Cyclophosphamide Reduced-dose, Immunosuppressive Therapy, Severe Aplastic Anemia
Conditions
Brief 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.
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.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall response rate | Within 3 months | Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Superior response rate | With in 6 months | Percentage of patients with superior response, including CR, VGPR and GPR. |
| Overall response rate | With in 6 months | Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR). |
| Incidence of the adverse event | Within 6 months | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event |
| Time to achieve robust superior response | Within 6 months | — |
| First time to response | Within 6 months | — |
Countries
China
Contacts
Primary ContactHong Pan, MD
Backup ContactJingyu Zhao, MPH
Outcome results
None listed