RecruitingPhase 2NCT06532084

Sorafenib Relapase Prophylaxis After HCT With PTBCy Regimen

Randomized Trial of Sorafenib Prophylaxis After Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplantation Bendamustine and Cyclophosphamide


Sponsor

St. Petersburg State Pavlov Medical University

Enrollment

88 participants

Start Date

May 22, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

This is a single-center randomized open-label phase II clinical trial to compare relapse prophylaxis with sorafenib and observation after graft-versus-host disease prophylaxis with post-transplantation bendamustine and cyclophosphamide in high-risk myeloid malignancies. This is an intention to treat study, where randomization is performed at first documentation of CR after engraftment.


Eligibility

Min Age: 18 YearsMax Age: 75 Years

Inclusion Criteria13

  • Patients must undergo allogeneic hematopoietic stem cell transplantation with post-transplantation bendamustine AND cyclophosphamide from any donor.
  • Patients must have high-risk myeloid malignancy as an indication for transplantation defined as:
  • acute myeloid leukemia not in hematological remission prior to transplantation,
  • ≥ 3 remission of acute myeloid leukemia,
  • any myeloid malignancy with bi-allelic tp53 mutation,
  • any myeloid malignancy with complex karyotype,
  • therapy-related myeloid malignancy not in MRD-negative response
  • myelodysplastic syndrome with very high IPSS-R risk
  • any myeloid malignancy with monosomal or t(3;3) karyotype,
  • any myeloid malignancy with ASXL1, bi-allelic tp53 or RUNX1 mutation, EVI1 overexpression
  • MDS/NPM unclassified not in hematologic remission.
  • Documented hematological remission in the bone marrow at the time of inclusion post-engraftment, measurable residual disease is allowed
  • First 100 days after allogeneic hematopoietic stem cell transplantation

Exclusion Criteria15

  • successfully treated relapse between transplantation and enrollment
  • use of any other planned method for prophylaxis of relapse besides sorafenib
  • donor lymphocyte infusion prior to randomization
  • Second malignancy not in complete remission within 6 months prior to randomization
  • Moderate or severe cardiac disease: ejection fraction \<50%, unstable angina, stable angina NYHA class III or IV, chronic heart failure NYHA class III or IV, Lawn grade V arrhythmia, myocardial infarction within 3 months before inclusion
  • Stroke within 3 months of inclusion, unless related to the underlying malignancy
  • Severe decrease in pulmonary function: FEV1 \<50% or DLCO\<50% of predicted or respiratory distress or need for oxygen support;
  • Severe organ dysfunction: AST or ALT \>10 upper normal limits, bilirubin \>2 upper normal limits, creatinine \>2 upper normal limits
  • Creatinine clearance \< 30 mL/min
  • Uncontrolled bacterial or fungal infection at the time of enrollment defined by CRP\> 70 mg/L
  • Requirement for vasopressor support at the time of enrollment
  • Requirement for positive-pressure oxygen at the time of enrollment
  • Karnofsky index \<30%
  • Pregnancy
  • Somatic or psychiatric disorder making the patient unable to sign informed consent

Interventions

DRUGSorafenib

Sorafenib 200 mg bid for 168 days starting in the time fram between engraftment with neutrophils \> 1.0 x 10\^9/L, white blood cells\> 1.5 x 10\^9/L, platelets\> 1.5 x 10\^9/L and day+100 after allogeneic hematopoietic cell trnaplantation


Locations(1)

RM Gorbacheva Research Institute

Saint Petersburg, Russia

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NCT06532084


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