RIC Regimen for Low- and Intermediate-risk MDS Receiving Haplo-HSCT

NCT03412266 | Unknown Phase 2

• 1 other identifier: RIC Haplo-MDS
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This study aimed to evaluate the efficacy of reduced intensity conditioning (RIC) regimen in low- and intermediate-risk myelodysplastic syndrome (MDS) patients who receive haploidentical hematopoietic stem cell transplantation (haplo-HSCT). Haplo-HSCT is an effective treatment option for MDS patients who did not have identical sibling donor (ISD) or unrelated donor (URD). However, post-transplant transplant-related mortality (TRM) is one of the major causes for transplant failure in MDS patients, and the risk of TRM for haplo-HSCT recipients was higher than that of ISD recipients. RIC regimen can decrease the risk of TRM for haplo-HSCT recipients; however, the risk for relapse may increase in these patients. Thus, RIC regimen may be more appropriate for low- and intermediate-risk MDS patients receiving haplo-HSCT. The study hypothesis: Using RIC haplo-HSCT regimen in patients with low- and risk MDS can reduce TRM and improve survival.

Conditions

Myelodysplastic Syndromes

Keywords

RIC regimen; MDS; haplo-HSCT; survival

Outcome Measures

Primary Outcomes (1)

• Transplant-related mortality

  Death without disease progression or relapse

  Participants will be followed for an expected average of 1 years

Study Arms (1)

RIC regimen

OTHER

Low- and intermediate MDS patients without identical sibling donor or unrelated donor would receive RIC haplo-HSCT. RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France).

Drug: Cytarabine

Interventions

Cytarabine DRUG

RIC preconditioning regimen consisted of cytarabine (2 g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m-2/day, days -6 to -2), semustine (250 mg/m-2, day -3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France).

Also known as: busulfan, Cyclophosphamide, Fludarabine, Semustine, antithymocyte globulin

RIC regimen

Eligibility Criteria

• Age: 1 Year - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation

You may not qualify if:

• Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University Institute of Hematology,Beijing

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

Cytarabine; Busulfan; Cyclophosphamide; fludarabine; Semustine; Antilymphocyte Serum

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Lomustine; Nitrosourea Compounds; Urea; Amides; Nitroso Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Study Officials

• Xiao-Jun Huang

  Peking University Institute of Hematology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiao-Dong Mo

CONTACT

8610-8832-6001; mxd453@163.com

Xiao-Dong Mo

CONTACT

8610-8832-4577

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: Director

Study Record Dates

• First Submitted: January 21, 2018
• First Posted: January 26, 2018
• Study Start: February 1, 2018
• Primary Completion: March 1, 2023
• Study Completion: March 1, 2023
• Last Updated: April 20, 2022

Record last verified: 2022-04

Locations

CN (1)