Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Ap… (NCT03988608) | Clinical Trial Compass
CompletedPhase 2
Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
China20 participantsStarted 2019-12-09
Plain-language summary
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA). Treatment with eltrombopag was started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day. The hematological response rate was assessed at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
Who can participate
Age range18 Years – 100 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Chinese patients aged greater than or equal to 18 years old.
* Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of \> 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab.
* Platelet count ≤ 30 × 10\^9/L at screening.
* Patient must not currently have the option of stem cell transplantation.
* Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
* Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or \<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.
Exclusion Criteria:
* Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months.
* Congenital aplastic anemia
* AST or ALT ≥3 times the upper limit of normal.
* Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome).
* Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%.
* Presence of chromosomal aberration (-7/7q- detected by fluorescence in situ hybridization (FISH), or other aberrations detected by G-band staining).
* Evidence of a clonal hematologic bone marrow disorder on cytogenetics.
* Past medical history of thromboembolism within 6 months…
What they're measuring
1
Hematologic Response Rate at 6 Months (Week 26) by Investigator