GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05632354
• Other study ID #: GBT021601-022
• Secondary ID: C5351005
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: January 5, 2023
• Est. completion date: April 11, 2029

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

An Open-label Extension Study of GBT021601 in Participants with Sickle Cell Disease

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 314
• Est. completion date: April 11, 2029
• Est. primary completion date: April 11, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months and older

Eligibility

Inclusion Criteria:

1. 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.

2. 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.

3. 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.

4. 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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• open-label GBT021601
open-label GBT021601

Locations

Country	Name	City	State
Nigeria	College of Medicine, University of Ibadan	Ibadan	OYO State
Nigeria	University College Hospital Ibadan	Ibadan	Oyo/ibadan North
Nigeria	Aminu kano Teaching Hospital	Kano
Nigeria	Lagos University Teaching Hospital	Lagos
United States	Our Lady of the Lake Hospital, Inc.	Baton Rouge	Louisiana
United States	Inova Schar Cancer Institute	Fairfax	Virginia
United States	University of Texas Health Science Center	Houston	Texas
United States	Mississippi Center for Advanced Medicine	Madison	Mississippi
United States	Edward Jenner Research Group LLC.	Miami	Florida
United States	University Medical Center Inpatient Pharmacy	New Orleans	Louisiana
United States	University Medical Center New Orleans	New Orleans	Louisiana
United States	University Medical Center New Orleans	New Orleans	Louisiana

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Nigeria, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate the effects of long-term use of GBT021601 on hemolytic anemia.	Change from baseline in hematological laboratory parameters.	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate the long-term effects of GBT021601 treatment on inflammation	Change from baseline in adhesion of whole blood to microfluidic channels.	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	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).	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate the change from baseline in hemoglobin	Change in hemoglobin of daily dosing of GBT021601 in participants with SCD	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate the change from baseline in reticulocytes	Change in reticulocytes from baseline of daily dosing of GBT021601 in participants with SCD	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate the long-term effects of GBT021601 treatment on inflammation	Change from baseline in adhesion of white blood cells to microfluidic channels.	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	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).	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	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).	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	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).	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate change from baseline in lactate dehydrogenase	Changes in lactate dehydrogenase from baseline of daily dosing of GBT021601 in participants with SCD	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Primary	To evaluate change from baseline in unconjugated bilirubin	Changes in unconjugated bilirubin from baseline of daily dosing of GBT021601 in participants with SCD	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years
Secondary	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.	Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years

External Links

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