NCT04417517

Brief Summary

This Phase 1/2 clinical study will evaluate evorpacept (ALX148) in combination with azacitidine for the treatment of patients with higher risk myelodysplastic syndrome (MDS).

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
65

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Oct 2020

Longer than P75 for phase_1

Geographic Reach
3 countries

19 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 2, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 4, 2020

Completed
4 months until next milestone

Study Start

First participant enrolled

October 2, 2020

Completed
4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 17, 2025

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 10, 2025

Completed
Last Updated

July 20, 2025

Status Verified

July 1, 2025

Enrollment Period

4.3 years

First QC Date

June 2, 2020

Last Update Submit

July 17, 2025

Conditions

Higher Risk Myelodysplastic Syndromes

Keywords

ALX148MDSCD47AzacitidineHMASIRP-alphaevorpaceptHigh risk

Outcome Measures

Primary Outcomes (3)

  • Phase 1: Dose Limiting Toxicities (DLT)

    Number of participants with a DLT

    Up to 28 days

  • Phase 1: Recommended Phase 2 Dose (RP2D)

    To identify the RP2D of ALX148 in combination with AZA

    Approximately 2 years

  • Phase 2: Complete response rate (CRR)

    Number of participants achieving a complete response per International Working Group (IWG) criteria

    Approximately 6 months

Study Arms (2)

evorpacept (ALX148) + azacitidine

EXPERIMENTAL

Phase 1: Participants will receive escalating doses of evorpacept (ALX148) in combination with azacitidine 75 mg/m2 IV or subcutaneous daily for 7 days of a 28 day cycle Phase 2: Participants will receive evorpacept (ALX148) at the recommended Phase 2 dose in combination with azacitidine 75 mg/m2 IV or subcutaneous daily for 7 days of a 28-day cycle

Drug: evorpaceptDrug: azacitidine

azacitidine

ACTIVE COMPARATOR

Phase 2 only: Participants will receive azacitidine 75 mg/m2 IV or subcutaneous daily for 7 days of a 28-day cycle

Drug: azacitidine

Interventions

evorpaceptDRUG

Fusion protein that blocks CD47-SIRPalpha pathway

Also known as: ALX148
evorpacept (ALX148) + azacitidine
azacitidineDRUG

Hypomethylating agent (HMA)

Also known as: Vidaza
azacitidineevorpacept (ALX148) + azacitidine

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Phase 1: Diagnosis of higher risk MDS that is either previously untreated or relapsed/refractory.
  • Phase 2: Diagnosis of higher risk MDS that is previously untreated.
  • Adequate renal and liver function.
  • Age ≥18 years.
  • Adequate performance status.

You may not qualify if:

  • Previous allogeneic hematopoietic stem cell transplant (allo-HSCT) for MDS or AML.
  • Prior treatment with any anti-CD47 or anti-SIRPα (signal regulatory protein alpha) agent.
  • Known active viral infections, including hepatitis B and C, human immunodeficiency virus (HIV), acquired immunodeficiency syndrome (AIDS) related illness, or SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

University of Southern California, Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

Northwestern University Feinberg School of Medicine

Chicago, Illinois, 60611, United States

Location

IU Simon Cancer Center

Indianapolis, Indiana, 46202, United States

Location

START Midwest

Grand Rapids, Michigan, 49546, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Levine Cancer Institute

Charlotte, North Carolina, 28204, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37203, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Asan Medical Center

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Seoul Saint Mary's Hospital

Seoul, South Korea

Location

Severance Hospital

Seoul, South Korea

Location

Hospital General Universitario de Alicante

Alicante, 03010, Spain

Location

Hospital San Pedro de Alcantara

Cáceres, 10003, Spain

Location

Hospital Universitario de Salamanca

Salamanca, 37007, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

MeSH Terms

Interventions

ALX148Azacitidine

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 2, 2020

First Posted

June 4, 2020

Study Start

October 2, 2020

Primary Completion

January 17, 2025

Study Completion

June 10, 2025

Last Updated

July 20, 2025

Record last verified: 2025-07

Data Sharing

IPD Sharing
Will not share

Locations

ES(4)US(10)KR(5)