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临床试验/EUCTR2020-005234-15-AT
EUCTR2020-005234-15-AT进行中（未招募）1 期

A Phase 1/2, Single-arm Study to Evaluate the Safety, Pharmacokinetics, and Antitumor Activity of Avapritinib in Pediatric Patients with Solid Tumors Dependent on KIT or PDGFRA Signaling

Blueprint Medicines Corporation0 个研究点目标入组 37 人开始时间: 2021年12月13日最近更新:
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试验速览

阶段
1 期
状态
进行中（未招募）
入组人数
37
研究概览入排标准研究者记录与标识符相似试验

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

  • •- Patient is 2 to <18 years of age at the time the parent/guardian signs the Informed Consent Form.
  • •- Confirmed diagnosis of R/R solid tumor, including CNS tumors, with a mutation (including nonsynonymous point mutations, insertions, and deletions) PDGFRA and/or KIT that has progressed despite standard therapy and no alternative treatment options are available. Patient with R/R solid tumors with only PDGFRA and/or KIT amplifications may be included with approval from the Sponsor OR
  • •Confirmed diagnosis of DMG-H3K27a that has failed standard therapy or for which no standard therapy that may convey clinical benefit exists, as judged by the Investigator.
  • •- Disease extent:
  • •-Part 1: All patients must have at least 1 measurable lesion as defined by RECIST v1.1 or RANO (for CNS tumors). If radiation therapy has been administered, at least 1 measurable lesion must not have been irradiated, or must have clearly progressed since being irradiated as per RANO and must be = 12 weeks from radiation to any target lesion.
  • •-Part 2: All patients must have at least 1 measurable lesion as defined by RECIST v1.1 or RANO (for CNS tumors). For patients with DMG-H3K27a or PDGFRA and/or KIT mutant/amplified solid tumors, including CNS tumors, that have progressed despite prior therapy who have received radiation therapy, at least 1 measurable lesion must not have been irradiated, or must have clearly progressed since being irradiated as per RANO and must be = 12 weeks from radiation to any target lesion. For up to 5 patients with newly diagnosed DMG-H3K27a where there is no standard therapy that may convey clinical benefit exists as judged by the investigator, progression of disease of a measurable lesion after irradiation is not required.
  • •- Patients with CNS disease should be on a stable dose of corticosteroids for at least 7 days prior to first dose of avapritinib, with no plans for dose escalation.
  • •- Patients must have a Lansky (<16 years of age) or Karnofsky (=16 years of age) score of at least 50.
  • •Are the trial subjects under 18? yes
  • •Number of subjects for this age range: 37
  • •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

  • •1. Patient has any of the following within 14 days before the first dose of study treatment:
  • •a. Platelet count <75 × 10*9/L (<100 × 10*9/L if a CNS tumor) with no platelet transfusion within 14 days prior to the measurement.
  • •b. Absolute neutrophil count (ANC) <1.0 × 10*9/L.
  • •c. Hemoglobin <8.0 g/dL with no RBC transfusion =7 days prior to the measurement.
  • •d. AST or ALT >3 × the ULN for age; except in patients with tumor involvement of the liver who must not have AST and ALT >5 × ULN for age.
  • •e. Total bilirubin xULN for age; and in presence of Gilbert’s syndrome, total bilirubin >3 × ULN or direct bilirubin > 1.5 × ULN.
  • •f. Serum creatinine >1.5 × ULN for age.
  • •g. International normalized ratio or prothrombin time (PT) >ULN (>1.5 × ULN if on prophylactic reversible anticoagulants).
  • •2. Patient has a QTcF >470 msec. Patient has a familial or personal history of prolonged QT syndrome or Torsades de pointes.
  • •3. Patient has clinically significant, uncontrolled cardiovascular disease including congestive heart failure Grade III or IV according to the New York Heart Association (NYHA) classification; myocardial infarction or unstable angina within the previous 6 months, uncontrolled hypertension (>95th percentile for age), or clinically significant, uncontrolled arrhythmias, including bradyarrhythmias that may cause QT prolongation (eg, Type II second degree heart block or third-degree eart block).
  • •4. Patient received the following systemic antineoplastic therapies:
  • •a. Temozolomide within 4 weeks prior to the first dose of study drug
  • •b. Nitrosurea within 6 weeks prior to the first dose of study drug
  • •c. Any other systemic antineoplastic therapy (including experimental therapy within 5 half-lives or 28 days prior to the first dose of study drug, whichever is shorter.
  • •d. Focal external beam radiotherapy, including stereotactic radiosurgery, within 6 weeks prior to the first dose of avapritinib to either target or nontarget lesions. Systemic radiopharmaceuticals, including nonstereotactic radiosurgery, within 2 weeks of the first dose of avapritinib (within 6 weeks for patients with CNS tumors). Craniospinal irradiation within 6 weeks prior to the first dose of avapritinib.
  • •e. All AEs related to other antineoplastic therapies (eg, systemic antineoplastics, radiotherapy) must have resolved to Grade ?1 (Grade =2 for peripheral neuropathy and/or ototoxicity) prior to the first dose of avapritinib.
  • •5. Patient has previously received treatment with avapritinib.
  • •6. Patient received autologous stem cell transplant (SCT) following myeloablative therapy or chimeric antigen receptor T cell (CAR-T) therapy within 3 months prior to the first dose of avapritinib or prior allogeneic stem cell transplant within 1 year and no evidence of Grade 1 or greater graft-versus-host disease and no immunosuppressants for graft-versus-host disease (steroid for primary malignancy being permitted). Patients who received stem cell reinfusion following nonmyeloablative therapy are eligible once they meet the peripheral blood count criteria in Exclusion Criterion #1.
  • •7. Patient requires ongoing treatment or has received treatment within 28 days before the start of avapritinib administration with drugs or foods that are strong CYP3A inhibitors or inducers.
  • •8. Patient has had a major surgical procedure within 14 days of the first dose of study treatment (procedures such as central venous catheter placement, tumor needle biopsy, and feeding tube placement are not considered major surg

研究者

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