Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000170

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
First published:
01/02/2024
Institution

Contact details

Michelle Xu

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

GBT, Pfizer
Study protocol
Initial protocol

GBT440-4R2 (C5341019) Registry Protocol (Prospective) GBT template 3 February 2021_FINAL.pdf

English (770.73 KB - PDF)View document
Updated protocol

No Pedigree GBT440-4R2 (C5341019)_Non-Interventional Study Protocol _Final_v1.0_10Oct2023.pdf

English (1.6 MB - PDF)View document
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

Not applicable