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Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD (PROSPECT)

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ClinicalTrials.gov Identifier: NCT04930445
Recruitment Status : Recruiting
First Posted : June 18, 2021
Last Update Posted : March 25, 2022
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
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Brief Summary:
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.

Condition or disease Intervention/treatment
Sickle Cell Disease Drug: Oxbryta® (voxelotor) 500mg Tablets

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.

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 1000 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)
Actual Study Start Date : November 12, 2021
Estimated Primary Completion Date : December 31, 2028
Estimated Study Completion Date : December 31, 2028

Resource links provided by the National Library of Medicine

Drug Information available for: Voxelotor

Groups and Cohorts
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Group/Cohort Intervention/treatment
Oxbryta Product Registry Drug: Oxbryta® (voxelotor) 500mg Tablets
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. There are no pre-defined treatment requirements.
Other Names:
  • Voxelotor
  • Oxbryta®


Outcome Measures
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Primary Outcome Measures :
  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, 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


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04930445


Contacts
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Contact: Michelle Xu, MD 650-534-2574 mxu@gbt.com
Contact: Eleanor Sales esales@gbt.com

Locations
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United States, New York
Montefiore Medical Center Recruiting
Bronx, New York, United States, 10467
Contact: Shuo You    678-596-5183    shuo.you@einsteinmed.org   
Principal Investigator: Susanna Curtis, MD         
United States, North Carolina
Levine Cancer Institute - Atrium Health Recruiting
Charlotte, North Carolina, United States, 28204
Contact: Alicia Patrick    980-292-1746    Alicia.Patrick@atriumhealth.org   
Principal Investigator: Daniel McMahon, MD         
Duke University Hospital Recruiting
Durham, North Carolina, United States, 27710
Contact: Stephanie Padrick    919-684-2406    stephanie.padrick@duke.edu   
Principal Investigator: Nirmish Shah, MD         
East Carolina University - Brody School of Medicine Recruiting
Greenville, North Carolina, United States, 27834
Contact: Ramatu Samura Pham    252-744-2534    SAMURAR18@ECU.EDU   
Principal Investigator: Darla Liles, MD         
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: David Purdie, PhD Global Blood Therapeutics, Inc.
More Information
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT04930445    
Other Study ID Numbers: GBT440-4R2
First Posted: June 18, 2021    Key Record Dates
Last Update Posted: March 25, 2022
Last Verified: February 2022

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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
Keywords provided by Global Blood Therapeutics:
Registry Sickle Cell Disease
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn