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| ID | Type | Description | Link |
|---|---|---|---|
| C5351002 | Other Identifier | Alias Study Number |
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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.
This is an open-label intrapatient single dose followed by a multiple dose escalation study in at least six (6) participants with SCD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single-dose Period (Part A) | Experimental | Refer to Study Description |
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| Multiple Ascending-dose Period (Part B and Part C) | Experimental | Refer to Study Description |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GBT021601 | Drug | Tablets and capsules which contain GBT021601 drug substance |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. A treatment-emergent AE (TEAE) was an AE that occurs or worsens during the on-treatment period defined as the time from the first dose of study drug through minimum of 56 days after last dose of study treatment. A Serious Adverse Event (SAE) was any untoward medical occurrence at any dose that: resulted in death; was life-threatening experience (immediate risk of death); required inpatient hospitalization or prolongation if existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; was considered an important medical event. TEAEs and SAEs were reported for both Sickle Cell Disease (SCD) and non-SCD related events, in this outcome measure. | From first dose of study drug (Day 1) to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
| Number of Participants With Clinically Significant Physical Examination Findings | Physical examination included were general appearance, head, ears, eyes, nose, throat, neck, skin, cardiovascular system, respiratory system, gastrointestinal system, musculoskeletal system, lymph nodes, and nervous system. Clinically significant physical examination abnormalities were considered as adverse events based on investigator's discretion. | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
| Number of Participants With Clinically Significant Change From Baseline in Laboratory Parameters | Laboratory parameters included hematology(hemoglobin,hematocrit,red blood cell count,platelet count,white blood cell count,total neutrophils,eosinophils,monocytes,basophils,lymphocytes);blood chemistry(blood urea,nitrogen, creatinine,glucose,calcium,sodium,potassium,chloride,total bicarbonate,aspartate aminotransferase,alanine aminotransferase,total bilirubin,alkaline phosphatase,uric acid albumin,total protein);urinalysis(decimal logarithm of reciprocal of hydrogen ion activity [pH], glucose, protein, blood,ketones, microscopy[urine tested positive for blood or protein]),urine drug screening:cannabinoids,amphetamines,methamphetamines,opiates,methadone,cocaine,benzodiazepines,phencyclidine,barbiturates,alcohol breath test. Hemoximetry RBC deformability,dense cells test; erythropoietin,follicle stimulating hormone,pregnancy test,Serology panel for HIV 1/2 antibody,Hepatitis A, B and C,SARS CoV-2.Clinical significance of any parameter was determined at the investigator's discretion. |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Advanced Pharma CR, LLC | Miami | Florida | 33147 | United States | ||
| Visionaries Clinical Research LLC |
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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 | Title | Description |
|---|---|---|
| FG000 | Part A: Single Dose Period | Participants received a single oral dose of GBT021601 100 mg on Day 1. |
| FG001 | Part B: Multiple Ascending- Dose Period | Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants. |
| FG002 | Part C: Extended Treatment Period | Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Part A: Single Dose Period |
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| Part B: Multiple Dose Period |
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| Part C: Extended Treatment Period |
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Safety population was defined as all participants who received any amount of study drug (GBT021601).
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| ID | Title | Description |
|---|---|---|
| BG000 | Overall Study | All participants who received GBT021601 as single dose, multiple dose or extended treatment in Part A, Part B and Part C, respectively were included. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a study participant administered a product or medical device; the event need not necessarily have a causal relationship with the treatment or usage. A treatment-emergent AE (TEAE) was an AE that occurs or worsens during the on-treatment period defined as the time from the first dose of study drug through minimum of 56 days after last dose of study treatment. A Serious Adverse Event (SAE) was any untoward medical occurrence at any dose that: resulted in death; was life-threatening experience (immediate risk of death); required inpatient hospitalization or prolongation if existing hospitalization; resulted in persistent or significant disability/ incapacity; resulted in congenital anomaly/birth defect; was considered an important medical event. TEAEs and SAEs were reported for both Sickle Cell Disease (SCD) and non-SCD related events, in this outcome measure. | Safety population was defined as all participants who received any amount of study drug (GBT021601). | Posted | Count of Participants | Participants | From first dose of study drug (Day 1) to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
From baseline (Day 1) up to at least 56 days after last dose of study drug (for a maximum of 316 days)
Same event may appear as both non-SAE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and non-serious in another participants, or one participant may have experienced both serious and non-serious event during the study. AEs presented were related to both SCD and Non SCD related events.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: Single Dose Period (SCD Related) | Participants received a single oral dose of GBT021601 100 mg on Day 1 and had SCD related adverse events. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 8, 2022 | Dec 6, 2023 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 24, 2023 | Dec 6, 2023 | SAP_001.pdf |
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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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| Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
| Number of Participants With Clinically Significant Change From Baseline in Vital Signs | Vital signs assessments included were systolic and diastolic blood pressure, heart rate, respiratory rate and body temperature. These measurements were taken after the participants had rested for at least 5 minutes in the supine position. Any clinically significant abnormal vital sign assessment required at least one repeat measurement. Clinical significance of any parameter was determined based on investigator's discretion. | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
| Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) | ECG values included here were Heart rate (HR), PR, QRS, QT, and QTcF intervals, interpretation of the tracings (eg, rhythm, presence of arrhythmia or conduction defects, any evidence of myocardial ischemia/infarction, or ST segment, T-wave, and U-wave abnormalities). Abnormal and clinically significant 12-lead ECG included QT interval corrected for heart rate according to Fridericia's formula (QTcF) > 450 millisecond (ms), QRS interval >= 120 ms, PR interval > 220 ms, based on the average of triplicated ECG, assessed at Screening and Day-1. If any of these test results were out of range, then the test could be repeated once (in triplicate). | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
| Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112 |
| Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42 |
| Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B | Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation. | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112 |
| Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C | Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation. | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42 |
| Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Maximum observed concentration was defined as the peak concentration observed directly from the experimental data without any interpolation. The time to the maximum observed concentration was defined as the time corresponding to Cmax. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| 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 | AUC0-tau was defined from time 0 to time of the last quantifiable concentration and was calculated using the linear or logarithmic trapezoid rule. AUCtau was calculated by using hours*microgram per milliliter (hr*mcg)/mL. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| 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 | AUCinf was defined as the area calculated by linear/log trapezoid rule from time 0 to infinity with the area extrapolated from the last quantifiable concentration to infinity. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| 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 | AUC0-24 was defined as the area under the free plasma concentration time curve from time 0 to 24 hours post-dose. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs post-dose on Day 1 |
| Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | CL/F was a quantitative measure of the rate at which a drug substance was removed from the blood. It was calculated as dose of GBT021601 by AUC from time 0 to infinity. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | t1/2 was the time measured for the plasma concentration to decrease by one half. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vz/F was determined based on the fraction absorbed. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (%Hb Occupancy) refers to the proportion of hemoglobin molecules within red blood cells that were bound to study drug (GBT021601). Cmin and Cmax values was used to calculate %Hb occupancy: %Hb Occupancy = GBT021601*RBC per Mean Corpuscular Hemoglobin Concentration (MCHC). | Part A: Pre-dose,0.25,0.5,1,2,4, 6,8,12,24,36,48,72,168,336,504,672,1008 hrs on Day 1; Part B: Pre-dose,0.25 to 1,2 to 4 hrs post-dose on Day 56,63,70,77,84,91,98,105,112; Part C:Pre-dose on Day 1,14,28,42 and 0.25 to 1, 2 to 4 hrs post-dose on Day 28,42 |
| Plasma Concentrations Versus Time Summary of GBT021601: Part A | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Plasma Concentrations Versus Time Summary of GBT021601: Part B | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
| Plasma Concentrations Versus Time Summary of GBT021601: Part C | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
| Whole Blood Concentrations Versus Time Summary of GBT021601: Part A | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Whole Blood Concentrations Versus Time Summary of GBT021601: Part B | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs, post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
| Whole Blood Concentrations Versus Time Summary of GBT021601: Part C | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
| Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A | The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time. | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
| Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B | The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time. | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
| Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C | The RBC concentration versus time summary involved tracking a quantity of RBC in a sample of blood over a specified duration. This analysis helped to monitor RBC level over time. | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
| Atlanta |
| Georgia |
| 30329 |
| United States |
| Children's Healthcare of Atlanta AFLAC Center | Atlanta | Georgia | 30342 | United States |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| ID | Title | Description |
|---|---|---|
| OG000 | Part A: Single Dose Period | Participants received a single oral dose of GBT021601 100 mg on Day 1. |
| OG001 | Part B: Multiple Ascending- Dose Period | Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants. |
| OG002 | Part C: Extended Treatment Period | Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks. |
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| Primary | Number of Participants With Clinically Significant Physical Examination Findings | Physical examination included were general appearance, head, ears, eyes, nose, throat, neck, skin, cardiovascular system, respiratory system, gastrointestinal system, musculoskeletal system, lymph nodes, and nervous system. Clinically significant physical examination abnormalities were considered as adverse events based on investigator's discretion. | Safety population was defined as all participants who received any amount of study drug (GBT021601). | Posted | Count of Participants | Participants | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
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| Primary | Number of Participants With Clinically Significant Change From Baseline in Laboratory Parameters | Laboratory parameters included hematology(hemoglobin,hematocrit,red blood cell count,platelet count,white blood cell count,total neutrophils,eosinophils,monocytes,basophils,lymphocytes);blood chemistry(blood urea,nitrogen, creatinine,glucose,calcium,sodium,potassium,chloride,total bicarbonate,aspartate aminotransferase,alanine aminotransferase,total bilirubin,alkaline phosphatase,uric acid albumin,total protein);urinalysis(decimal logarithm of reciprocal of hydrogen ion activity [pH], glucose, protein, blood,ketones, microscopy[urine tested positive for blood or protein]),urine drug screening:cannabinoids,amphetamines,methamphetamines,opiates,methadone,cocaine,benzodiazepines,phencyclidine,barbiturates,alcohol breath test. Hemoximetry RBC deformability,dense cells test; erythropoietin,follicle stimulating hormone,pregnancy test,Serology panel for HIV 1/2 antibody,Hepatitis A, B and C,SARS CoV-2.Clinical significance of any parameter was determined at the investigator's discretion. | Safety population was defined as all participants who received any amount of study drug (GBT021601). | Posted | Count of Participants | Participants | No | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
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| Primary | Number of Participants With Clinically Significant Change From Baseline in Vital Signs | Vital signs assessments included were systolic and diastolic blood pressure, heart rate, respiratory rate and body temperature. These measurements were taken after the participants had rested for at least 5 minutes in the supine position. Any clinically significant abnormal vital sign assessment required at least one repeat measurement. Clinical significance of any parameter was determined based on investigator's discretion. | Safety population was defined as all participants who received any amount of study drug (GBT021601). | Posted | Count of Participants | Participants | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
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| Primary | Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) | ECG values included here were Heart rate (HR), PR, QRS, QT, and QTcF intervals, interpretation of the tracings (eg, rhythm, presence of arrhythmia or conduction defects, any evidence of myocardial ischemia/infarction, or ST segment, T-wave, and U-wave abnormalities). Abnormal and clinically significant 12-lead ECG included QT interval corrected for heart rate according to Fridericia's formula (QTcF) > 450 millisecond (ms), QRS interval >= 120 ms, PR interval > 220 ms, based on the average of triplicated ECG, assessed at Screening and Day-1. If any of these test results were out of range, then the test could be repeated once (in triplicate). | Safety population was defined as all participants who received any amount of study drug (GBT021601). | Posted | Count of Participants | Participants | Baseline (Day 1) up to at least 56 days after last dose of study drug (up to a maximum of 316 days) |
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| Secondary | Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | Microgram per milliliter (mcg/mL) | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
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| Secondary | Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | mcg/mL | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112 |
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| Secondary | Maximum GBT021601 Concentration (Cmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C | Cmax was defined as the peak concentration observed directly from the experimental data without any interpolation. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | mcg/mL | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42 |
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| Secondary | Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part B | Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | mcg/mL | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112 |
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| Secondary | Minimum GBT021601 Concentration (Cmin) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part C | Cmin was defined as the lowest concentration observed directly from the experimental data without any interpolation. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | mcg/mL | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28 and 42 |
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| Secondary | Time to Attain Maximum Serum Concentration (Tmax) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Maximum observed concentration was defined as the peak concentration observed directly from the experimental data without any interpolation. The time to the maximum observed concentration was defined as the time corresponding to Cmax. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Median | Full Range | Hours | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
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| 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 | AUC0-tau was defined from time 0 to time of the last quantifiable concentration and was calculated using the linear or logarithmic trapezoid rule. AUCtau was calculated by using hours*microgram per milliliter (hr*mcg)/mL. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | (hr*mcg)/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
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| 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 | AUCinf was defined as the area calculated by linear/log trapezoid rule from time 0 to infinity with the area extrapolated from the last quantifiable concentration to infinity. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | (hr*mcg)/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| 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 | AUC0-24 was defined as the area under the free plasma concentration time curve from time 0 to 24 hours post-dose. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | (hr*mcg)/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs post-dose on Day 1 |
|
|
|
| Secondary | Apparent Oral Clearance (CL/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | CL/F was a quantitative measure of the rate at which a drug substance was removed from the blood. It was calculated as dose of GBT021601 by AUC from time 0 to infinity. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | Liter per hour | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Terminal Elimination Half-Life (t1/2) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | t1/2 was the time measured for the plasma concentration to decrease by one half. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Median | Full Range | hr | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Apparent Volume of Distribution (Vz/F) in Plasma, Whole Blood and Red Blood Cells (RBCs): Part A | Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vz/F was determined based on the fraction absorbed. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Geometric Mean | Geometric Coefficient of Variation | Liter | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Percentage Hemoglobin Occupancy | Percentage hemoglobin occupancy (%Hb Occupancy) refers to the proportion of hemoglobin molecules within red blood cells that were bound to study drug (GBT021601). Cmin and Cmax values was used to calculate %Hb occupancy: %Hb Occupancy = GBT021601*RBC per Mean Corpuscular Hemoglobin Concentration (MCHC). | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Mean | Standard Deviation | Percentage of Hemoglobin | Part A: Pre-dose,0.25,0.5,1,2,4, 6,8,12,24,36,48,72,168,336,504,672,1008 hrs on Day 1; Part B: Pre-dose,0.25 to 1,2 to 4 hrs post-dose on Day 56,63,70,77,84,91,98,105,112; Part C:Pre-dose on Day 1,14,28,42 and 0.25 to 1, 2 to 4 hrs post-dose on Day 28,42 |
|
|
|
| Secondary | Plasma Concentrations Versus Time Summary of GBT021601: Part A | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Plasma Concentrations Versus Time Summary of GBT021601: Part B | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
|
|
|
| Secondary | Plasma Concentrations Versus Time Summary of GBT021601: Part C | The plasma concentration versus time summary was measured by the amount of specific substance in the bloodstream over the specified timepoint. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
|
|
|
| Secondary | Whole Blood Concentrations Versus Time Summary of GBT021601: Part A | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Whole Blood Concentrations Versus Time Summary of GBT021601: Part B | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs, post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
|
|
|
| Secondary | Whole Blood Concentrations Versus Time Summary of GBT021601: Part C | The whole blood concentration versus time summary was measured to find concentration of various components in whole blood that changed over a specified period. This helped to monitor the dynamics of blood components such as hemoglobin levels, hematocrit, or glucose concentration over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
|
|
|
| Secondary | Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part A | The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hour (hr), 0.5 hr, 1 hr, 2 hrs, 4 hrs, 6 hrs, 8 hrs,12 hrs, 24 hrs, 36 hrs, 48 hrs, 72 hrs, 168 hrs, 336 hrs, 504 hrs, 672 hrs, 1008 hrs post-dose on Day 1 |
|
|
|
| Secondary | Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part B | The RBC concentration versus time summary was measured to identify quantity of RBC in a sample of blood over a specified duration. This analysis helped monitor RBC level over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose, 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 56, 63, 70, 77, 84, 91, 98, 105, 112, 140,168, 196, 218 |
|
|
|
| Secondary | Red Blood Cell Concentrations Versus Time Summary of GBT021601: Part C | The RBC concentration versus time summary involved tracking a quantity of RBC in a sample of blood over a specified duration. This analysis helped to monitor RBC level over time. | Pharmacokinetic population was defined as all participants who received at least 1 dose of study drug (GBT021601) and had at least 1 plasma or whole blood concentration data point. Here, 'Number Analyzed' signifies participants evaluable for the specified timepoints. | Posted | Mean | Standard Deviation | mcg/mL | Pre-dose on Day 1, 14, 28, 42 and 0.25 hr to 1 hr, 2 to 4 hrs post-dose on Day 28, 42, 70, 98 |
|
|
|
| 0 |
| 6 |
| 0 |
| 6 |
| 1 |
| 6 |
| EG001 | Part B: Multiple Ascending- Dose Period (SCD Related) | Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted % Hb occupancy of participants and had SCD related adverse events. | 0 | 6 | 2 | 6 | 2 | 6 |
| EG002 | Part C: Extended Treatment Period (SCD Related) | Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had SCD related adverse events. | 0 | 4 | 0 | 4 | 1 | 4 |
| EG003 | Part A: Single Dose Period (Non-SCD Related) | Participants received a single oral dose of GBT021601 100 mg on Day 1 and had non-SCD related adverse events. | 0 | 6 | 0 | 6 | 5 | 6 |
| EG004 | Part B: Multiple Ascending- Dose Period (Non-SCD Related) | Participants received a loading dose on Day 56 (Week 8) followed by maintenance dose once weekly for up to 8 weeks. Dose administered in Part B of this study was in between a range of 50 mg to a maximum dose of 500 mg, based on the targeted percentage Hemoglobin (% Hb) occupancy of participants and had non-SCD related adverse events. | 0 | 6 | 0 | 6 | 5 | 6 |
| EG005 | Part C: Extended Treatment Period (Non-SCD Related) | Participants received a loading dose of 300 mg twice daily for 4 days followed by a maintenance dose of 150 mg once daily for up to 6 weeks and had non-SCD related adverse events. | 0 | 4 | 0 | 4 | 3 | 4 |
| Anaemia | Blood and lymphatic system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Thrombocytosis | Blood and lymphatic system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Flatulence | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Hyperbilirubinaemia | Hepatobiliary disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Adenoviral upper respiratory infection | Infections and infestations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Tooth infection | Infections and infestations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Thermal burn | Injury, poisoning and procedural complications | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Blood creatinine increased | Investigations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Electrocardiogram QT prolonged | Investigations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Transaminases increased | Investigations | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Urinary retention | Renal and urinary disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Thrombophlebitis | Vascular disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA Version: 24.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publication until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Plasma- Day 77 |
|
| Plasma- Day 84 |
|
| Plasma- Day 91 |
|
| Plasma- Day 98 |
|
| Plasma- Day 105 |
|
| Plasma- Day 112 |
|
| Whole Blood- Day 56 |
|
| Whole Blood- Day 63 |
|
| Whole Blood- Day 70 |
|
| Whole Blood- Day 77 |
|
| Whole Blood- Day 84 |
|
| Whole Blood- Day 91 |
|
| Whole Blood- Day 98 |
|
| Whole Blood- Day 105 |
|
| Whole Blood- Day 112 |
|
| RBC concentration- Day 56 |
|
| RBC concentration- Day 63 |
|
| RBC concentration- Day 70 |
|
| RBC concentration- Day 77 |
|
| RBC concentration- Day 84 |
|
| RBC concentration- Day 91 |
|
| RBC concentration- Day 98 |
|
| RBC concentration- Day 105 |
|
| RBC concentration- Day 112 |
|
| Title | Measurements |
|---|---|
|
| Whole Blood - Day 42 |
|
| RBC concentration - Day 28 |
|
| RBC concentration - Day 42 |
|
| Title | Measurements |
|---|---|
|
| Plasma- Day 84 |
|
| Plasma- Day 91 |
|
| Plasma- Day 98 |
|
| Plasma- Day 105 |
|
| Plasma- Day 112 |
|
| Whole Blood- Day 63 |
|
| Whole Blood- Day 70 |
|
| Whole Blood- Day 77 |
|
| Whole Blood- Day 84 |
|
| Whole Blood- Day 91 |
|
| Whole Blood- Day 98 |
|
| Whole Blood- Day 105 |
|
| Whole Blood- Day 112 |
|
| RBC concentration- Day 63 |
|
| RBC concentration- Day 70 |
|
| RBC concentration- Day 77 |
|
| RBC concentration- Day 84 |
|
| RBC concentration- Day 91 |
|
| RBC concentration- Day 98 |
|
| RBC concentration- Day 105 |
|
| RBC concentration- Day 112 |
|
| Title | Measurements |
|---|---|
|
| Whole Blood - Day 42 |
|
| RBC concentration - Day 28 |
|
| RBC concentration - Day 42 |
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|
| Part B: Cmax- Day 56 |
|
|
| Part B: Cmax - Day 63 |
|
|
| Part B: Cmin- Day 63 |
|
|
| Part B: Cmax- Day 70 |
|
|
| Part B: Cmin- Day 70 |
|
|
| Part B: Cmax- Day 77 |
|
|
| Part B: Cmin- Day 77 |
|
|
| Part B: Cmax- Day 84 |
|
|
| Part B: Cmin- Day 84 |
|
|
| Part B: Cmax- Day 91 |
|
|
| Part B: Cmin- Day 91 |
|
|
| Part B: Cmax- Day 98 |
|
|
| Part B: Cmin- Day 98 |
|
|
| Part B: Cmax- Day 105 |
|
|
| Part B: Cmin- Day 105 |
|
|
| Part B: Cmax- Day 112 |
|
|
| Part B: Cmin- Day 112 |
|
|
| Part C: Cmax- Day 28 |
|
|
| Part C: Cmin- Day 28 |
|
|
| Part C: Cmax- Day 42 |
|
|
| Part C: Cmin- Day 42 |
|
|
| Title | Measurements |
|---|---|
|
| 1 hour |
|
| 2 hours |
|
| 4 hours |
|
| 6 hours |
|
| 8 hours |
|
| 12 hours |
|
| 24 hours |
|
| 36 hours |
|
| 48 hours |
|
| 72 hours |
|
| 168 hours |
|
| 336 hours |
|
| 504 hours |
|
| 672 hours |
|
| 1008 hours |
|
|
| Day 56: 2-4 hours |
|
|
| Day 63: Pre-dose |
|
|
| Day 63: 0.25-1 hour |
|
|
| Day 63: 2-4 hours |
|
|
| Day 70: Pre-dose |
|
|
| Day 70: 0.25-1 hour |
|
|
| Day 70: 2-4 hours |
|
|
| Day 77: Pre-dose |
|
|
| Day 77: 0.25-1 hour |
|
|
| Day 77: 2-4 hours |
|
|
| Day 84: Pre-dose |
|
|
| Day 84 :0.25-1 hour |
|
|
| Day 84: 2-4 hours |
|
|
| Day 91: Pre-dose |
|
|
| Day 91; 0.25-1 hour |
|
|
| Day 91: 2-4 hours |
|
|
| Day 98: Pre-dose |
|
|
| Day 98: 0.25-1 hour |
|
|
| Day 98: 2-4 hours |
|
|
| Day 105: Pre-dose |
|
|
| Day 105: 0.25-1 hour |
|
|
| Day 105: 2-4 hours |
|
|
| Day 112 :Predose |
|
|
| Day 112: 0.25-1 hour |
|
|
| Day 112: 2-4 hours |
|
|
| Day 140: 2-4 hours |
|
|
| Day 168: 2-4 hours |
|
|
| Day 196: 2-4 hours |
|
|
| Day 218: 2-4 hours |
|
|
|
| Day 42: Pre-dose |
|
|
| Day 42: 0.25-1 hour |
|
|
| Day 42: 2-4 hours |
|
|
| Day 70: 2-4 hours |
|
|
| Day 98: 2-4 hours |
|
|
| Title | Measurements |
|---|---|
|
| 1 hour |
|
| 2 hours |
|
| 4 hours |
|
| 6 hours |
|
| 8 hours |
|
| 12 hours |
|
| 24 hours |
|
| 36 hours |
|
| 48 hours |
|
| 72 hours |
|
| 168 hours |
|
| 336 hours |
|
| 504 hours |
|
| 672 hours |
|
| 1008 hours |
|
|
| Day 56: 2-4 hours |
|
|
| Day 63: Pre-dose |
|
|
| Day 63: 0.25-1 hour |
|
|
| Day 63: 2- 4 hours |
|
|
| Day 70: Pre-dose |
|
|
| Day 70: 0.25-1 hour |
|
|
| Day 70: 2-4 hours |
|
|
| Day 77: Pre-dose |
|
|
| Day 77: 0.25-1 hour |
|
|
| Day 77: 2- 4 hours |
|
|
| Day 84: Predose |
|
|
| Day 84 :0.25-1 hour |
|
|
| Day 84: 2-4 hours |
|
|
| Day 91: Pre-dose |
|
|
| Day 91; 0.25-1 hour |
|
|
| Day 91: 2-4 hours |
|
|
| Day 98: Pre-dose |
|
|
| Day 98: 0.25-1 hour |
|
|
| Day 98: 2-4 hours |
|
|
| Day 105: Pre-dose |
|
|
| Day 105: 0.25-1 hour |
|
|
| Day 105: 2-4 hours |
|
|
| Day 112: Pre-dose |
|
|
| Day 112: 0.25-1 hour |
|
|
| Day 112: 2-4 hours |
|
|
| Day 140: 2-4 hours |
|
|
| Day 168: 2-4 hours |
|
|
| Day 196: 2-4 hours |
|
|
| Day 218: 2-4 hours |
|
|
|
| Day 42: Pre-dose |
|
|
| Day 42: 0.25-1 hour |
|
|
| Day 42: 2- 4 hours |
|
|
| Day 70: 2-4 hours |
|
|
| Day 98: 2-4 hours |
|
|
| Title | Measurements |
|---|---|
|
| 1 hour |
|
| 2 hours |
|
| 4 hours |
|
| 6 hours |
|
| 8 hours |
|
| 12 hours |
|
| 24 hours |
|
| 36 hours |
|
| 48 hours |
|
| 72 hours |
|
| 168 hours |
|
| 336 hours |
|
| 504 hours |
|
| 672 hours |
|
| 1008 hours |
|
|
| Day 56: 2-4 hours |
|
|
| Day 63: Pre-dose |
|
|
| Day 63: 0.25-1 hour |
|
|
| Day 63: 2-4 hours |
|
|
| Day 70: Pre-dose |
|
|
| Day 70: 0.25-1 hour |
|
|
| Day 70: 2-4 hours |
|
|
| Day 77: Pre-dose |
|
|
| Day 77: 0.25-1 hour |
|
|
| Day 77: 2-4 hours |
|
|
| Day 84: Pre-dose |
|
|
| Day 84 :0.25-1 hour |
|
|
| Day 84: 2-4 hours |
|
|
| Day 91: Pre-dose |
|
|
| Day 91; 0.25-1 hour |
|
|
| Day 91: 2-4 hours |
|
|
| Day 98: Pre-dose |
|
|
| Day 98: 0.25-1 hour |
|
|
| Day 98: 2-4 hours |
|
|
| Day 105: Pre-dose |
|
|
| Day 105: 0.25-1 hour |
|
|
| Day 105: 2-4 hours |
|
|
| Day 112: Predose |
|
|
| Day 112: 0.25-1 hour |
|
|
| Day 112: 2-4 hours |
|
|
| Day 140: 2-4 hours |
|
|
| Day 168: 2-4 hours |
|
|
| Day 196: 2-4 hours |
|
|
| Day 218: 2-4 hours |
|
|
|
| Day 42: Pre-dose |
|
|
| Day 42: 0.25-1 hour |
|
|
| Day 42: 2-4 hours |
|
|
| Day 70: 2-4 hours |
|
|
| Day 98: 2-4 hours |
|
|