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

• Sponsor: Global Blood Therapeutics (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04930445
• Collaborator: (none)
• Enrollment: 1,000
• Locations: 4
• Anticipated Duration (Months): 85.6
• Patients Per Site: 250
• Patients Per Site Per Month: 2.9

Study Details

Study Description

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	Phase
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

• Study Type: Observational [Patient Registry]
• Anticipated Enrollment: 1000 participants
• Observational Model: Other
• Time Perspective: Prospective
• Official Title: An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)
• Actual Study Start Date: Nov 12, 2021
• Anticipated Primary Completion Date: Dec 31, 2028
• Anticipated Study Completion Date: Dec 31, 2028

Arms and Interventions

Arm	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

Primary Outcome Measures

1. Change from pre-Oxbryta treatment period in Hemoglobin (Hb) [1 year before and 1 year after the first dose of Oxbryta]

2. Change from pre-Oxbryta treatment period in percent Reticulocytes [1 year before and 1 year after the first dose of Oxbryta]

3. Change from pre-Oxbryta treatment period in absolute Reticulocytes [1 year before and 1 year after the first dose of Oxbryta]

4. Change from pre-Oxbryta treatment period in bilirubin [1 year before and 1 year after the first dose of Oxbryta]

5. Incidence of significant SCD-related clinical events [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 [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 [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) [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 [1 year before and 1 year after the first dose of Oxbryta]

10. Incidence and severity of serious adverse events (SAEs) [1 year before and 1 year after the first dose of Oxbryta]

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

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Locations

	Site	City	State	Country	Postal Code
1	Montefiore Medical Center	Bronx	New York	United States	10467
2	Levine Cancer Institute - Atrium Health	Charlotte	North Carolina	United States	28204
3	Duke University Hospital	Durham	North Carolina	United States	27710
4	East Carolina University - Brody School of Medicine	Greenville	North Carolina	United States	27834

Sponsors and Collaborators

• Global Blood Therapeutics

Investigators

• Study Director: David Purdie, PhD, Global Blood Therapeutics, Inc.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Global Blood Therapeutics
• ClinicalTrials.gov Identifier: NCT04930445
• Other Study ID Numbers: GBT440-4R2
• First Posted: Jun 18, 2021
• Last Update Posted: Mar 25, 2022
• Last Verified: Feb 1, 2022

• 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: Anemia, Sickle Cell

Study Results

No Results Posted as of Mar 25, 2022