Strong SCD

  • Research type

    Research Study

  • Full title

    A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell Disease

  • IRAS ID

    267041

  • Contact name

    Baba Inusa

  • Contact email

    baba.inusa@gstt.nhs.uk

  • Sponsor organisation

    Cyclerion Therapeutics, Inc.

  • Eudract number

    2019-001899-11

  • Clinicaltrials.gov Identifier

    NCT03285178

  • Duration of Study in the UK

    0 years, 9 months, 15 days

  • Research summary

    The safety, and effects of, taking IW-1701 (study drug) compared to placebo, for participants with Sickle Cell Disease, will be studied in this Clinical Trial. 88 participants from the USA, Lebanon, and the UK will take part in this Trial.

    Participants will take part in this Trial for approximately four to five months. The Trial is divided into four phases:
    Screening;
    Blinded Run-In
    Blinded Treatment; and
    Follow-up

    During the Run-In phase, all participants will take one tablet of study drug, once a day, for 14-17 days.

    After the Run-In, participants who are still eligible to take part in the study will begin the Treatment phase. Participants will be randomly assigned to receive a study drug (IW-1701 or placebo) and will have a 75% chance of receiving IW-1701 (study drug) and a 25% chance of receiving a placebo.

    Participants will begin the Treatment phase taking two tablets per day. The study drug dosage will be increased, in two stages (four tablets a day, and then 6 tablets a day), depending on the safety and tolerability of the study drug.

    Participants would make 5 treatment visits, if they complete the trial. At each of these visits, participants will need to have fasted for at least 2 hours before the visit, and for at least one hour after taking the study drug.

    Participants will be contacted by phone on 7 occasions, to check on their health and any medicines they are taking, or have taken since their last visit.

    Following the End of Trial Visit, participants will be invited to attend a Follow-up visit 28 days after the end of their treatment.

  • REC name

    London - City & East Research Ethics Committee

  • REC reference

    19/LO/1566

  • Date of REC Opinion

    7 Nov 2019

  • REC opinion

    Further Information Favourable Opinion