drug or device within 14 days or 5 half-lives of dosing, whichever is longer.
• Participant has any clinically significant medical condition, which in the opinion of the Investigator may place the participant at an unacceptable risk.
• Active uncontrolled systemic bacterial, fungal, or viral infection
• Participant has a history of serious allergic reactions, which in the opinion of the Investigator may pose an increased risk of serious infusion reactions.
• Participant has had any systemic antileukemia treatment within 14 days except hydroxyurea, which is permitted until 24 hours prior to MGTA-117 dosing.
• Participant has received prior anti-CD117 antibody treatment.
• Participant has received gemtuzumab ozogamicin (Mylotarg) within the last 3 months prior to dosing.
• Participant has received recent monoclonal antibody as anti-leukemic therapy within the last 30 days or 5 half-lives, whichever is longer.

Sponsors & Collaborators

• Magenta Therapeutics, Inc.: lead

Study Sites (8)

City of Hope

Duarte, California, 91010, United States

Location

Sarah Cannon Research Institute at HealthONE

Denver, Colorado, 80218, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

The University of Kansas Cancer Center

Westwood, Kansas, 66205, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Roswell Park Comprehensive Cancer Center

Buffalo, New York, 14203, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Anemia, Refractory, with Excess of Blasts

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

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

Study Record Dates

• First Submitted: January 14, 2022
• First Posted: February 4, 2022
• Study Start: February 14, 2022
• Primary Completion: February 2, 2023
• Study Completion: February 2, 2023
• Last Updated: February 9, 2023

Record last verified: 2023-02

Locations

US (8)