Menu
Recruiting NCT06918834

Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients

Phase II Interventional Myelodysplastic Syndromes

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Immediate HSCT Group, Bridging Therapy Group.
Who it may be relevant to
Registry conditions: Myelodysplastic Syndromes. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients: A Multicenter, Randomized Controlled, Open-Label, Phase 2 Clinical Trial

Overview

This study aims to evaluate whether immediate allogeneic hematopoietic stem cell transplantation (HSCT) is non-inferior to HSCT following bridging therapy in patients with higher-risk myelodysplastic syndrome (HR-MDS).

Detailed description

A total of 236 patients will be randomized in a 1:1 ratio into the immediate transplantation group (n=118) and the disease control group (n=118). The study will continue until at least 124 events occur.

Interventions

  • Other Immediate HSCT Group
    Patients undergo direct allogeneic HSCT.
  • Other Bridging Therapy Group
    Patients receive one to two cycles of bridging therapy before undergoing allogeneic HSCT. o Bridging Therapy Regimen: Hypomethylating agents (HMA) alone or HMA-based combination chemotherapy, e.g., azacitidine (AZA) 100 mg/day + venetoclax (VEN) 400 mg/day for 7 days. Targeted therapies (e.g., IDH1 inhibitors) will be used for eligible patients.

Primary outcome measures

  • 2-year Disease-Free Survival (DFS) post-HSCT [Time frame: 2-year]
Secondary outcome measures (6)
  • Cumulative Incidence of Allogeneic HSCT [Time frame: The proportion of patients who undergo HSCT at 4, 8, 16, and 24 weeks post-randomization.]
  • Complete Remission (CR) or CR Equivalent Rate from Randomization [Time frame: 2-year]
  • 2-year Overall Survival (OS) post-HSCT [Time frame: 2-year]
  • 2-year Leukemia-Free Survival (LFS) from Randomization [Time frame: 2-year]
  • 2-year Quality of Life (QoL) Assessment: 2-year Quality of Life (QoL) Assessment from Randomization [Time frame: 2-year]
  • Molecular Clearance Rate [Time frame: 2-year]

Eligibility criteria

Inclusion criteria

  • Age ≥18 years
  • High relapse risk MDS, defined by:
  • IPSS-R score ≥3.5.
  • IPSS-M stratification as intermediate-high, high, or very high risk.
  • Eligible for allogeneic HSCT (including matched or mismatched related/unrelated donor transplantations).
  • Karnofsky Performance Status (KPS) ≥60.
  • Signed informed consent.

Exclusion criteria

  • Severe organ dysfunction:
  • Left ventricular ejection fraction <50%.
  • Oxygen supplementation requirement.
  • Serum bilirubin >1.5x upper limit of normal (unless due to Gilbert syndrome) or AST/ALT >5x upper limit of normal.
  • Estimated glomerular filtration rate (eGFR) <50 mL/min.
  • History of prior allogeneic HSCT.
  • Any condition deemed unsuitable by the investigator.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Open label
Primary purpose
Treatment

Study locations

China · 5 centers
  • Peking University People's Hospital — Beijin
  • Zhengzhou University First Affiliated Hospital — Zhengzhou
  • The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation A — Jinan
  • People's Liberation Army The General Hospital of Western Theater Command — Chengdu
  • The Second Hospital of Hebei Medical University — Shijia Zhuang

Identifiers

NCT: NCT06918834 · IIT2025016

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗