Study to assess safety/efficacy of ARQ092 in subjects with PROS and PS

  • Research type

    Research Study

  • Full title

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

  • IRAS ID

    264994

  • Contact name

    Lea Solman

  • Contact email

    lea.solman@gosh.nhs.uk

  • Sponsor organisation

    ArQule, Inc.

  • Eudract number

    2016-000558-37

  • Clinicaltrials.gov Identifier

    NCT03094832

  • Duration of Study in the UK

    4 years, 6 months, 12 days

  • Research summary

    Research Summary

    The main purpose of this study is to assess the efficacy of ARQ 092 (miransertib) in pediatric and adult patients (2 years of age and older) with PIK3CA-related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS) with documented PIK3CA or AKT1 mutations.
    The study drug is ARQ 092 (Miransertib).
    The study consists of Part A and Part B. Only Part B will be conducted in the UK and will consist of 4 cohorts: Cohort 1 will enroll subjects with PROS; Cohort 2 will enroll subjects with PS; Cohort 3 will enroll subjects with either PROS or PS who do not meet the eligibility criteria for Cohorts 1 or 2 and Cohort 4 will enroll subjects previously treated with ARQ 092 or currently receiving ARQ 092 under Compassionate Use/Expanded Access.
    Up to 65 subjects will be enrolled globally (France, Spain, UK, Italy, Australia, United States, Latin America). Only one site will participate in the UK.
    We expect the duration of each patient’s participation to be approximately 48 months.

    Summery of results

    https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agba4yu73OCS9U-2BkKS40W1kfatiYvtk1-2B2gi1K6vKImual9G6VG4-2FeFhNKGHEnJKTVSp1XWAgONgc-2B5UGwt-2B-2BnBZjLU9XMaTeyBQzZqULiiaNTFO3C_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YKRAIkYPlu9QHaL92V0771GiqHnXm3FCXVHm1qDBeJexkfaJjFWDXKAknkAOlVCjD81qTPfOh9SxFCr1w8w0IZ9uUDR0pse8oZ6E-2B1yP0ITglUhLbx3940-2BS5x5wLIAhQ7SBGhGnWCyUaJsxvK-2F5cQhEqg-2B852muF5M-2F9IersdevQ-3D-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7Cc00113222f3b462d7fa408dacbb3071a%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638046267017312484%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=ssXN8dOP16sbabvDSQR3jODf1uSVnVPcn2DLpzJLW4s%3D&reserved=0

  • REC name

    London - Brighton & Sussex Research Ethics Committee

  • REC reference

    20/LO/0047

  • Date of REC Opinion

    16 Jul 2020

  • REC opinion

    Further Information Favourable Opinion