Study of GBT021601 in Participants With Renal Impairment

Study Overview
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Brief Summary
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Renal Impairment study of GBT021601.

Detailed Description
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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.

Official Title
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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

Conditions
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Renal Impairment

Intervention / Treatment
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* Drug: GBT021601


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment<br>GBT021601 | Drug: GBT021601<br>* Oral<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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End stage renal disease, Sickle Cell Disease