GBT440-031 - GBT440 in Patients With Sickle Cell Disease (HOPE)

• Research type

  Research Study

• Full title

  A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease

• IRAS ID

  219400

• Contact name

  Jo Howard

• Contact email

  Jo.Howard@gstt.nhs.uk

• Sponsor organisation

  Global Blood Therapeutics, Inc.

• Eudract number

  2016-003370-40

• Duration of Study in the UK

  1 years, 10 months, 22 days

• Research summary

  Sickle Cell Disease (SCD) is a serious life-long illness that causes low red blood cell counts (anaemia), fatigue, and damage to blood vessels leading to problems such as acute painful episodes, strokes and leg ulcers. It can also result in organ damage and lead to premature death.

  The main purpose of this research study is to determine the effect of the study drug GBT440, compared to placebo in increasing red blood cell counts and reducing the severity of symptoms of SCD. A placebo is a “dummy drug”: it looks like the study drug but contains no medicine. Participants will be asked to complete a short fit-for-purpose questionnaire each day on an eDiary to capture changes in SCD symptom severity.

  Up to approximately 400 adults and adolescents with SCD will be enrolled as follows:
  - Group 1: approximately 60 participants will be randomised in a 1:1:1 ratio to receive GBT440 900 mg/day, GBT440 1500 mg/day, or matching placebo
  - Group 2: Up to approximately 90 participants will be randomised in a 1:1:1 ratio to receive GBT440 900 mg/day, GBT440 1500 mg/day, or matching placebo
  - Group 3: At least 190 participants and up to 250 participants will be randomised in a 1:1 ratio to receive GBT440 (900 mg/day or 1500 mg/day), or matching placebo

  The study will take place over a minimum of 10 weeks and up to 81 weeks. At least 50 adolescent participants (12 to <18 years at the time of screening) will be included. The amount of time participants are on study will depend on when they enrol relative to other participants.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  17/LO/0113

• Date of REC Opinion

  5 Apr 2017

• REC opinion

  Further Information Favourable Opinion