A trial to evaluate inclacumab for VOC prevention

  • Research type

    Research Study

  • Full title

    A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Safety and Efficacy of Inclacumab in Participants with Sickle Cell Disease Experiencing Vaso-occlusive Crises

  • IRAS ID

    1003589

  • Contact name

    Kalyan Obalampalli

  • Contact email

    kobalampalli@gbt.com

  • Sponsor organisation

    Global Blood Therapeutics, Inc.

  • Eudract number

    2020-005286-13

  • Clinicaltrials.gov Identifier

    NCT04935879

  • Research summary

    The study’s sponsor is Global Blood Therapeutics, Inc and this is a Phase 3 study which involves the examination of the study drug inclacumab to help reduce Vaso-occlusive Crises (VOCs) in patients with Sickle Cell Disease (SCD).
    SCD is known as an inherited disorder which affects the haemoglobin (the part of a red blood cell that carries oxygen through the body). Usually red blood cells are round shaped and are flexible to move easily through the blood vessels. However, with SCD the red blood cells are misshaped and sticky, which means the cells cannot bend and move easily through the body, which in turn can cause the blockage of blood flow and oxygen to parts of the body. This can cause pains known as VOCs. SCD is a lifelong illness and signs of SCD commonly appear during childhood and the symptoms include fatigue, severe pains, anaemia and increased risk of serious infections.
    Inclacumab is a fully human monoclonal antibody which binds to a protein, called P-selectin, on the lining of the blood vessels and stops the sickle red blood cells from sticking to the blood vessel walls and therefore stops the blockage of blood flow. The blockage of the blood vessels leads to painful VOCs and inclacumab is designed to prevent this from occurring so that blood can flow normally around the body and to reduce the painful events.
    Approximately 240 adult and adolescent participants (≥ 12 years of age) with SCD are expected to participate in this study at approximately 75 global clinical sites with the duration of the study lasting approximately 52 to 64 weeks. This includes a 28-day screening period, a 48-week treatment period and either a 12-week or 24-week follow-up period, which depends on whether the participate chooses to take part in the open-labelled study.
    Some Study Assessments will include physical examination, blood samples, discussion of medical and VOC history, height and weight measurements.

  • REC name

    South Central - Berkshire Research Ethics Committee

  • REC reference

    21/SC/0242

  • Date of REC Opinion

    24 Sep 2021

  • REC opinion

    Further Information Favourable Opinion