NCT06972641

Molecular Genetics Guide the Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation

A Prospective, Multicenter Umbrella Study Based on Molecular Genetics to Guide the Efficacy and Safety of Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia/Myelodysplastic Syndrome

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 126 (estimated)
• Sponsor: Ruijin Hospital · Academic / Other
• Sex: All
• Age: 14 Years – 75 Years
• Healthy volunteers: Not accepted

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 stem cell transplantation. Subjects eligible for enrollment were grouped based on the results of the initial myeloid genomic second-generation sequencing, and were given different regimens of maintenance therapy, with the aim of evaluating the efficacy and safety of the maintenance regimen.

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-transplantation patients . The study will include allogeneic hematopoietic stem cell transplantation patients who meet the enrollment criteria, applying myeloid gene second-generation sequencing data to classify the patients into four subgroups and set the corresponding treatment arms according to the underlying molecular characteristics of the different subgroups, which will be treated with cerinisol in combination with vinaiquelat, FLT3 inhibitors, avastinib, and decitabine, respectively. Maintenance therapy was initiated 60-90 days post-transplant and continued for a total of 2 years. The study is divided into a screening period (screening is initiated 30-60 days post-transplant) and a treatment follow-up period (after screening, subjects are scheduled for treatment. Maintenance therapy is initiated 60-90 days post-transplantation with follow-up monitoring). Until one of the following occurs: disease progression, toxicity intolerance, or other reasons as specified in the protocol. Subjects who end treatment enter the follow-up period for safety or survival follow-up.

Conditions

• AML
• MDS

Interventions

Type	Name	Description
DRUG	Decitabine	Decitabine 3.5 mg/m2 1-5d for 28 days for 6 cycles of dosing
DRUG	Sorafenib (BAY-43-9006)，giritinib	Sorafenib 0.2g 2/day or giritinib 40mg 2/day orally for 2 years
DRUG	Avastinib	100 mg 1/day
DRUG	Venetoclax;Selenisol	Vinaclat: 50-100mg 1/day orally, d1-14 1/day per month (adjust drug dosage based on blood levels), 28-day cycle; Selenisol: 20mg qw orally (weeks 1 and 2), 28-day cycle If the above treatment is not tolerated, adjust the regimen to decitabine 3.5 mg/m2 1-5 d; 28 days as a cycle of 6 cycles of medication; Vinaclat: 50-100 mg 1/day orally, d1-14 1/day per month orally (adjust the dose of the drug according to the blood concentration), 28 days as a cycle of medication

Timeline

• Start date: 2025-06-10
• Primary completion: 2030-03-31
• Completion: 2030-03-31
• First posted: 2025-05-15
• Last updated: 2025-05-18

Locations

1 site across 1 country: China