Aquarius

  • Research type

    Research Study

  • Full title

    A Phase 2, Multicohort Study of Daratumumab-Based Therapies in Participants with Amyloid Light Chain (AL) Amyloidosis

  • IRAS ID

    310371

  • Contact name

    Zina Vas

  • Contact email

    zina.vas@parexel.com

  • Sponsor organisation

    Janssen Research & Development, LLC

  • Eudract number

    2021-002639-48

  • Clinicaltrials.gov Identifier

    NCT05250973

  • Duration of Study in the UK

    2 years, 5 months, 27 days

  • Research summary

    Amyloid light chain (AL) amyloidosis is a rare disorder caused by a usually small plasma cell clone producing a misfolded light chain called amyloid that deposits in vital tissues such as heart, liver, kidney GI tract and so on. Deposition of amyloid in vital organs results in serious and life-threatening organ dysfunction.
    The purpose of this study is to compare safety of different daratumumab plus bortezomib, cyclophosphamide and dexamethasone treatment regimens, in newly diagnosed participants with systemic AL amyloidosis. The study will also research the levels of daratumumab study drug in the blood of participants with diverse backgrounds with newly diagnosed amyloid light-chain (AL) amyloidosis.
    The study treatment phase will consist of 28-day cycles lasting for up to 24 months. There are 2 treatment groups in this study. Group 1 will include participants with AL amyloidosis heart disease. Group 2 includes participants with AL amyloidosis that affect the heart as well as other organs and will include participants from diverse backgrounds.
    Participants in both groups will receive dexamethasone (a steroid), diphenhydramine (an anti-allergic), acetaminophen (a fever reducer) and montelukast (to prevent breathing difficulties, if needed) to prepare their body for daratumumab. These pre-medications will be given 1 to 3 hours before the daratumumab injection. Daratumumab will be administered via subcutaneous infusion for 5 minutes. Starting at cycle 1, participants will receive daratumumab weekly (for 2 months), then every 2 weeks (for 4 months) and then once a month.
    Participants in Group 1 will receive bortezomib, cyclophosphamide and dexamethasone starting at Cycle 1 and those in Group 2 will receive bortezomib, cyclophosphamide and dexamethasone starting at Cycle 4. Both groups will receive bortezomib, cyclophosphamide and dexamethasone for up to 6 cycles.
    About 150 participants will take part in this study worldwide, with around 10 patients expected in the UK.

  • REC name

    East of England - Cambridge South Research Ethics Committee

  • REC reference

    22/EE/0066

  • Date of REC Opinion

    7 Jun 2022

  • REC opinion

    Further Information Favourable Opinion