Higher Risk Myelodysplastic SyndromesPhase 1NCT04417517
A Study of Evorpacept (ALX148) With Azacitidine for Higher Risk Myelodysplastic Syndrome (ASPEN-02)
A Phase 1 study testing evorpacept and azacitidine in people with Higher Risk Myelodysplastic Syndromes, run by ALX Oncology Inc. at 19 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Higher Risk Myelodysplastic Syndromes
- What is being tested?
- evorpacept and azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 19 sitesin 3 countries
- Study dates
- 2 Oct 2020to 10 Jun 2025
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
Trial locations and contacts
19 sites in 3 countries.
19 sites in 3 countries
United States10 sites
Levine Cancer Institute
Charlotte, North Carolina, 28204
Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
Duke University Medical Center
Durham, North Carolina, 27710
START Midwest
Grand Rapids, Michigan, 49546
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
IU Simon Cancer Center
Indianapolis, Indiana, 46202
University of Southern California, Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Vanderbilt University Medical Center
Nashville, Tennessee, 37203
Mayo Clinic
Rochester, Minnesota, 55905
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
South Korea5 sites
Asan Medical Center
Seoul
Samsung Medical Center
Seoul
Seoul National University Hospital
Seoul
Seoul Saint Mary's Hospital
Seoul
Severance Hospital
Seoul
Spain4 sites
Hospital General Universitario de Alicante
Alicante, 03010
Hospital San Pedro de Alcantara
Cáceres, 10003
Hospital Universitario de Salamanca
Salamanca, 37007
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
About this study
Official title: 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).
Read the 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.
- Conditions
- Higher Risk Myelodysplastic Syndromes
- Being tested
- evorpacept, azacitidine
- Keywords
- ALX148, MDS, CD47, Azacitidine, HMA, SIRP-alpha, evorpacept, High risk
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
evorpaceptDrug
Also called ALX148
Fusion protein that blocks CD47-SIRPalpha pathway
azacitidineDrug
Also called Vidaza
Hypomethylating agent (HMA)
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: evorpacept; Drug: 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 Receives: Drug: azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1: Dose Limiting Toxicities (DLT)
Number of participants with a DLT
Measured: Up to 28 days
Phase 1: Recommended Phase 2 Dose (RP2D)
To identify the RP2D of ALX148 in combination with AZA
Measured: Approximately 2 years
Phase 2: Complete response rate (CRR)
Number of participants achieving a complete response per International Working Group (IWG) criteria
Measured: Approximately 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2020
- Study start2 Oct 2020
- Primary completion17 Jan 2025
- Study completion10 Jun 2025
Why it stopped: While intended to be a Phase 1/2 clinical study, the study did not proceed to Phase 2.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Higher Risk Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- ALX Oncology Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04417517
- Sponsor's protocol ID
- AT148002
- Status last verified
- Jul 2025
- First submitted
- 2 Jun 2020
- Last updated
- 20 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No