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 moderate-dose cyclophosphamide for severe aplastic anemia.
Interventions
DRUGCyclophosphamide added to standard immunosuppressive therapy with herombopag
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. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.
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
* Male or female age ≥ 12 years * Subject has a diagnosis of naïve severe or very severe aplastic anemia * ECOG performance status ≤2 * Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation. * 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 either herombopag or cyclophosphamide. * Have an allergy to ALG * Uncontrolled systemic fungal, bacterial, or viral infection * Poorly controlled hypertension (≥140/90mmHg) or diabetes (a fasting plasma glucose concentration ≥7.0mmol/L or a random venous plasma glucose concentration ≥11.1mmol/L) * Abnormal liver or kidney function: ALT or AST \>3 ULN, or serum creatinine (sCr)≥ 2.5 ULN. * History of radiotherapy and chemotherapy for malignant solid tumors * 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 |
|---|---|---|
| Robust response | Within 3 months | Percentage of patients with robust response, including CR, near CR, VGPR and GPR. |
| All-cause mortality | Within 3 months | — |
| Time to achieve robust hematological response | Within 6 months | — |
| Incidence of the adverse event | Within 6 months | Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event |
Countries
China
Outcome results
None listed