RecruitingPhase 2NCT06695741

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy as Front-line Therapy in Patients With Severe Aplastic Anemia

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Enrollment

75 participants

Start Date

Nov 1, 2024

Study Type

INTERVENTIONAL

Conditions

Severe Aplastic Anemia Cyclophosphamide Reduced-dose

Summary

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.

Eligibility

Min Age: 12 Years

Inclusion Criteria5

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 Criteria10

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.

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Interventions

DRUGReduced dose of cyclophosphamide combined with standard immunosuppressive therapy

Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. Cyclophosphamide (20mg/kg) will be administered on days 15-16.