Actively Recruiting

Phase 2
Age: 18Years - 70Years
All Genders
ID05841771

A Phase 2 Study Evaluating Safety and Efficacy of Maintenance Therapy With Hypomethylating Agent and Venetoclax After Allogeneic Stem Cell Transplantation in Patients With High-risk Myeloid Malignancies

Led by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2024-08-09

78

Participants Needed

1

Research Sites

52 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of maintenance therapy using hypomethylating agents combined with Venetoclax to improve leukemia-free survival in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after allogeneic hematopoietic stem cell transplantation. This phase 2, single-arm study focuses on patients aged 18 to 70 years with specific high-risk factors for their conditions. Participants will receive hypomethylating agents, either azacitidine (32 mg/m2 daily subcutaneously) or decitabine (5 mg/m2 daily intravenously) for 5 days, plus Venetoclax (400 mg orally daily for 7 days). This maintenance treatment starts between 60 and 120 days after transplantation and is repeated every 28 days for up to 10 cycles within the first year post-transplant. Dosage adjustments for Venetoclax are made if patients are taking certain CYP450 inhibitors. During the study, participants will be monitored for leukemia-free survival, cumulative incidence of relapse, overall survival, and treatment-related toxicity up to one year after transplantation. Assessments include regular blood counts and evaluations of disease status to track response and safety throughout the maintenance period and follow-up.

CONDITIONS

Brief Title

Hypomethylating Agent and Venetoclax After Allo-HSCT in Patients With High-risk Myeloid Malignancies.

Who Can Participate

Age: 18Years - 70Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients aged 18 to 70 years
  • Diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)
  • Have received allogeneic hematopoietic cell transplantation
  • AML patients must have high-risk factors such as adverse cytogenetics, need for more than 2 induction chemotherapy courses, extramedullary disease, CR2 or beyond, or measurable residual disease at transplantation
  • MDS patients must have high-risk factors such as high or very high IPSS-R scores, TP53 mutation, or measurable residual disease at transplantation
  • Blood counts: ANC ≥ 1.0 × 10e9/L, hemoglobin ≥ 80 g/L, platelets ≥ 50 × 10e9/L
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Not Eligible

You will not qualify if you...

  • Concurrent use of targeted drugs
  • Resistance to Venetoclax before transplantation
  • Allergy to decitabine, azacitidine, or venetoclax
  • Active grade II or higher acute graft-versus-host disease (GVHD)
  • Active moderate or severe chronic GVHD
  • Disease recurrence or graft rejection (abnormal myeloid cells >0.01%, donor cells <90%, or extramedullary malignancy)
  • Blood counts: ANC < 1.0 × 10e9/L or platelets < 50 × 10e9/L
  • Severe organ dysfunction (elevated liver enzymes or bilirubin, reduced kidney function)
  • Active uncontrolled systemic fungal, bacterial, or viral infection
  • Pregnant or lactating women
  • Other severe complications judged unsuitable by researchers

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Treatment

Duration - From Day 60 to up to 1 year post-transplant, repeated every 28 days

Participants receive maintenance therapy with hypomethylating agents (azacitidine or decitabine) for 5 days and venetoclax for 7 days, starting from 60 days after allogeneic stem cell transplantation. This cycle repeats every 28 days for up to 1 year post-transplant.

Up to 10 treatment cycles, each with 1 visit for drug administration

Trial Site Locations

Total: 1 location

1

Shanghai Jiao Tong University School of Medicine Affilated Shanghai General Hospital

Shanghai, Shanghai Municipality, China, 200080

Actively Recruiting

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Research Team

X

Xueying Ding, Ph.D.

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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