Switching to Avatrombopag for Patients with Refractory Aplastic Anemia

Avatrombopag in TPO-RA Refractory Aplastic Anemia Patients Safety and Efficacy Study --Single-arm, Multicenter, Open, Phase Π Clinical Study

This study is testing if switching to a new medication called avatrombopag can help people with refractory aplastic anemia who haven't improved with other treatments feel better and have better blood counts.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 39 (estimated)
• Ages: 14 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Locations: 1 site (Tianjin, Tianjin)
• Trial ID: NCT05518331 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of switching patients with refractory aplastic anemia to avatrombopag after they have not responded to previous treatments. It involves patients who have been treated with immunosuppressive therapy and another thrombopoietin receptor agonist for at least three months without achieving a hematologic response. Participants will receive avatrombopag for a minimum of three months while continuing their original immunosuppressive therapy, followed by a three-month follow-up to assess their hematologic response and the safety of the treatment. The study aims to determine if this switch can improve patient outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are patients aged 14 and older with confirmed refractory aplastic anemia who have not responded to prior treatments.

Not a fit: Patients who are suitable or willing to undergo hematopoietic stem cell transplantation may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could provide a new treatment option for patients with refractory aplastic anemia who have not responded to existing therapies.

How similar studies have performed: While this approach is novel in the context of refractory aplastic anemia, similar studies with other thrombopoietin receptor agonists have shown promising results.

Eligibility criteria

Inclusion Criteria: Subjects eligible for inclusion in this study must meet all of the following criteria:

1. 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
2. Age \> 14 years old, male or female.
3. Subjects must complete all screening assessments listed in the trial protocol.
4. ECOG score ≤ 2 points.
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: Subjects meeting any of the following criteria were excluded from this study:

1. Patients with severe infectious diseases (uncured tuberculosis, pulmonary aspergillosis, various bacterial and viral infections) and active bleeding that cannot be controlled after standard treatment.
2. Patients with AIDS, active viral hepatitis B, and hepatitis C RNA nucleic acid test positive.
3. Those who are pregnant or breastfeeding, have fertility but are unwilling to take effective contraceptive measures.
4. Congenital hematopoietic failure diseases (such as Fanconi anemia).
5. Patients with cytogenetic clonal changes (excluding germline mutations and acquired chromosome clones of +8, 20q- and -y).
6. Combined with malignant tumor within 3 years.
7. Combined with other systemic diseases that cannot be controlled.
8. Significant abnormalities in cardiopulmonary function.
9. 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.
10. Those who are considered unsuitable for enrollment by the investigator.

Where this trial is running

Tianjin, Tianjin

• Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin, China (RECRUITING)

Study contacts

• Study coordinator: Fengkui Zhang, Doctor

Conditions: Refractory Aplastic Anemia, Avatrombopag, TPO-RA, refractory aplastic anemia