A Study of Evorpacept (ALX148) With Azacitidine for Higher Risk Myelodysplastic Syndrome (ASPEN-02)

A Phase 1/2 Study of Evorpacept (ALX148) in Combination With Azacitidine in Patients With Higher Risk Myelodysplastic Syndrome (MDS) (ASPEN-02)

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).

Study Overview

• Status: Terminated
• Conditions: Higher Risk Myelodysplastic Syndromes
• Intervention / Treatment: Drug: evorpacept; Drug: azacitidine

Detailed Description

The Phase 1 will consist of a dose escalation of evorpacept (ALX148) in combination with azacitidine to evaluate safety and tolerability, and to identify the recommended Phase 2 dose of ALX148 in combination with azacitidine. The Phase 2 will evaluate the efficacy of evorpacept (ALX148) in combination with azacitidine compared to azacitidine alone for patients with previously untreated higher risk MDS.

• Study Type: Interventional
• Enrollment (Actual): 65
• Phase: Phase 1

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Seoul National University Hospital
  • Seoul, Korea, Republic of
    • Asan Medical Center
  • Seoul, Korea, Republic of
    • Samsung Medical Center
  • Seoul, Korea, Republic of
    • Severance Hospital
  • Seoul, Korea, Republic of
    • Seoul Saint Mary's Hospital
• Spain
  • Alicante, Spain, 03010
    • Hospital General Universitario de Alicante
  • Cáceres, Spain, 10003
    • Hospital San Pedro de Alcantara
  • Salamanca, Spain, 37007
    • Hospital Universitario de Salamanca
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe de Valencia
• United States
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California, Norris Comprehensive Cancer Center
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University Feinberg School of Medicine
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Iu Simon Cancer Center
  • Michigan
    • Grand Rapids, Michigan, United States, 49546
      • START Midwest
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Levine Cancer Institute
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: evorpacept (ALX148) + azacitidine; 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: evorpacept; Fusion protein that blocks CD47-SIRPalpha pathway; Other Names: ALX148; Drug: azacitidine; Hypomethylating agent (HMA); Other Names: Vidaza
Active Comparator: azacitidine; Phase 2 only: Participants will receive azacitidine 75 mg/m2 IV or subcutaneous daily for 7 days of a 28-day cycle	Drug: azacitidine; Hypomethylating agent (HMA); Other Names: Vidaza

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1: Dose Limiting Toxicities (DLT)	Number of participants with a DLT	Up to 28 days
Phase 2: Complete response rate (CRR)	Number of participants achieving a complete response per International Working Group (IWG) criteria	Approximately 6 months
Phase 1: Recommended Phase 2 Dose (RP2D)	To identify the RP2D of ALX148 in combination with AZA	Approximately 2 years

Collaborators and Investigators

• Sponsor: ALX Oncology Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 2, 2020
• Primary Completion (Actual): January 17, 2025
• Study Completion (Actual): June 10, 2025

Study Registration Dates

• First Submitted: June 2, 2020
• First Submitted That Met QC Criteria: June 2, 2020
• First Posted (Actual): June 4, 2020

Study Record Updates

• Last Update Posted (Actual): July 20, 2025
• Last Update Submitted That Met QC Criteria: July 17, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: MDS; Azacitidine; CD47; ALX148; High risk; HMA; SIRP-alpha; evorpacept
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Hematologic Diseases; Precancerous Conditions; Bone Marrow Diseases; Syndrome; Preleukemia; Myelodysplastic Syndromes; Antineoplastic Agents; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Azacitidine
• Other Study ID Numbers: AT148002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No