EUCTR2016-000558-37-FR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
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- ArQule, Inc.
- 入组人数
- 65
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
入选标准
- •Inclusion Criteria - Part B
- •1.Signed informed consent, and when applicable, signed assent
- •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 with BSA of = 0.33 m2 and who fail to meet the eligibility criteria for Cohorts 1 or 2
- •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.Male or female subjects of child-producing potential must agree to use double-barrier contraceptive measures, oral contraception, or avoidance of intercourse during the study and for 90 days after the last dose of ARQ 092
- •10.Ability to complete the study questionnaires by the subject or his/her caregiver
- •Are the trial subjects under 18? yes
- •Number of subjects for this ag
排除标准
- •Exclusion Criteria – Part B
- •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)
- •Major surgery or locoregional therapy within four weeks of the first dose of ARQ 092
- •3.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
- •4.Intolerance of or severe toxicity attributed to AKT inhibitors (e.g., ARQ 092, uprosertib, afuresertib, ipatasertib)
- •5.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
- •6.Pregnant or breastfeeding
- •7.Inability to comply with study evaluations or to follow drug administration guidelines
研究者
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