Avatrombopag Combined With IST as First-line Treatment for SAA

Last updated: February 7, 2023
Sponsor: Institute of Hematology & Blood Diseases Hospital
Overall Status: Active - Recruiting

Phase

2

Condition

Aplastic Anemia

Anemia

Treatment

N/A

Clinical Study ID

NCT05720234
AVA&IST-001
  • Ages 12-60
  • All Genders

Study 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).

Eligibility Criteria

Inclusion

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 fullyunderstand the research procedure and purpose and sign the informed consent form. Ifthe patient's signature is not conducive to the treatment of the disease, thepatient's immediate family should sign the informed consent form.

Exclusion

Exclusion Criteria:

  1. Congenital bone marrow failure (eg. Fanconi anemia).
  2. Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detectionfound somatic cloning abnormalities; Simple -Y abnormality can be included in thisstudy;) .
  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, pulmonaryaspergillosis, viral infections).
  7. AIDS patients.
  8. Pregnant or breastfeeding, fertile but unwilling to take effective contraceptivemeasures.
  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.

Study Design

Total Participants: 53
Study Start date:
November 10, 2022
Estimated Completion Date:
November 30, 2024

Study 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.

Connect with a study center

  • Institute of Hematology & Blood Diseases Hospital

    Tianjin, Tianjin 300020
    China

    Active - Recruiting

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