An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)® (Voxelotor)

Study identification

• EU PAS number:
  EUPAS1000000170
• Study ID:
  1000000170
• Official title and acronym:
  An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)® (Voxelotor)
• DARWIN EU® study:
  No
• Study countries:
  United States
• Study description:
  The primary objective is to gather long term data on Oxbryta® (Voxelotor)® in a real-world setting. The; following are categories of interest in participants with SCD treated with Oxbryta® (Voxelotor): ; - Clinical outcomes, as assessed by clinical and laboratory assessments of hematological parameters and end organ damage, and rate of significant clinical events ; - Healthcare resource utilization ; - Health-related quality of life (HRQoL), as assessed by participants, parents/caregivers, and clinicians ; - Assess the safety and tolerability of Oxbryta® (Voxelotor)®
• Study status:
  Finalised

Research institutions and networks

Institutions

Pfizer

Study timelines

Date when funding contract was signed

Planned:02/02/2022

Actual:02/02/2022

Study start date

Planned:02/02/2022

Actual:02/02/2022

Data analysis start date

Planned:02/02/2022

Actual:02/02/2022

Date of final study report

Planned:30/04/2030

Actual:06/03/2025

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

GBT, Pfizer

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable

Other study registration identification numbers and links

NCT04930445