The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

NCT ID: NCT06069180

Last Updated: 2023-11-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-11-15

Study Completion Date

2025-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Severe Aplastic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Busulfan

Intervention Type DRUG

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

Control group

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

Group Type OTHER

Flu/Cy/ATG or Cy/ATG

Intervention Type DRUG

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

Interventions

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Busulfan

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

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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

1. Age \< 18 years old
2. 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
Minimum Eligible Age

1 Year

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Peking University People's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Xiao-Jun Huang

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Xiao-Jun Huang

Role: PRINCIPAL_INVESTIGATOR

Peking University People's Hospital

Locations

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Deparment of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Zheng-Li Xu, M.D.

Role: CONTACT

+8613501338951

Facility Contacts

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Xiaojun Huang, doctor

Role: primary

8601088326666

Other Identifiers

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2023PHB232-001

Identifier Type: -

Identifier Source: org_study_id

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