This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells. The study will include about 26 participants from Japan, South Korea, and Taiwan whose disease has not responded to standard treatment, who cannot receive standard treatment, or whose disease has returned after previous treatment. The goal is to learn how well avatrombopag works and how safe it is for these patients.
Patients must be able to provide informed consent.
Age ≥18.
Diagnosis of aplastic anemia confirmed by peripheral blood and bone-marrow aspirate/biopsy.
Refractory to or relapsed after at least one course of immunosuppressive therapy including horse or rabbit anti-thymocyte globulin (ATG); or ineligible for ATG treatment and refractory to or relapsed after CyA.
Patients with bone marrow fibrosis MF-2 or MF-3 at screening, graded according to the WHO/European Consensus Reticulin Fibrosis Grading System (MF-0 to MF-3; Appendix E) documented on a bone marrow aspirate/biopsy obtained during screening.
Patients with >2% bone marrow blasts documented on a bone marrow aspirate/biopsy obtained during screening.
Patients with MDS-defining cytogenetic abnormalities per WHO 2022 (5th Edition), including -7/del(7q), -5/del(5q), complex (≥3) or monosomal karyotype, 3q26/EVI1 rearrangements, and other recognized MDS/AML-defining lesions, or with unequivocal dysplasia/blast excess; isolated +8, -Y, del(20q), or small (<10%) non-dysplastic clones are eligible with enhanced surveillance.
Patients with a history of cirrhosis, portal hypertension, or chronic active hepatitis.
Single group
Avatrombopag 20 mg Oral Tablet
DrugTrial participants will receive avatrombopag as 20 mg film-coated tablets. On-site administration by the trial team is required on (1) Day 1/Visit 2 (first dose) and (2) the first day a participant is titrated to 80 mg once daily (earliest Visit 6).
The effect of avatrombopag on the proportion of trial participants achieving a hematological response at Week 26 in trial participants diagnosed with AA.
Time to first hematological response defined as the number of days from baseline to first improvement in at least one of the three blood cell lineages (RBCs, platelets, and neutrophils).
Hematological response at Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 18, 22, and 26 in the Primary Investigation Phase (Core Phase) and every 4 weeks in the Extension Phase.
Duration of hematological response: Time from first documented response to loss of response on two consecutive scheduled assessments or at End of treatment (EOT).
Changes from baseline in Quality of Life (QOL) assessed using the EORTC QLQ-C30 questionnaire at Weeks 4, 8, 12, 18, and 26 in the Primary Investigation Phase (Core Phase) and every 4 weeks in the Extension Phase.
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This trial is not recruiting at the moment. You can still explore other options:
