A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.
Overview
- Phase
- Phase 1
- Intervention
- GBT021601
- Conditions
- Sickle Cell Disease
- Sponsor
- Pfizer
- Enrollment
- 129
- Locations
- 4
- Primary Endpoint
- Safety, as assessed by frequency and severity of adverse events (AEs)
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
Detailed Description
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Healthy males and females ≥ 18 to ≤ 55 years of age
- •Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
- •Body weight ≥ 50 kg at screening and Day -1
Exclusion Criteria
- •Positive pregnancy test or currently breastfeeding.
Arms & Interventions
Placebo
Placebo as a tablet or capsule with dose based off of preceding cohort's data.
Intervention: GBT021601
GBT021601
GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.
Intervention: GBT021601
Outcomes
Primary Outcomes
Safety, as assessed by frequency and severity of adverse events (AEs)
Time Frame: 119 days from screening Part A, 134 days from screening Part B
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Safety, as assessed by changes in Heart Rate.
Time Frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in heart rate (bpm) as compared to baseline.
Safety, as assessed by changes in eGFR
Time Frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in eGFR from baseline
Safety, as assessed by changes in alanine aminotransferase (ALT)
Time Frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in alanine aminotransferase (ALT)
Safety, as assessed by changes in Blood pressure
Time Frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Plasma concentration
Time Frame: 134 days from screening Part B
Cmax on D1-D15
Secondary Outcomes
- Determine whole blood concentration of GBT021601(119 days from screening Part A)
- Safety, as assessed by changes in QTcF(119 days from screening Part A, 134 days from screening Part B)
- Determine plasma concentration of GBT021601.(134 days from screening Part B)