Status:
RECRUITING
Molecular Genetics Guide the Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
Lead Sponsor:
Ruijin Hospital
Conditions:
AML
MDS
Eligibility:
All Genders
14-75 years
Phase:
PHASE2
PHASE3
Brief Summary
This study is a prospective, multicenter, open-label umbrella clinical study planned to enroll 126 subjects with acute myeloid leukemia/myelodysplastic syndromes undergoing allogeneic hematopoietic st...
Detailed Description
This study is a prospective, multicenter, open-label, umbrella study designed to evaluate the efficacy and safety of mutation-based guided post-transplantation maintenance therapy in post-transplantat...
Eligibility Criteria
Inclusion
- 1\) Age 14-75 years and gender; (2) Initial diagnosis, pre-transplant myelogenetic second-generation sequencing data refinement; 3)ARM1: TP53 mutation (VAF ≥ 2%, excluding germline mutations), and/or AML-MR (SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR, STAG2) by second-generation sequencing of the initial bone marrow gene; ARM2 Initial bone marrow gene II sequencing showed FLT3-ITD mutation with or without NPM1 mutation, no TP53 mutation, and no AML/MR. ARM3 Initial bone marrow gene II sequencing showed KIT mutations (loci: D816, N822, exon 8, VAF ≥2%, excluding germline mutations); no TP53 mutations, AML/MR, FLT3-ITD; ARM4 MDS in the intermediate/high/very high risk group as assessed by IPSS-M or AML in the intermediate/high risk group as assessed by ELN2022 without ARM1, 2, or 3 related mutations/fusions; 4) Subjects receive their first allogeneic hematopoietic stem cell transplantation within 30-60 days and STR-PCR shows complete donor chimerism; 5) Evaluation of the primary disease prior to maintenance therapy as complete remission and negative bone marrow flow MRD; 6) No active infection and no acute graft-versus-host disease requiring systemic treatment 7) Have appropriate organ function and laboratory results within 7 days prior to the start of treatment need to meet the following criteria:
- Aspartate aminotransferase (AST) ≤ 3 times ULN (upper limit of normal, ULN);
- Alanine aminotransferase (ALT) ≤ 3x ULN;
- Total serum bilirubin ≤ 1.5 times the upper limit of normal ULN unless the patient has documented Gilbert's syndrome; patients with Gilbert's syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included;
- Hemoglobin ≥ 70 g/L (had not received a red blood cell transfusion within 1 week prior to administration); Absolute neutrophil count (ANC) ≥ 0.8 x 10\^9/L (had not received long-acting colony-stimulating factor (LACSF) within 1 week prior to administration and short-acting colony-stimulating factor (SACSF) within 3 days prior to administration); and Platelet count ≥ 20 x 10\^9/L (1 week prior to administration) have not received a platelet transfusion);
- Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min;
- Coagulation function: International Normalized Ratio (INR) ≤1.5×ULN, Activated Partial Thromboplastin Time (APTT) ≤1.5×ULN;
- Left ventricular ejection fraction (LVEF) ≥45%; 8) Women of childbearing potential must have a negative serum pregnancy study within 7 days prior to the first dose. Female subjects of childbearing potential and male subjects whose partner is a woman of childbearing potential must agree to use a highly effective method of contraception from the time of signing the informed consent form until 180 days after the last dose of study drug is given (9) Voluntarily signing the informed consent, understanding and complying with the requirements of the study, good compliance, and cooperating with the follow-up visits.
Exclusion
- Patients were not enrolled if they met any of the following criteria:
- Bone marrow or peripheral blood MRD changes from negative to positive;
- Presence of acute or chronic GVHD, active infection requiring systemic immunosuppressive therapy prior to maintenance therapy;
- Have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- Other serious medical conditions that may limit the patient's participation in this trial (e.g., uncontrolled diabetes, active autoimmune disease, etc.);
- Known human immunodeficiency virus (HIV) infection, or chronic infection with hepatitis B virus (HBsAg-positive) or hepatitis C virus (anti-HCV-positive) that is uncontrolled by drugs;
- Patients with neurological or psychiatric disorders;
- Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form;
- The researcher does not consider it appropriate to participate in this study.
Key Trial Info
Start Date :
June 10 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 31 2030
Estimated Enrollment :
126 Patients enrolled
Trial Details
Trial ID
NCT06972641
Start Date
June 10 2025
End Date
March 31 2030
Last Update
May 18 2025
Active Locations (1)
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1
Ruijin Hospital
Shanghai, Shanghai Municipality, China, 200025