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临床试验/EUCTR2016-000558-37-DE
EUCTR2016-000558-37-DE进行中（未招募）1 期

A Phase 1/2 Study of ARQ 092 (Miransertib) in Subjects with PIK3CA-related Overgrowth Spectrum and Proteus Syndrome

Merck Sharp & Dohme Corp0 个研究点目标入组 65 人开始时间: 2020年3月18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
65
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

  • •1.Signed informed consent, and when applicable, signed assent. Adults who are unable to consent except by means of a Legally Authorized Representative (LAR) may participate in the study.
  • •2.Cohort 1 (PROS) specific criteria
  • •Male or female subjects = 2 years and =30 years of age with BSA of = 0.33 m2
  • •Have clinical diagnosis of PROS per Diagnostic Criteria for PROS and documented somatic PIK3CA variant
  • •Have at least one lesion that can be measured by study- standardized volumetric MRI
  • •3.Cohort 2 (PS) specific criteria
  • •Male or female subjects = 2 years and =18 years of age with BSA of = 0.33 m2
  • •Have clinical diagnosis of PS per Diagnostic Criteria for PS and documented somatic AKT1 variant
  • •Have at least one plantar CCTN and pre-CCTN lesion that can be measured by standardized photography.
  • •4.Cohort 3 specific criteria
  • •Male or female subjects =2 years old (there is no upper age limit at the time of enrollment) with BSA of = 0.33 m2
  • •PROS specific inclusion criterion: Have clinical diagnosis of PROS per Diagnostic Criteria for PROS (see Appendix 1) and documented somatic PIK3CA variant
  • •PS specific criterion: Have clinical diagnosis of PS per Diagnostic Criteria for PS (see Appendix 1) and documented somatic AKT1 variant
  • •Subjects must not be eligible for enrollment in Cohort 1 (PROS) or Cohort 2 (PS): Older than 30 years of age (PROS) or 18 years of age (PS) (exceed the upper limit of age allowed for Cohort 1 or Cohort 2 enrollment)
  • •Do not have measurable disease:
  • •-PROS: do not have at least one lesion that can be measured by study- standardized volumetric MRI or to whom MRI cannot be performed
  • •-PS: no plantar CCTN lesion(s) or the CCTN lesion(s) do not satisfy protocol defined criteria of the measurable lesion
  • •5.Cohort 4 (PROS or PS) specific criteria
  • •Subjects previously treated with ARQ 092 or currently receiving ARQ 092 under Compassionate Use/Expanded Access
  • •Note: Subjects should meet the age criterion by/on the date of the first dose, Cycle 1 Day 1.
  • •All Cohorts:
  • •6.Archival or fresh overgrowth tissue sample available to be shipped to Sponsor or designee
  • •7.Except for Cohort 4, clinically progressive or worsening disease defined as an increase in number or size of the overgrowth lesion(s) in the last 6 months as assessed by the Investigator
  • •8.Adequate organ function as indicated by the following laboratory values:
  • •Hematological
  • •a.Hemoglobin (Hgb) depending on age:
  • •2-5 years male and female: = 10.0 g/dL
  • •6-9 years male and female: = 11.5 g/dL
  • •10-17 years female: = 11.0 g/dL
  • •10-17 years male: = 11.5 g/dL
  • •=18 years male and female: = 10.0 g/dL
  • •b.Glycated hemoglobin (HbA1c): = 8% (= 64 mmol/mol)
  • •c.Absolute neutrophil count (ANC): = 1.5 x 10_9/L
  • •d.Platelet count = 150 x 10_9/L
  • •a.Total bilirubin = 1.5 x upper limit of normal (ULN)/L
  • •b.Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) = 3 x ULN
  • •a.Serum creatinine depending on age:
  • •2-5 years male and female: maximum 0.80 mg/dL
  • •6-10 years male and female: maximum 1.0 mg/dL
  • •11-15 years male and female: maximum 1.2 mg/dL
  • •> 15 years male and female: maximum 1.5 mg/dL
  • •b.Estimated Glomerular Filtration Rate (eGFR): = 60 mL/min/1.73 m2
  • •Metabolic (lipids)
  • •a.Cholesterol: = 400 mg/dL (= 10.34 mmol/L)
  • •b.Triglyceride: = 500 mg/dL (= 5.7 mmol/L)
  • •9.Female subjects must be of non-child producing potential or in postmenopausal state. Women of non-child producing potential are defined as those who underwent permanent sterilization (permanent ster

排除标准

  • •1. History of Type 1 diabetes mellitus or Type 2 uncontrolled diabetes mellitus requiring regular medication (other than metformin or other oral hypoglycemic agents) or fasting glucose = 160 mg/dL (if > 12 years old) and = 180 mg/dL (if = 12 years old) at the screening visit
  • •2. History of significant cardiac disorders:
  • •Myocardial infarction (MI) or congestive heart failure defined as Class II-IV per the New York Heart Association (NYHA) classification within 6 months of the first dose of ARQ 092 (MI occurring > 6 months of the first dose of ARQ 092 will be permitted)
  • •Grade 2 (per current version of National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE]) or worse conduction defect (e.g., right or left bundle branch block)
  • •3. Major surgery or locoregional therapy within four weeks of the first dose of ARQ 092
  • •4. Any experimental systemic therapy for the purposes of treating PROS or PS (e.g., sirolimus, everolimus, high dose steroids) within two weeks of the first dose of ARQ 092
  • •5. Intolerance of or severe toxicity attributed to AKT inhibitors (e.g., ARQ 092, uprosertib, afuresertib, ipatasertib)
  • •6. Concurrent severe uncontrolled illness not related to PROS or PS, e.g.,
  • •Ongoing or active infection
  • •Known human immunodeficiency virus (HIV) infection
  • •Malabsorption syndrome
  • •Psychiatric illness/substance abuse/social situation that would limit compliance with study requirements
  • •7. Female of child-producing potential, pregnant or breastfeeding. Female subject of child-producing potential is defined as a
  • •premenopausal female capable of becoming pregnant, including women from the onset of menstruation (menarche) until the onset of menopause.
  • •8. Inability to comply with study evaluations or to follow drug administration guidelines

研究者

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