The Safety and Efficacy Study of Avatrombopag Switch in TPO-RA Refractory AA

  • STATUS
    Recruiting
  • End date
    Jan 1, 2026
  • participants needed
    39
  • sponsor
    Institute of Hematology & Blood Diseases Hospital
Updated on 7 October 2022

Summary

This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug

Description

This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug.Selection of study population Severe aplastic anemia patients with poor efficacy of IST combined with TPO-RA Patients should be judged for inclusion and exclusion criteria. Number of subjects: 35 effective cases, 39 patients should be included according to the dropout rate of 10%.

Details
Condition Refractory Aplastic Anemia
Treatment Avatrombopag
Clinical Study IdentifierNCT05518331
SponsorInstitute of Hematology & Blood Diseases Hospital
Last Modified on7 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Subjects eligible for inclusion in this study must meet
all of the
following criteria
Patients with confirmed TDNSAA/SAA/VSAA aplastic anemia who received standard IST therapy for at least 6 months, combined with Haitrombopag (15mg/d) or Eltrombopag (>50mg/d) for at least 3 Patients who have not obtained a hematological response (NR) for months and are not suitable or unwilling to undergo HSCT
Age > 14 years old, male or female
Subjects must complete all screening assessments listed in the trial protocol
ECOG score ≤ 2 points
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

Subjects meeting any of the following criteria were excluded from this
study
Patients with severe infectious diseases (uncured tuberculosis, pulmonary
aspergillosis, various bacterial and viral infections) and active bleeding that cannot
be controlled after standard treatment
Patients with AIDS, active viral hepatitis B, and hepatitis C RNA nucleic acid test
positive
Those who are pregnant or breastfeeding, have fertility but are unwilling to take
effective contraceptive measures
Congenital hematopoietic failure diseases (such as Fanconi anemia)
Patients with cytogenetic clonal changes (excluding germline mutations and acquired
chromosome clones of +8, 20q- and -y)
Combined with malignant tumor within 3 years
Combined with other systemic diseases that cannot be controlled
Significant abnormalities in cardiopulmonary function
Abnormal liver and kidney function: creatinine level > 1.5 times the upper limit of
normal, transaminase and bilirubin level > 2 times the upper limit of normal, and
those who cannot be enrolled in the group as judged by the clinician
Those who are considered unsuitable for enrollment by the investigator
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