Retrospective Real World Oxbryta® Data Collection and Analysis Study (RETRO)

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

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Oxbryta® (voxelotor) 500-mg Tablets

Detailed 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.

• Study Type: Observational
• Enrollment (Actual): 216

Contacts and Locations

Study Locations

• United States
  • Connecticut
    • Farmington, Connecticut, United States, 06030
      • University of Connecticut Health
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Central Michigan University/Children's Hospital of Michigan
  • New Jersey
    • New Brunswick, New Jersey, United States, 08903-2681
      • Rutgers Cancer Institute of New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center
  • New York
    • Bronx, New York, United States, 10467
      • Montefiore Medical Center
  • North Carolina
    • Charlotte, North Carolina, United States, 28204
      • Levine Cancer Institute
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Texas
    • Dallas, Texas, United States, 75390
      • UT Southwestern Medical Center
    • Dallas, Texas, United States, 75235
      • Parkland Health & Hospital System
    • Houston, Texas, United States, 77030
      • University of Texas Health Science Center at Houston

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.

Description

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:

-

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Retrospective Data Collection	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.; Other Names: Voxelotor; Oxbryta®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from pre-Oxbryta treatment period in Hemoglobin (Hb)		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in percent Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits		1 year before and 1 year after the first dose of Oxbryta
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
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
Incidence and severity of serious adverse events (SAEs)		1 year before and 1 year after the first dose of Oxbryta
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
Change from pre-Oxbryta treatment period in Absolute Reticulocytes		1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Bilirubin		1 year before and 1 year after the first dose of Oxbryta
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
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

Collaborators and Investigators

• Sponsor: Pfizer
• Collaborators: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): March 20, 2021
• Primary Completion (Actual): February 25, 2022
• Study Completion (Actual): February 25, 2022

Study Registration Dates

• First Submitted: June 4, 2021
• First Submitted That Met QC Criteria: June 10, 2021
• First Posted (Actual): June 18, 2021

Study Record Updates

• Last Update Posted (Actual): March 6, 2024
• Last Update Submitted That Met QC Criteria: March 4, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Sickle Cell Disease Registry
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: GBT440-4R1; C5341018 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No