Back to resultsGBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
Primary Purpose
Sickle Cell Disease
Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
open-label GBT021601
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease
Eligibility Criteria
Inclusion Criteria: Male or female age 6 months or older with SCD who participated and received study drug or placebo in the previous GBT-Sponsored GBT021601 clinical study and remained on the previous study within 30 calendar days of the Day 1 visit for Study GBT021601-022. Note: Participants who discontinued study drug in the originating study due to an TEAE, but who remained on study, may be eligible for treatment in this study provided the TEAE does not pose a risk for treatment with GBT021601. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Female participants who become of childbearing potential during the study must be willing to have negative urine pregnancy tests to remain in the study. If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 120 days after the last dose of study drug. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements. Exclusion Criteria: Participant withdrew consent or was noncompliant from the originating GBT021601 clinical study. Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1.
Sites / Locations
- University Medical Center New Orleans
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment
Arm Description
open-label GBT021601
Outcomes
Primary Outcome Measures
To evaluate the incidence of SCD treatment-emergent adverse events with the daily dosing of GBT021601 in participants with sickle cell disease
Incidence of treatment-emergent adverse events.
To evaluate the effects of long-term use of GBT021601 on hemolytic anemia.
Change from baseline in hematological laboratory parameters.
To evaluate the long-term effects of GBT021601 treatment on inflammation
Change from baseline in adhesion of whole blood to microfluidic channels.
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) assessments
Change from baseline in QOL assessments including Patient Global Impression of Change (PGI-C).
To evaluate the change from baseline in hemoglobin
Change in hemoglobin of daily dosing of GBT021601 in participants with SCD
To evaluate the change from baseline in reticulocytes
Change in reticulocytes from baseline of daily dosing of GBT021601 in participants with SCD
To evaluate the long-term effects of GBT021601 treatment on inflammation
Change from baseline in adhesion of white blood cells to microfluidic channels.
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Change from baseline in QOL assessments including Clinical Global Impression of Change (CGI-C).
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Change from baseline in QOL assessments including Patient Global Impression of Severity (PGI-S).
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Change from baseline in QOL assessments including Clinical Global Impression of Severity (CGI-S).
To evaluate change from baseline in lactate dehydrogenase
Changes in lactate dehydrogenase from baseline of daily dosing of GBT021601 in participants with SCD
To evaluate change from baseline in unconjugated bilirubin
Changes in unconjugated bilirubin from baseline of daily dosing of GBT021601 in participants with SCD
Secondary Outcome Measures
To evaluate the pharmacokinetic parameters of long-term exposure to GBT021601
Trough and 1-to 2-hour post dose blood and plasma concentrations of GBT021601.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05632354
Brief Title
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
Official Title
An Open-label Extension Study to Evaluate the Long-term Safety of GBT021601 Administered to Participants With Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
January 5, 2023 (Actual)
Primary Completion Date
May 31, 2029 (Anticipated)
Study Completion Date
May 31, 2029 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease
Detailed Description
An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of GBT021601 Administered to Participants with Sickle Cell Disease Who Have Participated in a GBT021601 Clinical Trial
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
500 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Treatment
Arm Type
Experimental
Arm Description
open-label GBT021601
Intervention Type
Drug
Intervention Name(s)
open-label GBT021601
Intervention Description
open-label GBT021601
Primary Outcome Measure Information:
Title
To evaluate the incidence of SCD treatment-emergent adverse events with the daily dosing of GBT021601 in participants with sickle cell disease
Description
Incidence of treatment-emergent adverse events.
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the effects of long-term use of GBT021601 on hemolytic anemia.
Description
Change from baseline in hematological laboratory parameters.
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on inflammation
Description
Change from baseline in adhesion of whole blood to microfluidic channels.
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) assessments
Description
Change from baseline in QOL assessments including Patient Global Impression of Change (PGI-C).
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the change from baseline in hemoglobin
Description
Change in hemoglobin of daily dosing of GBT021601 in participants with SCD
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the change from baseline in reticulocytes
Description
Change in reticulocytes from baseline of daily dosing of GBT021601 in participants with SCD
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on inflammation
Description
Change from baseline in adhesion of white blood cells to microfluidic channels.
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Description
Change from baseline in QOL assessments including Clinical Global Impression of Change (CGI-C).
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Description
Change from baseline in QOL assessments including Patient Global Impression of Severity (PGI-S).
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL)
Description
Change from baseline in QOL assessments including Clinical Global Impression of Severity (CGI-S).
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate change from baseline in lactate dehydrogenase
Description
Changes in lactate dehydrogenase from baseline of daily dosing of GBT021601 in participants with SCD
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Title
To evaluate change from baseline in unconjugated bilirubin
Description
Changes in unconjugated bilirubin from baseline of daily dosing of GBT021601 in participants with SCD
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Secondary Outcome Measure Information:
Title
To evaluate the pharmacokinetic parameters of long-term exposure to GBT021601
Description
Trough and 1-to 2-hour post dose blood and plasma concentrations of GBT021601.
Time Frame
Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female age 6 months or older with SCD who participated and received study drug or placebo in the previous GBT-Sponsored GBT021601 clinical study and remained on the previous study within 30 calendar days of the Day 1 visit for Study GBT021601-022.
Note: Participants who discontinued study drug in the originating study due to an TEAE, but who remained on study, may be eligible for treatment in this study provided the TEAE does not pose a risk for treatment with GBT021601.
Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.
Note: Female participants who become of childbearing potential during the study must be willing to have negative urine pregnancy tests to remain in the study.
If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 120 days after the last dose of study drug.
Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements.
Exclusion Criteria:
Participant withdrew consent or was noncompliant from the originating GBT021601 clinical study.
Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
University Medical Center New Orleans
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT021601-022
Description
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Learn more about this trial
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
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