Trial Outcomes & Findings for Actigraphy Improvement With Voxelotor (ActIVe) Study (NCT NCT04400487)
NCT ID: NCT04400487
Last Updated: 2023-11-21
Results Overview
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.
COMPLETED
PHASE4
25 participants
Baseline, Week 10-12
2023-11-21
Participant Flow
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.
A total of 25 participants were enrolled in the study.
Participant milestones
| Measure |
Voxelotor
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Screening Period (28 Days)
STARTED
|
25
|
|
Screening Period (28 Days)
COMPLETED
|
25
|
|
Screening Period (28 Days)
NOT COMPLETED
|
0
|
|
Run-in Period (14 Days)
STARTED
|
25
|
|
Run-in Period (14 Days)
COMPLETED
|
25
|
|
Run-in Period (14 Days)
NOT COMPLETED
|
0
|
|
Treatment Period (24 Weeks)
STARTED
|
25
|
|
Treatment Period (24 Weeks)
Treated (With at Least 1 Dose of Study Treatment)
|
25
|
|
Treatment Period (24 Weeks)
COMPLETED
|
23
|
|
Treatment Period (24 Weeks)
NOT COMPLETED
|
2
|
|
Follow-up Period (28 Days)
STARTED
|
23
|
|
Follow-up Period (28 Days)
COMPLETED
|
21
|
|
Follow-up Period (28 Days)
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Voxelotor
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Treatment Period (24 Weeks)
Withdrawal by Subject
|
1
|
|
Treatment Period (24 Weeks)
Non-compliance
|
1
|
|
Follow-up Period (28 Days)
Started commercial product
|
2
|
Baseline Characteristics
Actigraphy Improvement With Voxelotor (ActIVe) Study
Baseline characteristics by cohort
| Measure |
Voxelotor
n=25 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Age, Continuous
|
25 Years
STANDARD_DEVIATION 12.05 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
23 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12
|
-155.6 Counts per minute
Standard Deviation 458.45
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 22-24
|
-79.4 Counts per minute
Standard Deviation 443.77
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Light Physical Activity to Week 10-12
|
5.8 minutes/day
Standard Deviation 62.67
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Light Physical Activity to Week 22-24
|
-11.7 minutes/day
Standard Deviation 115.34
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Moderate Physical Activity to Week 10-12
|
-9.0 minutes/day
Standard Deviation 25.55
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Moderate Physical Activity to Week 22-24
|
-5.7 minutes/day
Standard Deviation 30.01
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Vigorous Physical Activity to Week 10-12
|
0.0 minutes/day
Standard Deviation 4.94
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Vigorous Physical Activity to Week 22-24
|
-1.2 Minutes/day
Standard Deviation 6.52
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Total Nocturnal Sleep Time to Week 10-12
|
0.3 Minutes/day
Standard Deviation 77.64
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Total Nocturnal Sleep Time to Week 22-24
|
17.6 Minutes/day
Standard Deviation 141.10
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=16 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Wake Time After Sleep Onset to Week 10-12
|
2.0 Minutes/day
Standard Deviation 37.10
|
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
| Measure |
Voxelotor
n=14 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Change From Baseline in Wake Time After Sleep Onset to Week 22-24
|
2.0 Minutes/day
Standard Deviation 42.00
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Week 12Population: 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.
Outcome measures
| Measure |
Voxelotor
n=24 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Percentage of Participants With a More Than (>)1 Grams Per Deciliter (g/dL) Increase in Hemoglobin (Hb) at Week 12
|
41.7 Percentage of participants
|
PRIMARY outcome
Timeframe: Week 24Population: 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.
Outcome measures
| Measure |
Voxelotor
n=23 Participants
Participants received 1500 milligram (mg) of voxelotor (three tablets of 500 mg) orally once daily for 24 weeks (168 days).
|
|---|---|
|
Percentage of Participants With a >1 g/dL Increase in Hemoglobin (Hb) at Week 24
|
26.1 Percentage of participants
|
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 10-12Population: 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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Baseline, Week 22-24Population: 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.
Outcome measures
Outcome data not reported
Adverse Events
Voxelotor: SCD Related
Voxelotor: Non-SCD Related
Serious adverse events
| Measure |
Voxelotor: SCD Related
n=25 participants at risk
Participants who received voxelotor 1500 mg and had SCD related AEs.
|
Voxelotor: Non-SCD Related
n=25 participants at risk
Participants received voxelotor 1500 mg and had non-SCD related AEs.
|
|---|---|---|
|
Infections and infestations
Cellulitis
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Nervous system disorders
Headache
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
32.0%
8/25 • 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.
|
0.00%
0/25 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Acute chest syndrome
|
4.0%
1/25 • 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.
|
0.00%
0/25 • 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.
|
Other adverse events
| Measure |
Voxelotor: SCD Related
n=25 participants at risk
Participants who received voxelotor 1500 mg and had SCD related AEs.
|
Voxelotor: Non-SCD Related
n=25 participants at risk
Participants received voxelotor 1500 mg and had non-SCD related AEs.
|
|---|---|---|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
20.0%
5/25 • 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.
|
0.00%
0/25 • 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.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/25 • 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.
|
36.0%
9/25 • 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.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/25 • 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.
|
24.0%
6/25 • 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.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/25 • 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.
|
12.0%
3/25 • 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.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/25 • 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.
|
8.0%
2/25 • 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.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Nervous system disorders
Headache
|
0.00%
0/25 • 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.
|
24.0%
6/25 • 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.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Infections and infestations
COVID-19
|
0.00%
0/25 • 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.
|
8.0%
2/25 • 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.
|
|
Infections and infestations
Scrotal infection
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
General disorders
Fatigue
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Cardiac disorders
Sinus tachycardia
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Immune system disorders
Drug hypersensitivity
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Investigations
Neutrophil count increased
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
|
Reproductive system and breast disorders
Ovarian cyst
|
0.00%
0/25 • 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.
|
4.0%
1/25 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER