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| ID | Type | Description | Link |
|---|---|---|---|
| C5341018 | Other Identifier | Alias Study Number |
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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.
The following are categories of interest in patients with SCD treated with Oxbryta:
The safety objective is to assess the safety and tolerability of Oxbryta.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Retrospective Data Collection | Retrospective Data Collection |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| 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 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 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 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 |
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Inclusion Criteria:
Patients who meet all the following criteria will be eligible for inclusion in this study:
Exclusion Criteria:
-
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All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Connecticut Health | Farmington | Connecticut | 06030 | United States | ||
| Brigham and Women's Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41701977 | Derived | Andemariam B, Shah N, Ershler WB, Ivy ED, Darbari DS, Anderson A, Rai P, Wun T, Yu CR, Purdie DM, Valenzuela CM, Xu M. Safety and effectiveness of voxelotor in individuals with sickle cell disease in the RETRO and PROSPECT US registries. Blood Adv. 2026 May 12;10(9):3032-3043. doi: 10.1182/bloodadvances.2025018992. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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|
| 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 |
| Boston |
| Massachusetts |
| 02115 |
| United States |
| Dana-Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| Central Michigan University/Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center | New Brunswick | New Jersey | 08901 | United States |
| Rutgers Cancer Institute of New Jersey | New Brunswick | New Jersey | 08903-2681 | United States |
| Montefiore Medical Center | The Bronx | New York | 10467 | United States |
| Levine Cancer Institute | Charlotte | North Carolina | 28204 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Parkland Health & Hospital System | Dallas | Texas | 75235 | United States |
| UT Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| University of Texas Health Science Center at Houston | Houston | Texas | 77030 | United States |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000628792 | voxelotor |
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