RopeginterferonPhase 1/2NCT07646821
A Phase 1b/2 Study of Ropeginterferon Alfa-2b Added to Azacitidine in MDS/AML With TP53 Mutations
A Phase 1/2 study testing ropeginterferon alfa- 2b and Azacitidine (AZA) in people with Ropeginterferon and Alfa-2b, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ropeginterferon, Alfa-2b, Azacitidine
- What is being tested?
- ropeginterferon alfa- 2b and Azacitidine (AZA)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Oct 2026to 11 Dec 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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
None listed.
Who cannot join
- Adult patients with MDS/AML without a known pathogenic TP53 mutation
- Patients with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
- Patient 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).
- Patient with known and untreated major depressive disorder
- Recent stroke or myocardial infarction (in previous 6 months)
- Patient with known retinal disease except for known age related macular degeneration
Show all 18Show fewer
- Patient 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.
- Patients who are receiving any other leukemia treatment.
- Patients 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.
- Patients with psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant women are excluded from this study because AZA and potentially ropeginterferon has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants 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.
- Patients with severe or unstable cardiovascular disease, e.g., uncontrolled hypertension, congestive heart failure (≥ NYHA class 2), serious cardiac arrhythmia, significant coronary artery stenosis, unstable angina).
- Patients with eGFR <30 mL/min at screening.
- Patients with only extramedullary disease
- For Cohort 1B only (newly diagnosed cohort)
- Treated secondary AML (AML developing after prior exposure to hypomethylating agents, chemotherapy or allogeneic HSCT for previous myeloid disorders like MDS, CMML, other MDS/MPN overlap syndromes)
- Post MPN AML (advanced phase MPN)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Jayastu Senapati,, MD, 713-745-9200, jsenapati@mdanderson.org
Jayastu Senapati, MD
About this study
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.
Read the detailed description
Primary Objectives
Cohort 1A To evaluate the safety of ropeginterferon monotherapy and to determine the recommended phase 2 dose (RP2D) of ropeginterferon in patients with R/R MDS/AML with ≥5% blasts
Cohort 1B To evaluate the safety and determine the RP2D of ropeginterferon in combination with AZA in patients with newly diagnosed MDS/AML with ≥10% blasts
Secondary Objectives
To observe and record anti-tumor activity. Although the clinical benefit of ropeginterferon has not yet been established, the intent of offering this treatment is to provide a possible therapeutic benefit, and thus the patient will be carefully monitored for tumor response and symptom relief in addition to safety and tolerability.
Key secondary objective:
To assess the efficacy through EFS with AZA+ ropeginterferon separately in the R/R (Cohort 1A) and newly diagnosed patients (Cohort1B)
Other secondary objectives (separately for Cohort1A and Cohort1B):
- CR+CRi (CRc) rates achieved within 4 cycles of therapy
- Overall response rate (ORR; CRc+ morphologic leukemia free state)
- Rate of MRD negative CRc by multiparameter flow cytometry (MFC) at 10-4
- To determine duration of response (CRc and OR)
- To determine median duration of overall survival (OS)
- To determine rates of HSCT after study therapy
- Conditions
- Ropeginterferon, Alfa-2b, Azacitidine, MDS/AML
- Being tested
- ropeginterferon alfa- 2b, Azacitidine (AZA)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ropeginterferon alfa- 2bDrug
Given by iv
Azacitidine (AZA)Drug
Given by IV
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation | Experimental | Treatment with Ropeginterferon and AZA Receives: Drug: ropeginterferon alfa- 2b; Drug: Azacitidine (AZA) |
| Dose Expansion, | Experimental | Not described Receives: Drug: ropeginterferon alfa- 2b; Drug: Azacitidine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2026
- Study start27 Oct 2026 (estimated)
- Primary completion11 Dec 2028 (estimated)
- Study completion11 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RopeginterferonStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- PharmaEssentia
- Responsible party
- Sponsor
- Investigators
- Jayastu Senapati, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07646821
- Sponsor's protocol ID
- 2026-0329
- Other IDs
- NCI-2026-04492
- Status last verified
- Oct 2026
- First submitted
- 9 Jun 2026
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b/2 Study of Ropeginterferon Alfa-2b Added to Azacitidine in MDS/AML With TP53 Mutations
- ClinicalTrials.gov ID
- NCT07646821
- Acronym / sponsor ID
- 2026-0329
- Conditions
- Ropeginterferon, Alfa-2b, Azacitidine, MDS/AML
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- PharmaEssentia
- Study start
- 27 Oct 2026 (estimated)
- Primary completion
- 11 Dec 2028 (estimated)
- Study completion
- 11 Dec 2030 (estimated)
- First posted
- 15 Jun 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Primary Objectives
Cohort 1A To evaluate the safety of ropeginterferon monotherapy and to determine the recommended phase 2 dose (RP2D) of ropeginterferon in patients with R/R MDS/AML with ≥5% blasts
Cohort 1B To evaluate the safety and determine the RP2D of ropeginterferon in combination with AZA in patients with newly diagnosed MDS/AML with ≥10% blasts
Secondary Objectives
To observe and record anti-tumor activity. Although the clinical benefit of ropeginterferon has not yet been established, the intent of offering this treatment is to provide a possible therapeutic benefit, and thus the patient will be carefully monitored for tumor response and symptom relief in addition to safety and tolerability.
Key secondary objective:
To assess the efficacy through EFS with AZA+ ropeginterferon separately in the R/R (Cohort 1A) and newly diagnosed patients (Cohort1B)
Other secondary objectives (separately for Cohort1A and Cohort1B):
- CR+CRi (CRc) rates achieved within 4 cycles of therapy
- Overall response rate (ORR; CRc+ morphologic leukemia free state)
- Rate of MRD negative CRc by multiparameter flow cytometry (MFC) at 10-4
- To determine duration of response (CRc and OR)
- To determine median duration of overall survival (OS)
- To determine rates of HSCT after study therapy
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation | Experimental | Drug: ropeginterferon alfa- 2b, Drug: Azacitidine (AZA)Treatment with Ropeginterferon and AZA |
| Dose Expansion, | Experimental | Drug: ropeginterferon alfa- 2b, Drug: Azacitidine (AZA) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (18 listed)
- Adult patients with MDS/AML without a known pathogenic TP53 mutation
- Patients with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
- Patient 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).
- Patient with known and untreated major depressive disorder
- Recent stroke or myocardial infarction (in previous 6 months)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ropeginterferon alfa- 2bGiven by iv | Drug | Dose Escalation, Dose Expansion, |
| Azacitidine (AZA)Given by IV | Drug | Dose Escalation, Dose Expansion, |
Primary outcome
Safety and adverse events (AEs).
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
Related trials for Ropeginterferon
Not yet recruitingPhase 2
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RecruitingPhase 1
Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis
Myelofibrosis
RecruitingPhase 2
Phase II Study Assessing the Safety and Efficacy of Dasatinib in Combination With Ropeginterferon in Patients With Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase
Chronic Myeloid Leukemia
Active, not recruitingPhase 3
P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk
Primary Myelofibrosis, Myeloproliferative Neoplasm
RecruitingPhase 1
Ropeginterferon for High Risk JAK2 Clonal Hematopoiesis
JAK2 Mutation
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jayastu Senapati, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ropeginterferon alfa- 2b and Azacitidine (AZA) in people with Ropeginterferon and Alfa-2b, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jayastu Senapati, MD (713-745-9200, jsenapati@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs). (time frame: Through study completion; an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.