A Phase 1b/2 Study of Ropeginterferon Alfa-2b Added to Azacitidine in Newly Diagnosed MDS/AML (≥ 10% Blasts) With TP53 Mutations

NCT07646821 | Not Yet Recruiting Phase 1 FDA Drug

• 2 other identifiers: 2026-0329NCI-2026-04492
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

To find the recommended dose of ropeginterferon alfa-2b in combination with azacitidine in patients with newly diagnosed MDS/AML with TP53 mutations. To learn if the recommended dose of ropeinterferon alfa-2b found in Dose Escalation in combination with azacitidine can help to control the disease.

Conditions

Ropeginterferon; Alfa-2b; Azacitidine; MDS/AML

Outcome Measures

Primary Outcomes (1)

• Safety and adverse events (AEs).

  Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

  Through study completion; an average of 1 year

Study Arms (2)

Dose Escalation

EXPERIMENTAL

Treatment with Ropeginterferon and AZA

Drug: ropeginterferon alfa- 2b; Drug: Azacitidine (AZA)

Dose Expansion,

EXPERIMENTAL

Drug: ropeginterferon alfa- 2b; Drug: Azacitidine (AZA)

Interventions

ropeginterferon alfa- 2b DRUG

Given by iv

Dose Escalation; Dose Expansion,

Azacitidine (AZA) DRUG

Given by IV

Dose Escalation; Dose Expansion,

Eligibility Criteria

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

You may not qualify if:

• Adult participants with MDS/AML without a known pathogenic TP53 mutation
• Treated secondary AML (AML developing after prior exposure to hypomethylating agents, chemotherapy or allogeneic HSCT for previous myeloid disorders like MDS, CMML, other MDS/MON overlap syndromes)
• Post MPN AML (advanced phase MPN)
• Participants with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
• Participants with active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
• Participants with known and untreated major depressive disorder
• Participants with known and active autoimmune disorder
• Participants with known retinal disease except for known age related macular degeneration
• Participants with another active cancer warranting therapy. A maintenance therapy for a stable/treated prior cancer is acceptable if it does not interfere with the trial therapy. This needs to be discussed with the PI.
• Participants who are receiving any other leukemia treatment.
• Participants who are receiving any other investigational agents.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to Ropeginterferon alfa-2b, AZA or other agents used in study.
• Participants with psychiatric illness/social situations that would limit compliance with study requirements.
• Pregnant women are excluded from this study because AZA and potentially rIFN\_A has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infant's secondary to treatment of the mother with the combination, breastfeeding should be discontinued if the mother is treated with combination. These potential risks may also apply to other agents used in this study.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• PharmaEssentia: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center

MeSH Terms

Interventions

Azacitidine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Jayastu Senapati, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jayastu Senapati, MD

CONTACT

713-745-9200; jsenapati@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2026
• First Posted: June 15, 2026
• Study Start (Estimated): November 27, 2026
• Primary Completion (Estimated): December 11, 2028
• Study Completion (Estimated): December 11, 2030
• Last Updated: June 15, 2026

Record last verified: 2026-06

Locations

US (1)