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

• ClinicalTrials.gov Identifier: NCT05632354
• Recruitment Status: Recruiting
• First Posted: November 30, 2022
• Last Update Posted: April 8, 2024
• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

Study Description

Brief Summary:

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

Condition or disease	Intervention/treatment	Phase
Sickle Cell Disease	Drug: open-label GBT021601	Phase 2; Phase 3

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 314 participants
• Allocation: N/A
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: January 5, 2023
• Estimated Primary Completion Date: April 11, 2029
• Estimated Study Completion Date: April 11, 2029

Arms and Interventions

Arm	Intervention/treatment
Experimental: Treatment; open-label GBT021601	Drug: open-label GBT021601; open-label GBT021601

Outcome Measures

Primary Outcome Measures :

1. To evaluate the incidence of SCD treatment-emergent adverse events with the daily dosing of GBT021601 in participants with sickle cell disease [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Incidence of treatment-emergent adverse events.
2. To evaluate the effects of long-term use of GBT021601 on hemolytic anemia. [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in hematological laboratory parameters.
3. To evaluate the long-term effects of GBT021601 treatment on inflammation [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in adhesion of whole blood to microfluidic channels.
4. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) assessments [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in QOL assessments including Patient Global Impression of Change (PGI-C).
5. To evaluate the change from baseline in hemoglobin [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change in hemoglobin of daily dosing of GBT021601 in participants with SCD
6. To evaluate the change from baseline in reticulocytes [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change in reticulocytes from baseline of daily dosing of GBT021601 in participants with SCD
7. To evaluate the long-term effects of GBT021601 treatment on inflammation [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in adhesion of white blood cells to microfluidic channels.
8. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in QOL assessments including Clinical Global Impression of Change (CGI-C).
9. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Change from baseline in QOL assessments including Patient Global Impression of Severity (PGI-S).
10. To evaluate the long-term effects of GBT021601 treatment on quality of life (QOL) [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
    Change from baseline in QOL assessments including Clinical Global Impression of Severity (CGI-S).
11. To evaluate change from baseline in lactate dehydrogenase [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
    Changes in lactate dehydrogenase from baseline of daily dosing of GBT021601 in participants with SCD
12. To evaluate change from baseline in unconjugated bilirubin [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
    Changes in unconjugated bilirubin from baseline of daily dosing of GBT021601 in participants with SCD

Secondary Outcome Measures :

1. To evaluate the pharmacokinetic parameters of long-term exposure to GBT021601 [ Time Frame: Outcome measures will be evaluated from time of enrollment every 12 weeks through end of treatment up to 4 years ]
   Trough and 1-to 2-hour post dose blood and plasma concentrations of GBT021601.

Eligibility Criteria

• Ages Eligible for Study: 6 Months and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Contacts

Contact: Pfizer Pfizer CT.gov Call Center; 1-800-718-1021; ClinicalTrials.gov_Inquiries@pfizer.com

Locations

United States, Florida

Edward Jenner Research Group LLC.; Not yet recruiting

Miami, Florida, United States, 33317

United States, Louisiana

Our Lady of the Lake Hospital, Inc.; Recruiting

Baton Rouge, Louisiana, United States, 70808

University Medical Center Inpatient Pharmacy; Recruiting

New Orleans, Louisiana, United States, 70112

University Medical Center New Orleans; Recruiting

New Orleans, Louisiana, United States, 70112

University Medical Center New Orleans; Enrolling by invitation

New Orleans, Louisiana, United States, 70112

United States, Mississippi

Mississippi Center for Advanced Medicine; Recruiting

Madison, Mississippi, United States, 39110

United States, Texas

University of Texas Health Science Center; Recruiting

Houston, Texas, United States, 77030

United States, Virginia

Inova Schar Cancer Institute; Recruiting

Fairfax, Virginia, United States, 22031

Nigeria

College of Medicine, University of Ibadan; Recruiting

Ibadan, OYO State, Nigeria, 200212

University College Hospital Ibadan; Recruiting

Ibadan, Oyo/ibadan North, Nigeria, 200212

Aminu kano Teaching Hospital; Recruiting

Kano, Nigeria, 700233

Lagos University Teaching Hospital; Recruiting

Lagos, Nigeria, 100254

Sponsors and Collaborators

Pfizer

Investigators

• Study Director: Pfizer Pfizer CT.gov Call Center; Pfizer

More Information

Additional Information:

To obtain contact information for a study center near you, click here. 

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT05632354
• Other Study ID Numbers: GBT021601-022; C5351005 ( Other Identifier: Alias Study Number )
• First Posted: November 30, 2022
• Last Update Posted: April 8, 2024
• Last Verified: April 2024

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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.
• URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn