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Recruiting NCT07586735

A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA

— Observational Severe Aplastic Anemia Aplastic Anaemia Hematopoietic Cell Transplant

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: BFCA conditiong regimen.
Who it may be relevant to
Registry conditions: Severe Aplastic Anemia, Aplastic Anaemia, Hematopoietic Cell Transplant. Basic parameters: 40 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial

Overview

The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.

Interventions

  • Drug BFCA conditiong regimen
    busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).

Primary outcome measures

  • Faliure free survival (FFS) [Time frame: From enrollment to 2 years post-HSCT]
Secondary outcome measures (7)
  • The probability of Overall survival [Time frame: From enrollment to 2 years post-HSCT]
  • Myeloid and platelet engraftment [Time frame: 100 days post HSCT]
  • The incidence of mixed chimerism [Time frame: 1 year post HSCT]
  • The incidence of graft versus host disease(GVHD) [Time frame: 100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD]
  • Regimen related toxicity [Time frame: 100 days post HSCT]
  • The incidence of Cytomegalovirus(CMV) and Epstein-Barr virus(EBV) reactivation [Time frame: 180 days post HSCT]
  • The incidence of Transplantation related mortality [Time frame: 2 years post HSCT]

Eligibility criteria

Inclusion criteria

  • Severe aplastic anemia;
  • Aged 40-60 years old;
  • Weight 45Kg-100Kg;
  • Eastern Cooperative Oncology Group (ECOG) score ≤3;
  • No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
  • No severe infection;
  • Subjects voluntarily participated in this clinical trial and signed the informed consent.

Exclusion criteria

  • With other hematologic diseases;
  • Expected survival of less than 1 month;
  • Previous autologous or allogeneic hematopoietic stem cell transplantation;
  • Pregnant patients;
  • Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
  • Other conditions that the investigator determines to be inappropriate for enrollment.
  • Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
  • A history of symptomatic herpes zoster infection within 12 weeks prior to screening
  • Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
  • Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB
  • Exposure to a live vaccine within 12 weeks prior to enrollment or expected to receive a live vaccine during the study
  • Clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled
  • Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure
  • A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data
  • Any of the following specific abnormalities on screening laboratory tests:
  • ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN
  • hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL)
  • eGFR <50 mL/min/1.73 m2

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

China · 1 center
  • Peking Universtiy Peoples' Hospital — Beijing

Identifiers

NCT: NCT07586735 · PUPH20260508

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗