Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism (NCT NCT03938454)
NCT ID: NCT03938454
Last Updated: 2026-01-13
Results Overview
A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. Number of priapic events was summarized at Baseline (adjusted for 26 weeks) and by 26 weeks, and percent reduction from adjusted Baseline by 26 weeks was summarized.
COMPLETED
PHASE2
36 participants
Baseline up to 26 weeks
2026-01-13
Participant Flow
Participant milestones
| Measure |
Crizanlizumab 5 mg/kg
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Overall Study
STARTED
|
36
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Crizanlizumab 5 mg/kg
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Lost to Follow-up
|
3
|
Baseline Characteristics
A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
Baseline characteristics by cohort
| Measure |
Crizanlizumab 5 mg/kg
n=36 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Age, Continuous
|
31.5 years
STANDARD_DEVIATION 10.31 • n=210 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=210 Participants
|
|
Sex: Female, Male
Male
|
36 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=210 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=210 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
36 Participants
n=210 Participants
|
PRIMARY outcome
Timeframe: Baseline up to 26 weeksPopulation: The full analysis set (FAS) included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment.
A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. Number of priapic events was summarized at Baseline (adjusted for 26 weeks) and by 26 weeks, and percent reduction from adjusted Baseline by 26 weeks was summarized.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=36 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Percent Change in Priapic Events From Baseline to 26 Weeks
|
-61.3 Percent change from baseline
Interval -80.1 to -17.3
|
SECONDARY outcome
Timeframe: Baseline up to 26 and 52 weeksPopulation: The FAS included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment.
A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=36 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Annualized Rate of Priapic Events
Annualized rate of priapic events by 26 weeks
|
31.1 Events per year
Interval 10.2 to 97.4
|
|
Annualized Rate of Priapic Events
Annualized rate of priapic events by 52 weeks
|
25.6 Events per year
Interval 9.9 to 68.7
|
SECONDARY outcome
Timeframe: Baseline up to 26 and 52 weeksPopulation: The FAS included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment. Only participants with Acute Priapic Events are included in this analysis.
An acute priapic event was defined as an unwanted, painful erection that lasted more than 4 hours and required a visit to the emergency room.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=11 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Number of Acute Priapic Events From Baseline to 26 and 52 Weeks
Number of acute priapic events at Baseline
|
7.0 Number of acute priapic events
Interval 2.0 to 11.0
|
|
Number of Acute Priapic Events From Baseline to 26 and 52 Weeks
Number of post-baseline acute priapic events by 26 weeks
|
1.5 Number of acute priapic events
Interval 1.0 to 2.0
|
|
Number of Acute Priapic Events From Baseline to 26 and 52 Weeks
Number of post-baseline acute priapic events by 52 weeks
|
1.0 Number of acute priapic events
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: Baseline up to 26 and 52 weeksPopulation: The FAS included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment. Only participants who had uncomplicated VOC events within the specified time frame were included in the analysis.
An uncomplicated VOC event was defined as an acute event of pain with no known cause for pain other than a VOC event; and requiring treatment with a parenteral or oral opioids or other parenteral analgesic; but was NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism. Events included both healthcare and self-reported events. The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=24 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)
Annualized rate of uncomplicated VOCs by 26 weeks
|
4.1 Events per year
Interval 3.9 to 8.0
|
|
Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)
Annualized rate of uncomplicated VOCs by 52 weeks
|
2.5 Events per year
Interval 1.4 to 6.8
|
SECONDARY outcome
Timeframe: Baseline up to 26 and 52 weeksPopulation: The FAS included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment. Only participants who had complicated VOC events within the specified time frame were included in the analysis.
Complicated VOCs were defined as acute chest syndrome, hepatic sequestration, splenic sequestration, and acute priapism recorded by healthcare visit. The annualized rate of events was defined as the total number of events for a participant occurring from the date of initial infusion to the last contact date of the Treatment Phase of the study x 365.25 divided by the number of days during that same time period. The calculation accounted for early dropouts or lost to follow-up by extrapolating the priapism events rate of every participant to 1 year.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=2 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Annualized Rate of Complicated VOCs
Annualized rate of complicated VOCs by 26 weeks
|
1.9 Events per year
Interval 1.9 to 1.9
|
|
Annualized Rate of Complicated VOCs
Annualized rate of complicated VOCs by 52 weeks
|
1.4 Events per year
Interval 0.9 to 1.9
|
POST_HOC outcome
Timeframe: Baseline up to 57 weeksPopulation: The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
A TEAE was defined as an adverse event starting or worsening after the administration of study medication. TEAEs experienced by participants in association with infusion related reactions were classified as adverse events of special interest (AESIs).
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=36 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Infusion related reaction
|
2 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
With at least one AESI
|
9 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Axillary pain
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Non-cardiac chest pain
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Arthralgia
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Back pain
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Neck pain
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Headache
|
3 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Pruritis
|
1 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Infusion Related Reactions (Adverse Events of Special Interest)
Rash maculo-papular
|
1 Participants
|
POST_HOC outcome
Timeframe: Baseline up to 26 weeksPopulation: The FAS included all enrolled participants to whom the study treatment was assigned regardless of whether or not they received at least one dose of study treatment or had at least one post-baseline assessment. Analysis was performed excluding one outlier participant, who had a large amount of data unreported.
A priapic event was defined as an unwanted or painful penile erection lasting at least 60 minutes. Priapic events were self-reported via an electronic reporting system, and data was collected throughout the study period. Number of priapic events was summarized at Baseline (adjusted for 26 weeks) and by 26 weeks, and percent reduction from adjusted Baseline by 26 weeks was summarized. For one participant, the outlier, a large amount of data corresponding to 38 days of screening (45% of the period), was not reported. Additionally, data in the other days of the Screening Period were also not collected. This was a unique occurrence which did not happen with other participants. To understand its impact, the primary endpoint has also been analyzed excluding this outlier in a post-hoc sensitivity analysis.
Outcome measures
| Measure |
Crizanlizumab 5 mg/kg
n=35 Participants
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Percent Change in Priapic Events From Baseline to 26 Weeks Without the Outlier
|
-63.3 Percent change from baseline
Interval -81.1 to -17.6
|
Adverse Events
Crizanlizumab 5 mg/kg
Serious adverse events
| Measure |
Crizanlizumab 5 mg/kg
n=36 participants at risk
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
General disorders
Peripheral swelling
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Infections and infestations
Periorbital cellulitis
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Infections and infestations
Pneumonia
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Vascular disorders
Jugular vein thrombosis
|
2.8%
1/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
Other adverse events
| Measure |
Crizanlizumab 5 mg/kg
n=36 participants at risk
Participants received 5 mg/kg by IV infusion on Week 1 Day 1, Week 3 Day 1, and on Day 1 of every 4-week cycle until Week 51.
|
|---|---|
|
Cardiac disorders
Sinus bradycardia
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Gastrointestinal disorders
Constipation
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.1%
4/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Gastrointestinal disorders
Vomiting
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
General disorders
Chest discomfort
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
General disorders
Fatigue
|
8.3%
3/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
General disorders
Non-cardiac chest pain
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
General disorders
Pyrexia
|
16.7%
6/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Infections and infestations
COVID-19
|
13.9%
5/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Amylase increased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Aspartate aminotransferase increased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Blood bilirubin increased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Blood creatine phosphokinase increased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Blood lactate dehydrogenase increased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Lymphocyte count increased
|
8.3%
3/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Investigations
Neutrophil count decreased
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
8.3%
3/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
3/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Nervous system disorders
Headache
|
19.4%
7/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.3%
3/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.6%
2/36 • Adverse events were reported from first dose of study treatment until end of study treatment, up to a maximum duration of 57 weeks.
The Safety Analysis Set consisted of all participants who received at least 1 dose of study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER