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

NCT07586735 | Recruiting DMC

• 1 other identifier: PUPH20260508
• Study Type: observational
• Target: 64
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Severe Aplastic Anemia; Aplastic Anaemia; Hematopoietic Cell Transplant

Outcome Measures

Primary Outcomes (1)

• Faliure free survival (FFS)

  survival with a response to therapy after HSCT. Death, graft faliue and relapse were considered as treatment failure.

  From enrollment to 2 years post-HSCT

Secondary Outcomes (7)

• The probability of Overall survival

  From enrollment to 2 years post-HSCT

• Myeloid and platelet engraftment

  100 days post HSCT

• The incidence of mixed chimerism

  1 year post HSCT

• The incidence of graft versus host disease(GVHD)

  100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD

• Regimen related toxicity

  100 days post HSCT

Study Arms (4)

Peking unviversity Peoples' Hospital

Drug: BFCA conditiong regimen

The First Affiliated Hospital of Soochow University

Drug: BFCA conditiong regimen

Guangzhou First People's Hospital,

Drug: BFCA conditiong regimen

The First Affiliated Hospital of USTC

Drug: BFCA conditiong regimen

Interventions

BFCA conditiong regimen DRUG

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).

Guangzhou First People's Hospital,; Peking unviversity Peoples' Hospital; The First Affiliated Hospital of Soochow University; The First Affiliated Hospital of USTC

Eligibility Criteria

• Age: 40 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients aged 40-60 years old who are diagnosed with severe aplastic anemia according to NCCN guidelines and who will receive haploidentical hematopoietic stem cell transplantation at the centers will be recruited.

You may qualify if:

• 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.

You may not qualify if:

• 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:

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking Universtiy Peoples' Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Central Study Contacts

Tingting Han

CONTACT

8601088326666; htt1984.love@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 8, 2026
• First Posted: May 14, 2026
• Study Start: June 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 30, 2029
• Last Updated: July 6, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)