A trial to evaluate inclacumab for VOC prevention

• Research type

  Research Study

• Full title

  A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants with Sickle Cell Disease and Recurrent Vaso occlusive Crises

• IRAS ID

  1003787

• Contact name

  Kalyan Obalampalli

• Contact email

  kobalampalli@gbt.com

• Sponsor organisation

  Global Blood Therapeutics, Inc.

• Eudract number

  2020-005287-60

• Research summary

  The sponsor of this study is Global Blood Therapeutics, and this is a Phase 3 study which examines the effectiveness of a single dose of the drug, inclacumab, on its ability to reduce re-admission to hospital in patients with Sickle Cell Disease (SCD) who suffer from recurrent vaso-occlusive crises (VOCs).
  SCD is known as an inherited disorder which affects haemoglobin (the part of a red blood cell that carries oxygen through the body). Usually red blood cells are round & flexible to move easily through 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, causing blockage of blood flow and oxygen to parts of the body. These events are known as VOCs – they can cause severe pain, have the potential to cause organ damage, and in severe cases can lead to stroke or a severe complication known as Acute Chest Syndrome.
  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 blood vessels and therefore prevents 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 and to reduce the painful events, limiting the number of VOCs a patient may experience.
  Approximately 280 adult and adolescent participants (≥ 12 years of age) with SCD are expected to participate in this study at approx 60 global clinical sites. A single dose of inclacumab or a placebo will be given on day 1 and participants will remain on the trial for either 91 or 161 days, depending on whether they decide to enrol on the open-label extension trial (OLE) at day 91. The OLE is an optional, separate study where patients will receive only inclacumab (no placebo). Participants who choose not to enrol on the OLE will remain on the trial for 161 days

  SUMMARY OF RESULTS
  This study was terminated by the sponsor due to enrolment challenges, resulting in a smaller number of participants than planned. No meaningful differences in efficacy results were observed between the inclacumab and placebo groups. The single dose of inclacumab 30 mg administered in this study was generally safe and well tolerated in participants with SCD.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  21/SC/0241

• Date of REC Opinion

  24 Sep 2021

• REC opinion

  Further Information Favourable Opinion