Brief Summary
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
Brief Title
Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
Detailed Description
The study will be conducted at approximately 45 sites in the United States.
This registry is an observational study to evaluate the effects of Oxbryta in individuals with SCD. Any participant who is currently taking Oxbryta, or has been prescribed and will initiate treatment with Oxbryta, is eligible to participate. Eligible participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. This study will collect data that are recorded in the participants' medical records and other secondary data sources. Study data will be collected at regular intervals and entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. Participants will be considered to be on study for up to 5 years after their first dose of Oxbryta treatment, or until they withdraw their consent to participate, or are discontinued from the study. Treatment, including interruptions and restarting treatment, will continue at the discretion of the treating physician, and there are no pre-defined treatment requirements. Participants may receive any additional medications prescribed by their treating physician, or have any medical interventions that are deemed appropriate by the treating physician or study doctor. The participant or treating physician may discontinue Oxbryta at any time. Participants who discontinue treatment with Oxbryta earlier than 5 years will continue to be followed on study to collect clinical and quality of life (QoL) outcomes for up to 5 years after their first dose of Oxbryta treatment. Participant safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.
This registry is an observational study to evaluate the effects of Oxbryta in individuals with SCD. Any participant who is currently taking Oxbryta, or has been prescribed and will initiate treatment with Oxbryta, is eligible to participate. Eligible participants will receive treatment with Oxbryta as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per standard of care (SOC) and at the physician's discretion. This study will collect data that are recorded in the participants' medical records and other secondary data sources. Study data will be collected at regular intervals and entered in case report forms (CRFs) via an electronic data capture (EDC) system by the study staff. Participants will be considered to be on study for up to 5 years after their first dose of Oxbryta treatment, or until they withdraw their consent to participate, or are discontinued from the study. Treatment, including interruptions and restarting treatment, will continue at the discretion of the treating physician, and there are no pre-defined treatment requirements. Participants may receive any additional medications prescribed by their treating physician, or have any medical interventions that are deemed appropriate by the treating physician or study doctor. The participant or treating physician may discontinue Oxbryta at any time. Participants who discontinue treatment with Oxbryta earlier than 5 years will continue to be followed on study to collect clinical and quality of life (QoL) outcomes for up to 5 years after their first dose of Oxbryta treatment. Participant safety and tolerability will be assessed throughout the study data collection period by the study doctor and reported to the Sponsor.
Categories
Central Contacts
Central Contact Role
Contact
Central Contact Phone
1-800-718-1021
Central Contact Email
ClinicalTrials.gov_Inquiries@pfizer.com
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria:
* Participants who meet all the following criteria will be eligible for enrollment:
1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/ guardian consent and participant assent (aged ≥ 12 to \<18 years) per local regulations, or pediatric participants (aged 4 to \<12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines
2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes)
3. Undergoing treatment with Oxbryta according to the Oxbryta USPI
Exclusion Criteria:
* Participants meeting any of the following criteria will not be eligible for study enrollment:
1. Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment.
2. Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures
* Participants who meet all the following criteria will be eligible for enrollment:
1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/ guardian consent and participant assent (aged ≥ 12 to \<18 years) per local regulations, or pediatric participants (aged 4 to \<12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines
2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes)
3. Undergoing treatment with Oxbryta according to the Oxbryta USPI
Exclusion Criteria:
* Participants meeting any of the following criteria will not be eligible for study enrollment:
1. Current participation in an investigation clinical trial or expanded access program, in which the participant may be receiving voxelotor treatment.
2. Medical, psychological, or behavioral condition that, in the opinion of the study doctor, would confound or interfere with evaluation of safety and/or effectiveness of the study drug, prevent compliance with the study protocol; preclude informed consent; or render the participant unable/unlikely to comply with the study procedures
Inclusion Criteria
Inclusion Criteria:
* Participants who meet all the following criteria will be eligible for enrollment:
1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/ guardian consent and participant assent (aged ≥ 12 to \<18 years) per local regulations, or pediatric participants (aged 4 to \<12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines
2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes)
3. Undergoing treatment with Oxbryta according to the Oxbryta USPI
* Participants who meet all the following criteria will be eligible for enrollment:
1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/ guardian consent and participant assent (aged ≥ 12 to \<18 years) per local regulations, or pediatric participants (aged 4 to \<12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines
2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes)
3. Undergoing treatment with Oxbryta according to the Oxbryta USPI
Gender
All
Gender Based
false
Keywords
Registry Sickle Cell Disease
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
4 Years
NCT Id
NCT04930445
Org Class
Industry
Org Full Name
Pfizer
Org Study Id
GBT440-4R2
Overall Status
Terminated
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)
Primary Outcomes
Outcome Measure
Change from pre-Oxbryta treatment period in Hemoglobin (Hb)
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in percent Reticulocytes
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in absolute Reticulocytes
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in bilirubin
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Description
Such as vaso-occlusive crisis (VOC), acute chest syndrome (ACS), priapism, cerebral infarcts, transient ischemic attack (TIA), leg ulcers, measures of cardiac function and pulmonary hypertension (PH)
Outcome Measure
Incidence of significant SCD-related clinical events
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in incidence of unplanned clinic visits
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in incidence of emergency department (ED) visits
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in incidence of hospitalizations (including total length of stay, and time in intensive care unit [ICU], if applicable)
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Change from pre-Oxbryta treatment period in incidence of red blood cell transfusions
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Measure
Incidence and severity of serious adverse events (SAEs)
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Outcome Description
Such as Rash, Diarrhea, Headache, AEs leading to Oxbryta dose modification or discontinuation
Outcome Measure
Incidence and severity of adverse events (AEs) of interest
Outcome Time Frame
1 year before and 1 year after the first dose of Oxbryta
Secondary Ids
Secondary Id
C5341019
See Also Links
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Study Population
All patients at each participating study site who have been treated with Oxbryta will be considered for inclusion in this study.
Std Ages
Child
Adult
Older Adult
Locked Fields
Render the field
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
4
Investigators
Investigator Type
Principal Investigator
Investigator Name
Henny Billett
Investigator Email
hbillett@montefiore.org
Investigator Phone
718-920-6310