Clinical trial NCT03161015: Single-Dose PK Study of GBT440 in Subjects With Renal Impairment
Official title: A Non-Randomized, Open-Label, Parallel Group, Single-Dose Study to Compare the Pharmacokinetics of GBT440 in Subjects With Renal Impaired Function to Healthy Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Renal Impairment
Lead sponsor: Global Blood Therapeutics
Sponsor class: INDUSTRY
First posted: 2017-05-19
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Multiple-center, nonrandomized, open-label, parallel group study
Primary purpose: OTHER

Brief summary: A Phase 1, multiple center, nonrandomized, open-label, parallel group study of a single oral dose of GBT440 administered in subjects with mild, moderate, or severe renal impairment disease and healthy subjects with normal renal function.

Detailed description: Up to 40 adult subjects will be enrolled. Safety and PK assessments will be performed at selected time points throughout the study.

Eligibility criteria: Inclusion Criteria:

All subjects:

* Males or females, 18 to 80 years old
* Willing and able to give written informed consent

Subjects with renal impairment:

* Severe renal impairment (eGFR \< 30 mL/min/1.73m2, not on dialysis)
* Moderate renal impairment (30 mL/min/1.73m2 = or \< eGFR \< 60 mL/min/1.73m2)
* Mild renal impairment (60 mL/min/1.73m2 = or \< eGFR \< 90 mL/min/1.73m2)

Healthy subjects:

* Match in age, gender and body mass index with renal impaired subjects
* Healthy and without clinically significant abnormalities in vital signs, ECGs, physical exam, clinical laboratory evaluations, medical and surgical history

Exclusion Criteria:

All subjects:

* Participation in another clinical trial of an investigational drug (or medical device) within 30 days of the last dose of investigational drug or 5 half lives whichever is longer, prior to screening, or is currently participating in another trial of an investigational drug (or medical device)
* Any signs or symptoms of acute illness at screening or Day -1
* History or presence of clinically significant allergic, hematological, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, or neurological disease

Subjects with renal impairment:

- History of clinically significant hepatic disease e.g. hepatitis, cirrhosis and or liver enzymes (ALT, AST, GGT and total bilirubin) \> 5 times the upper limit of normal within the past year

Primary outcomes:
measure: Cmax of GBT440 in subjects with severe, moderate, or mild renal impairment versus subjects with normal renal function
timeFrame: 28 days max
measure: Tmax of GBT440 in subjects with severe, moderate, or mild renal impairment versus subjects with normal renal function
timeFrame: 28 days max
measure: AUC of GBT440 in subjects with severe, moderate, or mild renal impairment versus subjects with normal renal function
timeFrame: 28 days max
measure: T1/2 of GBT440 in subjects with severe, moderate, or mild renal impairment versus subjects with normal renal function
timeFrame: 28 days max
Secondary outcomes:
measure: Number of subjects with treatment-related Adverse events
timeFrame: 28 days max
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Orlando
country: United States
facility: OCRC
state: Florida
Study officials:
affiliation: Global Blood Therapeutics
name: Carla Washington
role: STUDY_DIRECTOR
References: