Clinical trial NCT05720234: Avatrombopag Combined With IST as First-line Treatment for SAA
Official title: The Efficacy and Safety Study of Avatrombopag Combined With IST as First-line Treatment for Severe Aplastic Anemia, A Single-arm, Phase II Clinical Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Severe Aplastic Anemia
Keywords: avatrombopag
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2023-02-09
Enrollment: 53
Sex: ALL
Minimum age (years): 12
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Treatment response
timeFrame: From the start of study treatment (Day1) to end of week 12.
Secondary outcomes:
measure: Treatment response
timeFrame: From the start of study treatment (Day1) to end of week 12.
measure: Treatment response
timeFrame: From the start of study treatment (Day1) to end of week 24.
measure: Supportive treatment
timeFrame: From the start study treatment (Day1) up to transfusion independence.
measure: Incidence of Treatment-Emergent Adverse Events by CTCAE
timeFrame: From the start study treatment (Day1) up to week 12.
measure: Dose-effect relationship
timeFrame: From the start study treatment(Day1) up to week 24.
measure: Change of CD34+ cell
timeFrame: From the start study treatment(Day1) up to end of week 12 and 24.
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Xin Zhao
role: PRINCIPAL_INVESTIGATOR
References: