Phase 1
N=6
A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)
Sickle Cell Disease
Bottom Line
View on ClinicalTrials.gov: NCT04983264 ↗Enrolled (actual)
6
Serious AEs
6.3%
Results posted
Jun 2024
Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 1; 3; 1; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- GBT021601 (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pfizer
- Primary completion
- Dec 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
1; 3; 1; 0; 2; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Physical Examination Findings |
0; 0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Laboratory Parameters |
1; 0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Vital Signs |
0; 0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) |
1; 0; 0 | — |
| SECONDARY Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
1.01; 16.2; 69.2 | — |
| SECONDARY Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B |
2.46; 1.28; 1.54; 1.17; 1.17; 5.15 | — |
| SECONDARY Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C |
2.66; 4.25; 166; 175; 531; 540 | — |
| SECONDARY Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B |
0.97; 0.98; 0.90; 0.884; 0.90; 1.94 | — |
| SECONDARY Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C |
1.54; 1.99; 122; 140; 392; 433 | — |
| SECONDARY Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
1.50; 8.06; 8.06 | — |
| SECONDARY Area Under the Concentration Time Curve From Time Zero to the Next Dose (AUC0-tau) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
67.102; 4597.8; 19627.6 | — |
| SECONDARY Area Under the Serum Concentration Time Curve From Time Zero Extrapolated to Infinite Time (AUCinf) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
72.03; 4914.50; 234.08 | — |
| SECONDARY Area Under the Concentration Time Curve From Time Zero up to Time 24 Hours (AUC 0-24) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
7.94; 322.0; 1363.3 | — |
| SECONDARY Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
1.39; 0.02; 0.004 | — |
| SECONDARY Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
236.191; 230.908; 231.272 | — |
| SECONDARY Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A |
480; 6.87; 1.61 | — |
| SECONDARY Percentage Hemoglobin Occupancy |
3.36; 8.15; 18.15; 13.40; 15.50; 14.03 | — |
| SECONDARY Plasma Concentrations Versus Time Summary of GBT021601: Part A |
0; 0.145; 0.617; 0.910; 0.821; 0.498 | — |
| SECONDARY Plasma Concentrations Versus Time Summary of GBT021601: Part B |
0.143; 1.163; 2.345; 1.015; 1.148; 1.250 | — |
| SECONDARY Plasma Concentrations Versus Time Summary of GBT021601: Part C |
2.448; 2.991; 3.621; 4.285; 4.485; 0.364 | — |
| SECONDARY Whole Blood Concentrations Versus Time Summary of GBT021601: Part A |
0; 1.034; 4.775; 8.745; 12.710; 14.07 | — |
| SECONDARY Whole Blood Concentrations Versus Time Summary of GBT021601: Part B |
0.071; 13.20; 41.23; 74.32; 88.23; 74.87 | — |
| SECONDARY Whole Blood Concentrations Versus Time Summary of GBT021601: Part C |
202.2; 193.6; 214.8; 213.5; 229.9; 33.50 | — |
| SECONDARY Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A |
0; 3.989; 18.68; 34.84; 52.51; 59.50 | — |
| SECONDARY Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B |
0.120; 52.57; 170.8; 317.4; 375.4; 318.6 | — |
| SECONDARY Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C |
621.3; 594.4; 642.4; 639.8; 691.1; 117.9 | — |
Summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses.
Eligibility Criteria
Inclusion Criteria
- Male or Female with SCD
- Participants with SCD ages 18 to 60 years, inclusive.
- Participant has provided documented informed consent.
- Patients with stable and close to baseline hemoglobin value
- Patients on HU should be on stable dose for at least 90 days prior to signing ICF
Exclusion Criteria
- Patients had more than 10 VOC within 12 months of screening
- Patients who are pregnant or nursing
- Patients who receive RBC transfusion therapy regularly or received an RBC transfusion for any reason within 60 days of signing the ICF
- Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF or within 24 days prior to Day 1 treatment
Data sourced from ClinicalTrials.gov (NCT04983264). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.