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

Phase 1

Active, not recruiting

• Conditions: Higher Risk Myelodysplastic Syndromes
• Interventions: Drug: azacitidine; Drug: evorpacept

• Registration Number: NCT04417517
• Lead Sponsor: ALX Oncology Inc.

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).

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 Timeline

• Study First Submitted: 2020-06-02
• Study First Submitted QC: 2020-06-02
• Study First Posted: 2020-06-04
• Study Start: 2020-10-02
• Primary Completion: 2025-01-17
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-01
• Last Update Posted: 2025-05-04
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 65

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
azacitidine	azacitidine	Phase 2 only: Participants will receive azacitidine 75 mg/m2 IV or subcutaneous daily for 7 days of a 28-day cycle
evorpacept (ALX148) + azacitidine	evorpacept	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
evorpacept (ALX148) + azacitidine	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

Primary Outcome Measures

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

Trial Locations

Locations (19)

University of Southern California, Norris Comprehensive Cancer Center; 🇺🇸 Los Angeles, California, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

IU Simon Cancer Center; 🇺🇸 Indianapolis, Indiana, United States

START Midwest; 🇺🇸 Grand Rapids, Michigan, United States

Northwestern University Feinberg School of Medicine; 🇺🇸 Chicago, Illinois, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Hospital Universitario de Salamanca; 🇪🇸 Salamanca, Spain

Hospital Universitari i Politecnic La Fe de Valencia; 🇪🇸 Valencia, Spain

Levine Cancer Institute; 🇺🇸 Charlotte, North Carolina, United States

Asan Medical Center; 🇰🇷 Seoul, Korea, Republic of

Fred Hutchinson Cancer Research Center; 🇺🇸 Seattle, Washington, United States

Seoul Saint Mary's Hospital; 🇰🇷 Seoul, Korea, Republic of

Hospital General Universitario de Alicante; 🇪🇸 Alicante, Spain

Severance Hospital; 🇰🇷 Seoul, Korea, Republic of

Hospital San Pedro de Alcantara; 🇪🇸 Cáceres, Spain

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

University of Texas MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States