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The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

Sponsor
Peking University People's Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT06069180
Collaborator
(none)
160
Enrollment
2
Arms
26
Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 4

Study Design

Study Type:
Interventional
Anticipated Enrollment :
160 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
Anticipated Study Start Date :
Nov 1, 2023
Anticipated Primary Completion Date :
Dec 31, 2024
Anticipated Study Completion Date :
Dec 31, 2025

Arms and Interventions

Arm Intervention/Treatment
Experimental: Busulfan included group

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

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

Outcome Measures

Primary Outcome Measures

  1. Failure free survival [1 year post HSCT]

Secondary Outcome Measures

  1. The incidence of mixed chimerism [1 year post HSCT]

  2. Regimen related toxicity [100 days post HSCT]

  3. Myeloid and platelet engraftment [100 days post HSCT]

  4. The incidence of graft versus host disease [100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD]

  5. CMV and EBV reactivation [100 days post HSCT]

  6. Transplantation related mortality [1 year post HSCT]

  7. Overall survival [1 year post HSCT]

Eligibility Criteria

Criteria

Ages Eligible for Study:
1 Year to 50 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  1. Diagnosed as SAA/vSAA

  2. Indication for hematopoietic stem cell transplantation

  3. Available HLA matched sibling or unrelated donor

  4. No active infection

  5. No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency)

  6. Signed informed consent

  7. High risk factors of mixed chimerism, at least one of the following

  8. Age < 18 years old

  9. Ferritin level ≥2500ng/ml before transplantation

Exclusion Criteria:

  1. Age > 50 years old

  2. ECOG≥3

  3. Active infections that were difficult to control

  4. Severe liver and kidney dysfunction

  5. Mental illness

  6. Not signing the informed consent

  7. pregnant or lactating women

  8. Any condition considered by the investigators to be unsuitable for enrollment

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

  • Peking University People's Hospital

Investigators

  • Principal Investigator: Xiao-Jun Huang, Peking University People's Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Xiao-Jun Huang, Professor, Peking University People's Hospital
ClinicalTrials.gov Identifier:
NCT06069180
Other Study ID Numbers:
  • 2023PHB232-001
First Posted:
Oct 5, 2023
Last Update Posted:
Oct 5, 2023
Last Verified:
Oct 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Xiao-Jun Huang, Professor, Peking University People's Hospital
Conditioning regimen, HLA matched donor, severe aplastic anemia
Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Busulfan

Study Results

No Results Posted as of Oct 5, 2023