HID-HSCT Versus IST as First-line Treatment for SAA | NCT07338422

Studies
HID-HSCT Versus IST as First-line Treatment for SAA

NCT07338422

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Status

Not yet recruiting

Last Update Posted

Jan 13, 2026Actual

Enrollment

116Estimated

Phase

Not Applicable

Conditions

Aplastic Anaemia

Interventions

IST (ATG + CsA+TPORA)
tranplantation

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2025

Overall Recruitment Status or Expanded Access Status

Not yet recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 14, 2026Estimated

Primary Completion Date

Sep 14, 2030Estimated

Completion Date

Sep 14, 2030Estimated

First Submitted Date

Sep 25, 2025

First Submission Date that Met QC Criteria

Jan 5, 2026

First Posted Date

Jan 13, 2026Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 5, 2026

Last Update Posted Date

Jan 13, 2026Actual

Description Module

Brief Summary

This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

116Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
HID-HSCT	Experimental		Procedure: tranplantation
IST	Other		Procedure: IST (ATG + CsA+TPORA)

Interventions

Name	Type	Description	Arm Group Labels	Other Names
IST (ATG + CsA+TPORA)	Procedure	The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation. Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA).

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Failure Free Survival, FFS	survival with complete response whereas death, graft failure and relapse are considered treatment failures	2 year

Secondary Outcomes

Measure	Description	Time Frame
Overall Survival, OS		2 year
Treatment Related Mortality, TRM	Transplantation-related mortality (TRM) was defined as death without graft failure	2 year

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

(1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.

(2) Age 14-70 years. (3) No HLA-matched related donor available. (4) HIV negative, HBV negative, HCV negative. (5) No absolute contraindications to transplantation or immunosuppressive therapy.

(6) Signed informed consent form must be obtained before study procedures begin; for subjects aged 18 years or older, informed consent shall be signed by the patient themselves or direct family members. Considering the patient's medical condition, if the patient's own signature is medically inadvisable, the informed consent shall be signed by a legal guardian or the patient's direct family member.

Exclusion Criteria:

Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;
Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;
Known severe allergy to ATG;
Previous allogeneic or autologous hematopoietic stem cell transplantation;
Previous solid organ transplantation;
Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;
Active HIV replication at enrollment, detectable HCV antibody positivity and HCV-RNA positivity within 90 days prior to enrollment, or HBsAg positivity; known seropositivity for HIV or active hepatitis C virus;
History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);
Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;
Inability or unwillingness to sign the consent form;
Other special circumstances deemed ineligible by the investigator.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

14 Years

Maximum Age

70 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D000080983	Bone Marrow Failure Disorders