Avatrombopag Combined With IST as First-line Treatment for SAA

Institute of Hematology & Blood Diseases Hospital, China

Status and phase: Enrolling; Phase 2

Conditions: Severe Aplastic Anemia

Treatments: Drug: avatrombopag

Study type: Interventional

Funder types: Other

Identifiers

NCT05720234

AVA&IST-001

Details and patient eligibility

About

This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).

Full description

This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled.

Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.

Enrollment: 53 estimated patients

Sex: All

Ages: 12 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Patients with newly diagnosed severe aplastic anemia.
2. Men and women aged between 12 and 60.
3. Subjects must complete all screening assessments as outlined in the test protocol.
4. Able to swallow or administer orally.
5. Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form.

Exclusion criteria

1. Congenital bone marrow failure (eg. Fanconi anemia).
2. Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) .
3. ATG or middle/high-dose cyclophosphamide was used in the past.
4. Previous treatment with cyclosporine or tacrolimus > 6 months.
5. The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month.
6. Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections).
7. AIDS patients.
8. Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures.
9. Patients with malignant tumors who are not suitable for ATG treatment.
10. A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months.
11. Those who are assessed as unsuitable for inclusion by the investigator.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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53 participants in 1 patient group

Treatment group

Experimental group

Description:

53 subjects will be enrolled with the indicated treatment dose of avatrombopag.

Treatment:

Drug: avatrombopag