EUCTR2015-003166-91-GB进行中（未招募）1 期
A phase II, open-label, non-controlled, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia.
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion Criteria specific to Cohort A (patients with relapsed/refractory SAA or recurrent AA):
- •1. Prior history of diagnosis of SAA.
- •2. Diagnosis of relapsed/refractory SAA or recurrent AA following IST for SAA at the time of enrollment. Patients with
- •recurrent AA (e.g., losing their response) are exempt from meeting the diagnostic criteria for relapsed SAA at the time of enrollment, but must have been previously diagnosed with SAA.
- •3. Agree to concurrent eltrombopag treatment with appropriate, investigator-selected IST with either hATG + CsA or CsA.
- •Inclusion Criteria specific to Cohort B (patients with previously untreated SAA):
- •4. Diagnosis of SAA at the time of enrollment.
- •5. Patients must not have been previously treated for SAA
- •6. Patients must agree to treatment with hATG + CsA concurrent with
- •eltrombopag.
- •Inclusion Criteria for all patients regardless of cohort:
- •7. Age 1 to <18 years
- •8. Where appropriate, assessments to rule out congenital/inherited bone marrow failure syndromes and other causes of immune-mediated pancytopenia, which may be treated with transplant, must be completed
- •prior to enrollment.
- •9. Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient. (Candidacy for HSCT will be determined as per local practice.)
- •10. Bone marrow aspirate/biopsy at any time during the 4 weeks prior to first dose of eltrombopag
- •[11. Removed]
- •12. Performance status score: Karnofsky =50 or Lansky =50 (depending on age)
- •[13. Removed]
- •[14. Removed]
- •15. Written informed consent signed by a parent or legal guardian prior
- •to initiation of any study specific procedure.
- •16. Normal karyotype within 4 weeks prior to first dose of eltrombopag. If there are insufficient metaphases (< 10) to determine karyotype, a repeat marrow aspirate is required. If upon repeat bone marrow aspirate, the number of metaphases is insufficient (< 10), then FISH probes performed in marrow aspirate as per protocol must be normal.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 60
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •2. Prior and/or active medical history of:
- •Fanconi anemia (via chromosomal breakage test or growth arrest by flow cytometry)
- •Other known underlying congenital/inherited marrow failure syndromes (such as, but not limited to, Dyskeratosis Congenita, Congenital Amegakaryocytic Thrombocytopenia, or Shwachman-Diamond Syndrome).
- •Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and/or PNH clones >50% of PMN or RBC at time of enrollment
- •Any cytogenetic abnormalities by karyotyping or FISH
- •Myelodysplastic syndrome (MDS)
- •Other known or suspected underlying primary immunodeficiency
- •Any malignancy
- •3. Active infection not responding to appropriate therapy
- •4. Prior eltrombopag or other thrombopoietin receptor (TPO-R) agonist treatment for at least 2 months and a lack of response.
- •5. Any out of range lab values:
- •Serum Creatinine >2.5 × upper limit of normal (ULN),
- •Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 × ULN.
研究者
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