A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000334
• EU PAS number:
  EUPAS1000000334
• Study ID:
  1000000334
• Official title and acronym:
  A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)
• DARWIN EU® study:
  No
• Study countries:
  United States
• Study description:
  This is a retrospective data collection and analysis study. Patients will have received treatment with Oxbryta as prescribed by their physician at the approved dose per local prescribing information, as part of their usual care.; ; Primary; The following are categories of interest in patients with SCD treated with Oxbryta:; • Clinical outcomes, as assessed by clinical and laboratory assessments of hematological parameters and end organ damage, and incidence of significant clinical events; • Healthcare resource utilization; • Health-related quality of life (HRQoL), as assessed by patient-reported outcome (PRO) measures and clinician-reported outcomes (ClinRO); ; Safety: The safety objective is to assess the safety and tolerability of Oxbryta.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Pfizer

Study timelines

Date when funding contract was signed

Planned: 26/01/2021

Actual: 26/01/2021

Study start date

Planned: 31/03/2021

Actual: 20/03/2021

Date of final study report

Planned: 14/10/2024

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Pfizer

Study protocol

Initial protocol

GBT440-4R1 Registry Protocol (Retrospective)_FINAL redacted.pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable