Evaluate the Efficacy and Safety of Allogeneic Haematopoietic Stem Cell Transplantation With FBM Conditioning for MDS

NCT06247917 | Unknown Phase 2

• 1 other identifier: RJ-2023-01
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 1

Brief Summary

To assess the safety and efficacy of FLU-BU-MEL as a conditioning regimen for allogeneic hematopoietic stem cell transplantation in patients with untreated MDS-EB or IPSS-R that is intermediate-risk (\>3.5 points), high-risk, or very high-risk. The investigators conducted this clinical trial.There will be three phases to this trial: screening, therapy, and follow-up. A) Screening phase: Qualified patients are screened for trial participation by a medical history, physical examination, laboratory testing, and disease evaluation after providing their informed consent. B) Treatment duration: patients receive allogeneic hematopoietic stem cell transplantation prepped with Flu-Bu-Mel in accordance with the protocol. C) Follow-up period: patients were checked on at the scheduled time to assess safety and efficacy. HSCT conditioning regiment: Flu 30 mg/m2/d d-6 days to d-2 9 (intravenously over two hours), : MEL 50 mg/m2/d intravenously, d-3 to d-2; BU 0.8 mg/kg/q6h d-6 to d-5 (intravenously, over 2 hours per drip). Fludarabine and melphalan do not require a dose adjustment based on body weight; however, if body mass index BMI\> 25, ideal body weight (IBW) should be calculated (as BMI=25), and then determine Busulfan dosage based on corrected body weight (AIBW25). AIBW25=IBW+25% x (actual body weight - IBW)

Conditions

Myelodysplastic Syndrome With Excess Blasts-1; Myelodysplastic Syndrome With Excess Blasts-2; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• cumulative incidence of relapse

  2 years cumulative incidence of relapse

  2 years

Secondary Outcomes (5)

• Disease free survival

  2 years

• non relapse mortality

  100 days

• non relapse mortality

  2 years

• cumulative incidence of acute graft-versus-host disease

  180 days

• cumulative incidence of chronic graft-versus-host disease

  2 years

Study Arms (1)

FBM group

EXPERIMENTAL

undergoing FBM regiment

Drug: Fludarabine, Busulfan and Melphalan

Interventions

Fludarabine, Busulfan and Melphalan DRUG

Use Fludarabine, Busulfan and Melphalan as myeloablative conditioning regimen for untreated MDS-EB patients.

FBM group

Eligibility Criteria

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

You may qualify if:

• Diagnosis: Myelodysplastic Syndromes (IB1/IB2)
• Patients did not receive any anti-tumour therapy (including chemotherapy, immunotherapy (lenalidomide, etc.), targeted therapy, hormone therapy, etc.).

You may not qualify if:

• CMML with 5q deletion or carrying PDGFRB fusion gene;
• secondary MDS;

Sponsors & Collaborators

• Ruijin Hospital: lead

Study Sites (1)

Ruijin Hospital, Shanghai JiaoTong University School of Medicine

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

fludarabine; Busulfan; Melphalan

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

• Luxiang Wang

  Ruijin Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoxia Hu

CONTACT

+86 021 64370045; 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: SPONSOR

Study Record Dates

• First Submitted: December 26, 2023
• First Posted: February 8, 2024
• Study Start: March 3, 2024
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2024
• Last Updated: March 21, 2024

Record last verified: 2023-12

Locations

CN (1)