GBTT440-032 Phase 3 study in participants with Sickle Cell Disease

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo Controlled Study of Voxelotor (GBT440) in Pediatric Participants with Sickle Cell Disease and an Open-label Study in Infants with Sickle Cell Disease (HOPE Kids 2)

• IRAS ID

  242661

• Contact name

  Baba Inusa

• Sponsor organisation

  Global Blood Therapeutics

• Eudract number

  2017-000903-26

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  The main purpose of this research study is to determine the effect of the study drug compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of Sickle Cell Disease. The study drug is known as voxelotor. It was designed to help the haemoglobin in red blood cells hold on to more oxygen as they travel through the body. This keeps the cells in their normal shape so they do not sickle. The study drug has been used in other smaller clinical studies which has shown that the drug has been tolerated well in healthy volunteers and patients with SCD. A Phase 3 study in adults and adolescents with SCD (study GBT440-031) is currently ongoing.
  Part A is randomised, double-blind, placebo-controlled participants will be divided into two groups, Group 1 and Group 2.Group 1 - Participants are ages 6 to less than 12 years old, Approximately 60 participants will be in this group.
  Group 2 Participants are ages 2 to less than 6 years old. Enrollment will only begin after safety data from 12 participants in Group 1 has been reviewed and analysed. Approximately 60 participants will be in this group
  Part B:
  Part B is open-label, single-arm and will consist of participants ages 9 months to less than 2 years old.
  Enrollment will only begin after data in 12 participants in Cohort 2 has been reviewed and analysed for safety and tolerability. Approximately 20 participants will be in this group Participants in Part B will all receive voxelotor. No one will receive placebo. The study will take approximately 50 weeks.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  18/LO/0359

• Date of REC Opinion

  24 Oct 2018

• REC opinion

  Further Information Favourable Opinion