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NCT Number: NCT04930328

Retrospective Real World Oxbryta® Data Collection and Analysis Study

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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

0 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Connecticut Health, Farmington, Connecticut, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

  • 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
  • Male or female patients with documented diagnosis of SCD (all genotypes)
  • Have been treated with Oxbryta for at least 2 weeks, according to the Oxbryta USPI

Exclusion criteria

-

Treatment and study plan

Oxbryta® (voxelotor) 500-mg Tablets

Drug

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®

Primary outcomes

  1. Change from pre-Oxbryta treatment period in Hemoglobin (Hb)

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  2. Change from pre-Oxbryta treatment period in percent Reticulocytes

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  3. Change from pre-Oxbryta treatment period in Absolute Reticulocytes

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  4. Change from pre-Oxbryta treatment period in Bilirubin

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  5. Incidence of significant SCD-related clinical events

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

    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)

  6. Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  7. Change from pre-Oxbryta treatment period in incidence of emergency department (ED) visits

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  8. Change from pre-Oxbryta treatment period in incidence of hospitalizations (including total length of stay and time in intensive care unit [ICU], if applicable)

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  9. Change from pre-Oxbryta treatment period in incidence of red blood cell transfusions

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  10. Incidence and severity of serious adverse events (SAEs)

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

  11. Incidence and severity of adverse events (AEs) of interest

    Time frame: 1 year before and 1 year after the first dose of Oxbryta

    Such as Rash, Diarrhea, Headache, AEs leading to Oxbryta dose modification or discontinuation

Sponsors and collaborators

Lead sponsor

Pfizer

Industry

Registry information

Official study title

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

Acronym: RETRO

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Jun 18, 2021
Registry last updated
Mar 6, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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