Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| C5341024 | Other Identifier | Alias Study Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
All participants will receive Voxelotor as treatment. There will be approximately 13 sites in the US.
Participant safety and tolerability will be monitored during the study using standard measures, including physical examinations, vital signs (including temperature, blood pressure, pulse rate, respiratory rate and peripheral oxygen saturation [SpO2]), clinical laboratory tests, and adverse event (AE) monitoring.
Screening Period (up to 4 weeks in duration): During this period, participants will sign the informed consent form (ICF), after which they will complete the screening assessments as detailed in the Schedule of Assessments (SOA).
Run-in Period (2 weeks in duration): During this period, participants will enter a 2-week run-in period (Day -14 to Day -1) during which baseline actigraphy measures of physical activity and sleep quality, overnight pulse oximetry assessments of oxygen saturation, and Patient-Reported Outcome (PRO) assessments will be collected before initiating treatment with voxelotor.
Treatment Period (24 weeks in duration): After completion of the 14-day Run-in Period, participants will enter the open label treatment period and receive voxelotor 1500 mg once daily for 24 weeks. Repeat actigraphy assessments of physical activity and sleep quality, and overnight pulse oximetry will be performed during the treatment period (Weeks 10 to 12 and Weeks 22 to 24). PRO and Clinical Global Impression (CGI) assessments will be completed at scheduled study visits. The open-label treatment period is considered the continuous 24 weeks of voxelotor treatment from date of first dose (Day 1).
Follow-up Period (4 weeks in duration): Immediately following the 24-week treatment period, participants will enter a 4-week Follow-up Period.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Voxelotor | Experimental | Participants will receive voxelotor at 1500 mg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Voxelotor | Drug | 500 mg Tablet, Oral, With or Without Food |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12 | Daily physical activity was measured by actigraphy in participants with sickle cell disease (SCD) and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 10-12 |
| Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24 | Daily physical activity was measured by actigraphy in participants with SCD and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Light Physical Activity to Week 10-12 | Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UConn Health | Farmington | Connecticut | 06030 | United States | ||
| Children's Healthcare of Atlanta |
Not provided
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
Not provided
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.
Not provided
Not provided
Not provided
A total of 25 participants were enrolled in the study.
The study included a 28-day screening period, a 14-day run-in period, a 24-week treatment period and a 28-day follow-up period after last dose.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Voxelotor | Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Screening Period (28 Days) |
| |||||||||||||
| Run-in Period (14 Days) |
| |||||||||||||
| Treatment Period (24 Weeks) |
| |||||||||||||
| Follow-up Period (28 Days) |
|
All participants who received at least 1 dose of study treatment were included in the safety population.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Voxelotor | Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days). |
| Units | Counts |
|---|---|
| Participants |
|
| 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 | Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12 | Daily physical activity was measured by actigraphy in participants with sickle cell disease (SCD) and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Counts per minute | Baseline, Week 10-12 |
|
From screening period to 28 days post last dose of study treatment (maximum of 238 days)
All participants who received at least 1 dose of study treatment were included in the safety population. Same event may appear as both non-SAE and SAE but what is presented are distinct events. An event may be categorized as serious in 1 participant and non-serious in other, or a participant may have experienced both serious and non-serious event. AEs were presented which were related to both SCD and Non-SCD related.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Voxelotor: SCD Related | Participants who received voxelotor 1500 mg and had SCD related AEs. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cellulitis | Infections and infestations | MedDRA v 25.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v 25.0 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquires@pfizer.com |
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 28, 2021 | Sep 15, 2023 | Prot_000.pdf |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000628792 | voxelotor |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Baseline, Week 10-12 |
| Change From Baseline in Light Physical Activity to Week 22-24 | Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Moderate Physical Activity to Week 10-12 | Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 10-12 |
| Change From Baseline in Moderate Physical Activity to Week 22-24 | Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Vigorous Physical Activity to Week 10-12 | Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 10-12 |
| Change From Baseline in Vigorous Physical Activity to Week 22-24 | Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Total Nocturnal Sleep Time to Week 10-12 | Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 10-12 |
| Change From Baseline in Total Nocturnal Sleep Time to Week 22-24 | Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Wake Time After Sleep Onset to Week 10-12 | Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 10-12 |
| Change From Baseline in Wake Time After Sleep Onset to Week 22-24 | Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Baseline, Week 22-24 |
| Change From Baseline in Sleep Efficiency to Week 10-12 | Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. | Baseline, Week 10-12 |
| Change From Baseline in Sleep Efficiency to Week 22-24 | Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. | Baseline, Week 22-24 |
| Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12 | Week 12 |
| Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24 | Week 24 |
| Change From Baseline in Mean Overnight Oxygen Saturation (SpO2 Percentage [%]) to Week 10-12 | Baseline, Week 10-12 |
| Change From Baseline in Mean Overnight SpO2 % to Week 22-24 | Baseline, Week 22-24 |
| Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 10-12 | Baseline, Week 10-12 |
| Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 22-24 | Baseline, Week 22-24 |
| Atlanta |
| Georgia |
| 30342 |
| United States |
| Children's Hospital of Michigan | Detroit | Michigan | 48201 | United States |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | United States |
| The Children's Hospital at Montefiore | The Bronx | New York | 10476 | United States |
| Duke Department of Pediatrics | Durham | North Carolina | 27710 | United States |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| The University of Texas Health Science Center at Houston | Houston | Texas | 77030 | United States |
| VCU Health | Richmond | Virginia | 23298 | United States |
| NOT COMPLETED |
|
|
|
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG000 | Voxelotor | Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days). |
|
|
| Primary | Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24 | Daily physical activity was measured by actigraphy in participants with SCD and chronic moderate anemia. Actigraphy assessments were performed using wrist-worn tri-axial accelerometry device. Actigraphy is an accepted methodology for tracking activity levels, time spent in moderate and vigorous physical activity, step counts, and energy expenditure. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Counts per minute | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Light Physical Activity to Week 10-12 | Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | minutes/day | Baseline, Week 10-12 |
|
|
|
| Primary | Change From Baseline in Light Physical Activity to Week 22-24 | Change in daily physical activity was measured by actigraphy in adolescent and adult participants with SCD and chronic moderate anemia. This outcome measure described the change from baseline in light physical activity. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | minutes/day | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Moderate Physical Activity to Week 10-12 | Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | minutes/day | Baseline, Week 10-12 |
|
|
|
| Primary | Change From Baseline in Moderate Physical Activity to Week 22-24 | Change in daily moderate physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | minutes/day | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Vigorous Physical Activity to Week 10-12 | Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | minutes/day | Baseline, Week 10-12 |
|
|
|
| Primary | Change From Baseline in Vigorous Physical Activity to Week 22-24 | Change in daily vigorous physical activity (minutes per day) was measured by actigraphy in participants with SCD and chronic moderate anemia. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Minutes/day | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Total Nocturnal Sleep Time to Week 10-12 | Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Minutes/day | Baseline, Week 10-12 |
|
|
|
| Primary | Change From Baseline in Total Nocturnal Sleep Time to Week 22-24 | Total nocturnal sleep time was measured by overnight actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Minutes/day | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Wake Time After Sleep Onset to Week 10-12 | Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Minutes/day | Baseline, Week 10-12 |
|
|
|
| Primary | Change From Baseline in Wake Time After Sleep Onset to Week 22-24 | Measured by actigraphy monitoring. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. In this outcome measure the average of at least 8 valid days during the specified two-week period was considered. A participant's daily wear must be 18 hours or more in a day to be considered a valid day. | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Minutes/day | Baseline, Week 22-24 |
|
|
|
| Primary | Change From Baseline in Sleep Efficiency to Week 10-12 | Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. | Data for this outcome measure could not be analyzed and reported as data for 'time in bed' was not collected which was required to calculate sleep efficiency. | Posted | Baseline, Week 10-12 |
|
|
| Primary | Change From Baseline in Sleep Efficiency to Week 22-24 | Sleep efficiency was defined as ration of total sleep time to time in bed. Change from baseline to follow-up visit was calculated as the baseline measurement subtracted from the follow-up measurement for all assessments. | Data for this outcome measure could not be analyzed and reported as data for 'time in bed' was not collected which was required to calculate sleep efficiency. | Posted | Baseline, Week 22-24 |
|
|
| Primary | Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12 | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Number | Percentage of participants | Week 12 |
|
|
|
| Primary | Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24 | Participants who received at least 1 dose of voxelotor were included in the safety population. Here, 'Overall Number of Participants Analyzed' signifies number of participants evaluable for this outcome measure. | Posted | Number | Percentage of participants | Week 24 |
|
|
|
| Primary | Change From Baseline in Mean Overnight Oxygen Saturation (SpO2 Percentage [%]) to Week 10-12 | Data for this outcome measure was not analyzed and reported because SpO2 data was not collected as the device that was supposed to measure SpO2 did not work. | Posted | Baseline, Week 10-12 |
|
|
| Primary | Change From Baseline in Mean Overnight SpO2 % to Week 22-24 | Data for this outcome measure was not analyzed and reported because SpO2 data was not collected as the device that was supposed to measure SpO2 did not work. | Posted | Baseline, Week 22-24 |
|
|
| Primary | Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 10-12 | Data for this outcome measure was not analyzed and reported because SpO2 data was not collected as the device that was supposed to measure SpO2 did not work. | Posted | Baseline, Week 10-12 |
|
|
| Primary | Change From Baseline in Median Number of Overnight SpO2 Dips > 3% Per Hour to Week 22-24 | Data for this outcome measure was not analyzed and reported because SpO2 data was not collected as the device that was supposed to measure SpO2 did not work. | Posted | Baseline, Week 22-24 |
|
|
| 0 |
| 25 |
| 8 |
| 25 |
| 5 |
| 25 |
| EG001 | Voxelotor: Non-SCD Related | Participants received voxelotor 1500 mg and had non-SCD related AEs. | 0 | 25 | 3 | 25 | 19 | 25 |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA v 25.0 | Non-systematic Assessment |
|
| Scrotal infection | Infections and infestations | MedDRA v 25.0 | Non-systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA v 25.0 | Non-systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Non-cardiac chest pain | General disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Sinus tachycardia | Cardiac disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Drug hypersensitivity | Immune system disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Neutrophil count increased | Investigations | MedDRA v 25.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
| Ovarian cyst | Reproductive system and breast disorders | MedDRA v 25.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications 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 |