Clinical trial NCT05878704: Study of GBT021601 in Participants With Renal Impairment
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Renal Impairment
Keywords: End stage renal disease
Sickle Cell Disease
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2023-05-26
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Renal Impairment study of 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Concentration of GBT021601 in whole blood and plasma
timeFrame: Up to 112 Days
measure: Single-dose PK parameters
timeFrame: Up to 112 Days
Secondary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs), clinically significant changes in laboratory assessments, electrocardiograms (ECGs), and vital signs
timeFrame: Up to 112 Days
measure: Incidence of treatment-emergent adverse events (TEAEs), clinically significant changes in laboratory assessments, electrocardiograms (ECGs), and vital signs
timeFrame: Up to 112 Days
Other outcomes:
measure: Single-dose PK parameters for GBT021601 in RBC
timeFrame: Up to 112 Days
measure: Amount of GBT021601 excreted in urine
timeFrame: Up to 112 Days
measure: Descriptive analysis of eGFR versus PK exposure parameters
timeFrame: Up to 112 Days
measure: Protein binding of GBT021601 in plasma
timeFrame: Up to 112 Days
measure: Single-dose PK parameters for GBT021601 in RBC
timeFrame: Up to 112 Days
measure: Extraction ratio of GBT021601 via dialysis and concentration of GBT021601 in dialysate
timeFrame: Up to 112 Days
Locations:
city: Lake Forest
country: United States
facility: Orange County Research Center
state: California
city: Tustin
country: United States
facility: Orange County Research Center
state: California
city: Miami
country: United States
facility: Advanced Pharma CR, LLC
state: Florida
city: Saint Paul
country: United States
facility: Nucleus Network
state: Minnesota
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: