Restoring p53 wild-type conformation in TP53-Y220C-mutant acute myeloid leukemia.
Bing Z Carter, Po Yee Mak, Edward Ayoub...
https://pubmed.ncbi.nlm.nih.gov/40608889Actively Recruiting
Phase 1
Age: 18Years +
All Genders
ID06616636
A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)
Led by M.D. Anderson Cancer Center · Updated on 2026-02-18
24
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
M
Memorial Sloan Kettering Cancer Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and possible benefits of a drug called rezatapopt combined with azacitidine in patients who have myeloid malignancies caused by a specific TP53Y220C gene mutation. These conditions include acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). This phase Ib clinical trial aims to assess how well patients tolerate the treatment and to explore its effects on disease progression and survival. Participants will take rezatapopt orally every day along with azacitidine given by intravenous infusion for seven days in each treatment cycle. Treatment may occur in either an inpatient or outpatient setting. The study also includes detailed assessments of genetic changes in the cancer cells, immune system responses, and how the drugs behave in the body. During the study, participants will be closely monitored for safety and side effects, with evaluations lasting about one year. Researchers will collect bone marrow samples and perform genetic sequencing to track changes in the cancer. Patients will have regular visits for treatment, testing, and assessments to understand the impact of the combination therapy on their condition.
CONDITIONS
Brief Title
A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Patients 18 years of age or older at the time of consent
- Willing and able to follow study visit schedule and protocol requirements
- Diagnosed with relapsed or primary refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)
- Patients with MDS must be classified as MDS-IB1 or IB2 according to WHO 2022 criteria
- Confirmed TP53Y220C mutation with variant allele frequency greater than 2%
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Adequate organ function including liver enzymes and kidney function within specified limits
- Women of childbearing potential must have negative pregnancy tests and agree to use effective contraception
- Men and women of childbearing potential must agree to use adequate contraception during the study and for a specified period after
- Able to understand and willing to sign informed consent
You will not qualify if you...
- Received chemotherapy, targeted therapy, immunotherapy, or investigational anticancer agents within 14 days or 5 half-lives before study drug
- Received radiotherapy within 14 days before study drug
- Diagnosis of acute promyelocytic leukemia
- Severe life-threatening leukemia complications such as uncontrolled bleeding or pneumonia with hypoxia
- Active, uncontrolled central nervous system leukemia involvement
- Active viral infections including HIV, hepatitis B or C
- Conditions limiting oral drug absorption such as dysphagia or gastroparesis
- Active uncontrolled systemic infections
- Unresolved toxicities from prior anticancer therapy greater than Grade 1 except alopecia and certain neuropathy
- Major surgery within 2 weeks prior to study treatment start
- Pregnant or lactating women
- History of allergic reactions to azacitidine, rezatapopt, or similar compounds
AI-Screening
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1
2
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Repeated cycles until disease progression or discontinuation
Participants receive daily oral rezatapopt and azacitidine by IV for 7 days every cycle, on an inpatient or outpatient basis.
Every cycle involves multiple visits for treatment administration
Trial Site Locations
Total: 1 location
1
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
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Research Team
C
Courtney DiNardo, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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