Sickle Cell DiseaseRETRONCT04930328
Retrospective Real World Oxbryta® Data Collection and Analysis Study
An observational study of Sickle Cell Disease, run by Pfizer at 12 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Oxbryta® (voxelotor) 500-mg Tablets
- Type of treatment
- Pill or oral medicine
- How many people
- 216 participants
- Where
- 12 sitesUnited States
- Study dates
- 20 Mar 2021to 25 Feb 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.
Who may be able to join
- 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
Who cannot join
- -
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Levine Cancer Institute
Charlotte, North Carolina, 28204
Parkland Health & Hospital System
Dallas, Texas, 75235
UT Southwestern Medical Center
Dallas, Texas, 75390
Central Michigan University/Children's Hospital of Michigan
Detroit, Michigan, 48201
Duke University Medical Center
Durham, North Carolina, 27710
University of Connecticut Health
Farmington, Connecticut, 06030
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Rutgers Robert Wood Johnson Medical School Pediatric Clinical Research Center
New Brunswick, New Jersey, 08901
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903-2681
Montefiore Medical Center
The Bronx, New York, 10467
About this study
Official title: 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.
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Oxbryta® (voxelotor) 500-mg Tablets
- Keywords
- Sickle Cell Disease Registry
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oxbryta® (voxelotor) 500-mg TabletsDrug
Also called Voxelotor, Oxbryta®
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.
| Study group | Role | What they receive |
|---|---|---|
| Retrospective Data Collection | Retrospective Data Collection Receives: Drug: Oxbryta® (voxelotor) 500-mg Tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from pre-Oxbryta treatment period in Hemoglobin (Hb)
Measured: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in percent Reticulocytes
Measured: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Absolute Reticulocytes
Measured: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Bilirubin
Measured: 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)
Measured: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits
Measured: 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
Measured: 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)
Measured: 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
Measured: 1 year before and 1 year after the first dose of Oxbryta
Incidence and severity of serious adverse events (SAEs)
Measured: 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
Measured: 1 year before and 1 year after the first dose of Oxbryta
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2021
- Study start20 Mar 2021
- Primary completion25 Feb 2022
- Study completion25 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 216 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04930328
- Sponsor's protocol ID
- GBT440-4R1
- Other IDs
- C5341018
- Acronym
- RETRO
- Status last verified
- Mar 2024
- First submitted
- 4 Jun 2021
- Last updated
- 6 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
Publications
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.
PubMed 41701977
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Data Collection and Analysis Study of Patients With Sickle Cell Disease (SCD) Who Have Been Treated With Oxbryta® (Voxelotor)
- ClinicalTrials.gov ID
- NCT04930328
- Acronym / sponsor ID
- RETRO / GBT440-4R1
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell Disease Registry
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 216 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 20 Mar 2021 (actual)
- Primary completion
- 25 Feb 2022 (actual)
- Study completion
- 25 Feb 2022 (actual)
- First posted
- 18 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Retrospective Data Collection | – | Drug: Oxbryta® (voxelotor) 500-mg TabletsRetrospective Data Collection |
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Oxbryta® (voxelotor) 500-mg TabletsAlso: Voxelotor, Oxbryta®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. | Drug | Retrospective Data Collection |
Primary outcome
Change from pre-Oxbryta treatment period in Hemoglobin (Hb)
Time frame: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in percent Reticulocytes
Time frame: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Absolute Reticulocytes
Time frame: 1 year before and 1 year after the first dose of Oxbryta
Change from pre-Oxbryta treatment period in Bilirubin
Time frame: 1 year before and 1 year after the first dose of Oxbryta
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)
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
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
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
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
Incidence and severity of serious adverse events (SAEs)
Time frame: 1 year before and 1 year after the first dose of Oxbryta
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2024.
- Derived from this study: PubMed 41701977Andemariam 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.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: No. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Pfizer at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from pre-Oxbryta treatment period in Hemoglobin (Hb) (time frame: 1 year before and 1 year after the first dose of Oxbryta). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04930328, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Mar 2024.