Study of GBT021601 in Participants With Renal Impairment

A Phase I, Open-Label, Single-Dose, Non-Randomized, Parallel Group Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of GBT021601, an Oral Hemoglobin S Polymerization Inhibitor, in Adult Participants With Renal Impairment

Renal Impairment study of GBT021601.

Study Overview

• Status: Terminated
• Conditions: Renal Impairment
• Intervention / Treatment: Drug: GBT021601

Detailed Description

This is a Phase 1, two-part, non-randomized, open-label, parallel group study to evaluate the PK, safety, and tolerability of GBT021601 following a single dose administration in adult participants with RI.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Lake Forest, California, United States, 92630
      • Orange County Research Center
    • Tustin, California, United States, 92780
      • Orange County Research Center
  • Florida
    • Miami, Florida, United States, 33147
      • Advanced Pharma CR, LLC
  • Minnesota
    • Saint Paul, Minnesota, United States, 55114
      • Nucleus Network

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Males or females, ≥ 18 years of age at the time of Screening.
• Have liver (alanine aminotransferase [ALT], aspartate aminotransferase [AST], and total bilirubin [direct and indirect]) and renal function tests, Hb, and hematocrit values within normal limits or are not clinically significant at Screening and Day -1 (Cohort 2 only).
• Has a stable renal function with no clinically significant change in renal status at least 1 month prior to study drug administration.

Exclusion Criteria:

• Any clinically significant medical history or abnormal findings upon physical examination, or clinical laboratory tests unrelated to their medical condition related to renal impairment, that might confound the results of the study or pose an additional risk in administering study drug to the participant.
• Evidence or history of clinically significant allergic (except for untreated, asymptomatic seasonal allergies at the time of study drug administration), hematological, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, or neurological disease unrelated to renal impairment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Treatment; GBT021601	Drug: GBT021601; Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Concentration of GBT021601 in whole blood and plasma	To evaluate the single-dose whole blood and plasma PK of GBT021601 in participants with renal impairment (RI)	Up to 112 Days
Single-dose PK parameters	To evaluate the single-dose whole blood and plasma PK of GBT021601 in participants with renal impairment	Up to 112 Days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (TEAEs), clinically significant changes in laboratory assessments, electrocardiograms (ECGs), and vital signs	To evaluate the safety and tolerability of single dose GBT021601 in participants with renal impairment	Up to 112 Days
Incidence of treatment-emergent adverse events (TEAEs), clinically significant changes in laboratory assessments, electrocardiograms (ECGs), and vital signs	To evaluate the safety and tolerability of GBT021601 in ESRD participants receiving IHD following a single dose	Up to 112 Days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Single-dose PK parameters for GBT021601 in RBC	To evaluate the single-dose RBC PK of GBT021601 in participants with RI	Up to 112 Days
Amount of GBT021601 excreted in urine	To evaluate the urinary excretion of GBT021601 in participants with RI following a single dose	Up to 112 Days
Descriptive analysis of eGFR versus PK exposure parameters	The relationship of eGFR, as estimated using serum creatinine and serum cystatin C on GBT021601 PK exposure in participants with RI	Up to 112 Days
Protein binding of GBT021601 in plasma	To evaluate the protein binding of GBT021601 in participants with RI	Up to 112 Days
Single-dose PK parameters for GBT021601 in RBC	To evaluate the single-dose RBC PK of GBT021601 in participants with ESRD receiving IHD	Up to 112 Days
Extraction ratio of GBT021601 via dialysis and concentration of GBT021601 in dialysate	To evaluate the clearance of GBT021601 by IHD in participants with ESRD	Up to 112 Days

Collaborators and Investigators

• Sponsor: Pfizer
• Collaborators: Pfizer
• Investigators: Study Director: 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): June 29, 2023
• Primary Completion (Actual): July 29, 2024
• Study Completion (Actual): July 29, 2024

Study Registration Dates

• First Submitted: May 19, 2023
• First Submitted That Met QC Criteria: May 19, 2023
• First Posted (Actual): May 26, 2023

Study Record Updates

• Last Update Posted (Actual): October 15, 2024
• Last Update Submitted That Met QC Criteria: October 11, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: End stage renal disease; Sickle Cell Disease
• Additional Relevant MeSH Terms: Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Renal Insufficiency
• Other Study ID Numbers: GBT021601-014; C5351007 (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