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NCT Number: NCT07338422

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

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.

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Key information

Age range

14 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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).

tranplantation

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 transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).

Primary outcomes

  1. Failure Free Survival, FFS

    Time frame: 2 year

    survival with complete response whereas death, graft failure and relapse are considered treatment failures

Secondary outcomes

  1. Overall Survival, OS

    Time frame: 2 year

  2. Treatment Related Mortality, TRM

    Time frame: 2 year

    Transplantation-related mortality (TRM) was defined as death without graft failure

  3. hematology remission

    Time frame: 2 year

    hematology recovery, including WBC, Hb and PLT

  4. Infection incidence

    Time frame: 2 year

  5. GVHD incidence

    Time frame: 2 year

  6. QoL evaluation

    Time frame: 2 year

    life quality assessment

Study contacts

Contact information is provided by the study sponsor or research team.

Xiaoyu Zhang

CONTACT

[email protected]

022-23608569

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Official study title

Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Jan 13, 2026
Registry last updated
Jan 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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