A GBT021601 ADME Microtracer Study in Healthy Volunteers

A PHASE 1 STUDY TO ASSESS THE MASS BALANCE, EXCRETION, AND PHARMACOKINETICS OF [14C]-GBT021601, AN ORAL HEMOGLOBIN SPOLYMERIZATION INHIBITOR, IN HEALTHY PARTICIPANTS

An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: GBT021601

Detailed Description

This is an open-label Study administering GBT021601 as a single oral dose of 200 mg in healthy participants.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• Netherlands
  • Groningen, Netherlands, 9728
    • ICON Early Phase Clinic, LLC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Body mass index (BMI): 18.0 to 27.0 kg/m2, inclusive, at screening.
• Body weight ≥ 50 kg at screening
• Females must be nonlactating and nonpregnant (as confirmed by a negative serum pregnancy test at screening and admission for all females), or of nonchildbearing potential (ie, either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1 year postmenopausal [defined as at least 12 months no menses, and confirmed by a follicle-stimulating hormone test, at screening]).
• Creatinine clearance (glomerular filtration rate) as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula: ≥90 mL/min, at screening.

Exclusion Criteria:

• History or presence of conditions which, in the opinion of the Investigator, are known to interfere with the ADME of drugs, such as previous surgery on the gastrointestinal tract (including removal of parts of the stomach, bowel, liver, gall bladder, or pancreas). Participants who have a history of appendectomy are eligible for enrollment.
• History of chronic constipation, or recent complaints of an irregular defecation
• Significant and/or acute illness at screening or within 5 days prior to study drug administration that may impact safety assessments, in the opinion of the Investigator.
• Known personal or family history of congenital long QT syndrome or known family history of sudden death.
• Participation in another ADME study with a radiation burden >0.1 mSv in the period of 1 year prior to screening.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment; open-label GBT021601	Drug: GBT021601; Single oral dose of 200 mg GBT021601, containing ~74 kBq (~2 µCi) of [14C]-GBT021601

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the whole blood and plasma concentrations of 14C-GBT021601 total radioactivity.	Total radioactivity pharmacokinetics concentration over time profiles.	Day 1 to Day 206, maximum
To assess the mass balance by determining 14C-GBT021601 total radioactivity excreted in urine and feces.	Excretion of total radioactivity in urine and feces.	Day 1 to Day 206, maximum
To determine the pharmacokinetics of GBT021601 in whole blood, plasma, and urine.	GBT021601 pharmacokinetics concentration over time profiles in whole blood and plasma. Amount of GBT021601 excreted in urine.	Day 1 to Day 206, maximum

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To assess the safety and tolerability of GBT021601 administration in healthy participants.	Number of participants with adverse events	Baseline to Day 206, maximum
To characterize and identify metabolites of 14C-GBT021601 in whole blood, plasma, urine, and feces.	Identification of metabolites in whole blood, plasma, urine, and feces.	Day 1 to Day 206, maximum

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): December 22, 2022
• Primary Completion (Actual): August 10, 2023
• Study Completion (Actual): August 10, 2023

Study Registration Dates

• First Submitted: January 30, 2023
• First Submitted That Met QC Criteria: January 30, 2023
• First Posted (Actual): February 8, 2023

Study Record Updates

• Last Update Posted (Estimated): February 29, 2024
• Last Update Submitted That Met QC Criteria: February 27, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: GBT021601-013; C5351003 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No