Actively Recruiting

Phase 2
Age: 18Years +
All Genders
ID06783790

Study of Avapritinib with Azacitidine and Venetoclax for Relapsed Acute Myeloid Leukemia After Stem Cell Transplant

Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-01-20

20

Participants Needed

1

Research Sites

149 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are exploring the safety and effectiveness of avapritinib combined with azacitidine and venetoclax for patients who have relapsed acute myeloid leukemia (AML) after undergoing allogeneic hematopoietic stem cell transplantation and have specific genetic mutations, including C-KIT mutation along with RUNX1::RUNX1T1 or CBFB::MYH11 fusion genes. This is a single-center, phase 2, prospective, single-arm study aiming to evaluate treatment outcomes in this specific patient group. The treatment consists of two main phases: induction therapy and consolidation therapy. During the induction phase, patients receive 1 to 2 cycles of avapritinib (100 mg orally once daily for days 1-14), azacitidine (35 mg/m2 intravenously for days 1-5), and venetoclax (100 mg orally once daily for days 1-14) in a 28-day cycle. Patients who achieve complete remission then continue to the consolidation phase, receiving avapritinib monotherapy (100 mg orally daily) for 4 cycles, each lasting 28 days. Participants will undergo evaluations at the end of each induction cycle to assess treatment response, with the primary outcome being the overall response rate two months after induction therapy. Throughout the study, patients' physical status, organ function, and safety are monitored. The study requires patient consent and willingness to complete all trial procedures and follow-up visits during the treatment periods.

CONDITIONS

Official Title

Avapritinib Combined With Azacitidine and Venetoclax in the Treatment of Relapsed AML After Allo-HSCT

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Recurrence of leukemia after allogeneic transplantation confirmed by >5% leukemia cells in peripheral blood or bone marrow, extramedullary infiltration, or molecular/cytogenetic relapse
  • Presence of C-KIT D816 or N822 mutations detected by bone marrow molecular biology
  • Detection of CBFB::MYH11 or RUNX1::RUNX1T1 fusion genes
  • Eastern Cancer Collaboration Group (ECOG) physical status score between 0 and 2
  • Liver, kidney, and cardiopulmonary functions within specified limits: creatinine 1.5 times upper normal limit, left ventricular ejection fraction 50%, blood oxygen saturation above 91%, total bilirubin 2 times ULN, ALT and AST 2.5 times ULN, myocardial enzymes less than twice the upper limit for age
  • Willingness to participate and ability to complete all trial procedures with signed informed consent
Not Eligible

You will not qualify if you...

  • Known allergy to KIT inhibitor drug analogues
  • Previous treatment with Midostaurin
  • Prior treatment with mutation-specific C-KIT inhibitors with disease progression during treatment
  • FLT3-ITD mutation in recurrent/refractory disease except for low gene ratio
  • Active infection with HIV, HBV, or HCV
  • Uncontrolled cardiovascular, cerebrovascular diseases, coagulation disorders, connective tissue diseases, serious infections, or other significant diseases
  • Uncontrolled active graft-versus-host disease (GVHD) according to NIH and Glucksberg standards
  • Central nervous system leukemia

AI-Screening

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Trial Site Locations

Total: 1 location

1

Institute of Hematology & Blood Diseases Hospital

Tianjin, China

Actively Recruiting

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Research Team

E

Erlie Jiang

Y

Yigeng Cao

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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