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A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

Trial Profile

A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

Status: Discontinued
Phase of Trial: Phase II

Latest Information Update: 18 Nov 2024

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At a glance

  • Drugs Voxelotor (Primary)
  • Indications Sickle cell anaemia
  • Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
  • Acronyms HOPE; HOPE-KIDS
  • Sponsors Global Blood Therapeutics

Most Recent Events

  • 14 Nov 2024 Status changed from suspended to discontinued (Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment).
  • 29 Apr 2024 Planned End Date changed from 4 Jan 2025 to 20 Dec 2026.
  • 29 Apr 2024 Planned primary completion date changed from 4 Jan 2025 to 30 Nov 2026.

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