Retrospective Real World Oxbryta® Data Collection and Analysis Study

Sickle Cell Disease Clinical Trial

— RETRO  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04930328
• Other study ID #: GBT440-4R1
• Secondary ID: C5341018
• Status: Completed
• Start date: March 20, 2021
• Est. completion date: February 25, 2022

Study information

• Verified date: March 2024
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The aim of this study is to collect and analyze retrospective data on Oxbryta in a real-world setting. This is a multicenter, retrospective data collection and analysis study to characterize health outcomes in approximately 300 patients with SCD who have been treated with Oxbryta as part of their usual care. Any patient with SCD who received Oxbryta treatment for at least 2 weeks as part of their usual care according to the Oxbryta US Prescribing Information (USPI) is eligible to participate. Study data from 1 year before and up to 1 year after the first dose of Oxbryta will be entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff.

Description:

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) The safety objective is to assess the safety and tolerability of Oxbryta.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 216
• Est. completion date: February 25, 2022
• Est. primary completion date: February 25, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years and older

Eligibility

Inclusion Criteria:

Patients who meet all the following criteria will be eligible for inclusion in this study:

1. Willing and able to provide written informed consent (ages greater or equal to 18 years) or parental/guardian consent and patient assent (age <18 years), as required by the IRB or institution or IRB, per local regulations

2. Male or female patients with documented diagnosis of SCD (all genotypes)

3. Have been treated with Oxbryta for at least 2 weeks, according to the Oxbryta USPI Exclusion Criteria: -

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• Oxbryta® (voxelotor) 500-mg Tablets
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.

Locations

Country	Name	City	State
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	Montefiore Medical Center	Bronx	New York
United States	Levine Cancer Institute	Charlotte	North Carolina
United States	Parkland Health & Hospital System	Dallas	Texas
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Central Michigan University/Children's Hospital of Michigan	Detroit	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Connecticut Health	Farmington	Connecticut
United States	University of Texas Health Science Center at Houston	Houston	Texas
United States	Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey
United States	Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center	New Brunswick	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from pre-Oxbryta treatment period in Hemoglobin (Hb)		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in percent Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in Absolute Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in Bilirubin		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence of significant SCD-related clinical events	Such as vaso-occlusive crisis (VOC), acute chest syndrome (ACS), priapism, cerebral infarcts, transient ischemic attack (TIA), leg ulcers, and measures of cardiac function and pulmonary hypertension (PH)	1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of emergency department (ED) visits		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of hospitalizations (including total length of stay and time in intensive care unit [ICU], if applicable)		1 year before and 1 year after the first dose of Oxbryta
Primary	Change from pre-Oxbryta treatment period in incidence of red blood cell transfusions		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence and severity of serious adverse events (SAEs)		1 year before and 1 year after the first dose of Oxbryta
Primary	Incidence and severity of adverse events (AEs) of interest	Such as Rash, Diarrhea, Headache, AEs leading to Oxbryta dose modification or discontinuation	1 year before and 1 year after the first dose of Oxbryta

External Links

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