The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

NCT06069180 | Unknown Phase 4

• 1 other identifier: 2023PHB232-001
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

Brief Summary

Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.

Conditions

Severe Aplastic Anemia

Keywords

HLA matched donor transplantation

Outcome Measures

Primary Outcomes (1)

• Failure free survival

  Failure free survival was defined as survival with a response to therapy.

  1 year post HSCT

Secondary Outcomes (7)

• The incidence of mixed chimerism

  1 year post HSCT

• Regimen related toxicity

  100 days post HSCT

• Myeloid and platelet engraftment

  100 days post HSCT

• The incidence of graft versus host disease

  100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD

• The incidence of CMV and EBV reactivation

  100 days post HSCT

Study Arms (2)

Busulfan included group

EXPERIMENTAL

The conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.

Drug: Busulfan

Control group

OTHER

The conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.

Drug: Flu/Cy/ATG or Cy/ATG

Interventions

Busulfan DRUG

Conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.

Busulfan included group

Flu/Cy/ATG or Cy/ATG DRUG

Conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.

Control group

Eligibility Criteria

• Age: 1 Year - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed as SAA/vSAA
• Indication for hematopoietic stem cell transplantation
• Available HLA matched sibling or unrelated donor
• No active infection
• No serious organ damage: liver and kidney function (ALT and AST \< 2.5 times normal value, normal renal function, no cardiac insufficiency)
• Signed informed consent
• High risk factors of mixed chimerism, at least one of the following
• Age \< 18 years old
• Ferritin level ≥2500ng/ml before transplantation

You may not qualify if:

• Age \> 50 years old
• ECOG≥3
• Active infections that were difficult to control
• Severe liver and kidney dysfunction
• Mental illness
• Not signing the informed consent
• pregnant or lactating women
• Any condition considered by the investigators to be unsuitable for enrollment

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Deparment of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Busulfan

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Xiao-Jun Huang

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zheng-Li Xu, M.D.

CONTACT

+8613501338951; xuzhengli0202@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: October 1, 2023
• First Posted: October 5, 2023
• Study Start: November 15, 2023
• Primary Completion: December 31, 2024
• Study Completion: December 31, 2025
• Last Updated: November 18, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)