A Phase I study of Human Pharmacokinetics and Safety of ORY-1001

  • Research type

    Research Study

  • Full title

    A phase I study of Human Pharmacokinetics and Safety of ORY-1001, and LSD1 inhibitor, in relapsed or refractory acute leukaemia (AL)

  • IRAS ID

    140382

  • Contact name

    Tim Somervaille

  • Contact email

    tim.somervaille@cruk.manchester.ac.uk

  • Sponsor organisation

    Oryzon Genomics S.A.

  • Eudract number

    2013-002447-29

  • Research summary

    Mixed lineage leukemia is a very aggressive blood cancer with a poor prognosis and despite the availability of advanced treatment methods cure rates have stopped their progression over the last years.
    ORY-1001 targets leukemic stem cells, induces differentiation, and provokes apoptosis to different degrees and could function well as a monotreatment for specific AML subtypes or more broadly as an adjuvant treatment to standard cytotoxic or differentiation therapy for AML.

    The current study is performed to characterize the pharmacokinetics, safety, tolerability and pharmacodynamics of ORY-1001 for oral intake at various doses to patients with relapsed / refractory AL and to establish the maximum tolerated dose.

    This study will be performed to characterize the pharmacokinetics of ORY-1001 for oral intake.

    ORY-1001 is not been brought into clinical development for haematological malignancies yet. This phase I part of the clinical study, CL01-ORY-1001, will include patients with a relapsed or refractory acute leukaemia at age 16 or above.

  • REC name

    North West - Greater Manchester South Research Ethics Committee

  • REC reference

    13/NW/0830

  • Date of REC Opinion

    10 Feb 2014

  • REC opinion

    Further Information Favourable Opinion