Study identification

PURI

https://redirect.ema.europa.eu/resource/1000000225

EU PAS number

EUPAS1000000225

Study ID

1000000225

Official title and acronym

Effectiveness of voxelotor in individuals with sickle cell disease and a history of red blood cell transfusions: A non-interventional, retrospective cohort study using real-world data in the United States

DARWIN EU® study

No

Study countries

United States

Study description

After identifying the populations of interest using CEM on age and calendar time, the distribution of baseline covariates will be evaluated in the 365 days prior to index. Continuous variables will be described using mean values with standard deviations and median values with interquartile range; categorical variables will be described as the number of patients and percent. For variables derived from diagnoses, procedures, and prescription codes, patients are assumed to have experienced the event of interest if the relevant code(s) are found among their claim records. Otherwise, it is assumed that the patient did not experience the event, thus resulting in no missing data for these variables

Study status

Planned
Research institutions and networks

Institutions

Aetion
Spain
First published:
16/07/2024
InstitutionOtherENCePP partner

Contact details

Carmine Colavecchia

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Pfizer
Study protocol
Updated protocol

C5341060 Non Interventional Study Protocol V01_01 August 2024.pdf

English (567.29 KB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Unknown

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

Not applicable