Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia (hypo-CASH)

Institute of Hematology & Blood Diseases Hospital, China

Status and phase

Enrolling

Phase 2

Conditions

Immunosuppressive Therapy

Severe Aplastic Anemia

Cyclophosphamide Reduced-dose

Treatments

Drug: Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy

Study type

Interventional

Funder types

Other

Identifiers

NCT06695741

Hypo-CASH-2024

Details and patient eligibility

About

This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of anti-lymphocyte globulin plus herombopag in combination with the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.

Enrollment

75 estimated patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Subject has a diagnosis of naïve severe or very severe aplastic anemia
Male or female age ≥ 12 years
Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
ECOG performance status ≤2
Willing and able to comply with the requirements for this study and written informed consent.

Exclusion criteria

Previously received immunosuppressive therapy > 4 weeks
Previously treated with TPO-RA > 4 weeks
Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
Tested positive for HIV or syphilis
Presence of severe liver, kidney, or heart failure, or other life-threatening comorbidities.
History of radiotherapy and chemotherapy for malignant solid tumors in recent 5 years
Combined with other serious disorders
Pregnant or breast-feeding patients
Patients considered to be ineligible for the study by the investigator for reasons other than the above.