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

NCT ID: NCT06918834

Last Updated: 2025-09-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

236 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-07-24

Study Completion Date

2028-04-01

Brief Summary

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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

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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.

Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Immediate HSCT Group

Group Type EXPERIMENTAL

Immediate HSCT Group

Intervention Type OTHER

Patients undergo direct allogeneic HSCT.

Bridging Therapy Group

Group Type ACTIVE_COMPARATOR

Bridging Therapy Group

Intervention Type OTHER

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.

Interventions

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Immediate HSCT Group

Patients undergo direct allogeneic HSCT.

Intervention Type 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.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Age ≥18 years
2. High relapse risk MDS, defined by:

* IPSS-R score ≥3.5.
* IPSS-M stratification as intermediate-high, high, or very high risk.
3. Eligible for allogeneic HSCT (including matched or mismatched related/unrelated donor transplantations).
4. Karnofsky Performance Status (KPS) ≥60.
5. Signed informed consent.

Exclusion Criteria

1. 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.
2. History of prior allogeneic HSCT.
3. Any condition deemed unsuitable by the investigator.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Peking University People's Hospital

Beijin, Beijing Municipality, China

Site Status RECRUITING

Zhengzhou University First Affiliated Hospital

Zhengzhou, Henan, China

Site Status RECRUITING

The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army

Jinan, Shandong, China

Site Status RECRUITING

People's Liberation Army The General Hospital of Western Theater Command

Chengdu, Sichuan, China

Site Status RECRUITING

The Second Hospital of Hebei Medical University

Shijia Zhuang, , China

Site Status RECRUITING

Countries

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China

Central Contacts

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Jiang Erlie, doctor

Role: CONTACT

+86-15122538106

Xiao Zhijian, doctor

Role: CONTACT

+86-13821085716

Facility Contacts

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Hao Jiang

Role: primary

weijie cao

Role: primary

+86-15093360671

FANG ZHOU

Role: primary

+86-13969179221

hai yi

Role: primary

+86-17760428229

yan deng

Role: backup

+86-15828495714

Fuxu Wang

Role: primary

+86-13931100360

Other Identifiers

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IIT2025016

Identifier Type: -

Identifier Source: org_study_id

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