Reduced Intensity Conditioning for MDS

NCT07085156 | Not Yet Recruiting Phase 2

• 1 other identifier: RJ-BMT-012
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

Allogeneic hematopoietic cell transplantation (allo-HCT) is the only potential cure for Myelodysplastic syndrome (MDS). Currently, the conditioning regimen for MDS allogeneic transplantation still follows AML, and there are fewer explorations in this field. In a large-scale retrospective analysis with 532 MDS undergoing allo-HCT, reduced intensity conditioning is resulted in improved overall survival (OS), reduced non-relapse mortality (NRM) , while sparing relapse. Therefore, the investigators conduct a prospective, single-arm, multicentre study to evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan reduced-intensity conditioning in MDS patients.

Conditions

MDS

Keywords

MDS; Reduced intensity conditioning

Outcome Measures

Primary Outcomes (1)

• 2-year relapse-free survival after transplantation

  relapse

  through study completion, an average of 2 year

Secondary Outcomes (2)

• 2-year post-transplant survival

  through study completion, an average of 2 year

• Cumulative recurrence rate at 2 years post-transplant

  through study completion, an average of 2 year

Study Arms (1)

FBM arm

EXPERIMENTAL

Adult MDS patients eligible for allo-HCT will receive Fludarabine 30mg/m\^2 d-6 to -2; melphalan 70mg/m\^2 d-6; busulfan 3.2mg/kg d-4 to -3 as conditioning regimen.

Drug: Fludarabine; melphalan; busulfan

Interventions

Fludarabine; melphalan; busulfan DRUG

The conditioning regimen includes fludarabine, busulfan, and melphalan, with a transplant conditioning intensity of 3.0. The RIC is used in the allo-HCT in the MDS patients.

FBM arm

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 14-70 (including boundaries) and gender;
• Diagnosis of MDS is confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
• IPSS-R intermediate-risk or higher-risk or IPSS-M intermediate-risk or higher-risk MDS; transfusion-dependent MDS;
• ECOG score≤ 3 points;
• Have appropriate organ function, and laboratory results within 7 days prior to the start of trial 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 syndrome; patients with Gilbert-Meulengracht 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;
• Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min;
• Coagulation function: International Normalised Ratio (INR) ≤ 1.5 x ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5 x ULN;
• Left ventricular ejection fraction (LVEF) ≥50%;
• Voluntarily signing the informed consent, understanding and complying with the requirements of the study, good compliance, and cooperating with the follow-up visits.

You may not qualify if:

• Allergies or contraindications to any of the drugs in the protocol;
• Currently 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;
• Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
• Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
• Patients with neurological or psychiatric disorders;
• The patient is pregnant or breastfeeding;
• Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form.
• Other conditions that, in the opinion of the investigator, make the patient otherwise unsuitable for participation in this study;

Sponsors & Collaborators

• Ruijin Hospital: lead
• Wuhan TongJi Hospital: collaborator
• Wuhan Children's Hospital (Wuhan Maternal and Child Healthcare Hospital), Tongji Medical College, Huazhong University of Science and Technology: collaborator
• Zhejiang University: collaborator

Study Sites (1)

Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Interventions

fludarabine; Melphalan; Busulfan

Intervention Hierarchy (Ancestors)

Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Butylene Glycols; Glycols; Alcohols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Central Study Contacts

Xiaoxia HU, Doctor

CONTACT

02164370045; hu_xiaoxia@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 6, 2025
• First Posted: July 25, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): May 1, 2028
• Last Updated: July 25, 2025

Record last verified: 2025-07

Locations

CN (1)