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| ID | Type | Description | Link |
|---|---|---|---|
| C5351001 | Other Identifier | Pfizer |
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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.
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Placebo as a tablet or capsule with dose based off of preceding cohort's data. |
|
| GBT021601 | Experimental | GBT021601 as a tablet or capsule with dose based off of preceding cohort's data. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GBT021601 | Drug | Administered orally with water as a single dose in the morning. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety, as assessed by frequency and severity of adverse events (AEs) | AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized. | 119 days from screening Part A, 134 days from screening Part B |
| Safety, as assessed by changes in Heart Rate. | Number of participants with changes in heart rate (bpm) as compared to baseline. | 119 days from screening Part A, 134 days from screening Part B |
| Safety, as assessed by changes in eGFR | Number of participants with changes in eGFR from baseline | 119 days from screening Part A, 134 days from screening Part B |
| Safety, as assessed by changes in alanine aminotransferase (ALT) | Number of participants with changes in alanine aminotransferase (ALT) | 119 days from screening Part A, 134 days from screening Part B |
| Safety, as assessed by changes in Blood pressure | Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood | 119 days from screening Part A, 134 days from screening Part B |
| Plasma concentration | Time of Cmax | 119 days from screening Part A |
| Plasma concentration | Cmax on D1-D15 | 134 days from screening Part B |
| Measure | Description | Time Frame |
|---|---|---|
| Determine whole blood concentration of GBT021601 | Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb. | 119 days from screening Part A |
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Inclusion Criteria:
Exclusion Criteria:
- Positive pregnancy test or currently breastfeeding.
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| ICON Early Phase Services, LLC | San Antonio | Texas | 78209 | United States | ||
| Harry Perkins Institute of Medical Research |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| Determine plasma concentration of GBT021601. |
With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax). |
| 134 days from screening Part B |
| Safety, as assessed by changes in QTcF | Number of participants with changes in the QTcF interval from baseline | 119 days from screening Part A, 134 days from screening Part B |
| Nedlands |
| Western Australia |
| 6009 |
| Australia |
| Linear Clinical Research | Nedlands | Western Australia | 6009 | Australia |
| Oxford Compounding | North Perth | Western Australia | 6006 | Australia |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |