Brief Summary
The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.
Brief Title
A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
Detailed Description
Before participating in this study, information to determine key eligibility criteria was collected as a part of a 14-week Pre-Screening period. The study included a 12-week Screening period and a 52-week (1 year) Treatment period. Eligible participants received crizanlizumab 5 mg/kg by intravenous infusion (IV). Study treatment was received at clinic visits on Week 1 Day 1, Week 3 Day 1, and then on Day 1 of every 4-week cycle. Efficacy assessments included evaluation of priapic and vaso-occlusive (VOC) events. Safety assessments included laboratory tests, electrocardiograms (ECGs), vital signs and physical examinations. Participants had a safety follow-up for up to 15 weeks after the last dose.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Priapism
Eligibility Criteria
Inclusion criteria
* Male patients aged 12 years and above
* Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
* Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
* Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
* If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures
Exclusion criteria:
* Had penile prosthetic implants or shunts or any other surgical procedure on the penis performed within 12 months prior to consenting was not allowed
* Took drugs/medications that may induce priapism over the 14 weeks preceding study entry
* Received leuprolide acetate (Lupron) or any other gonadotropin releasing hormone receptor agonist agent within 3 months before pre-screening
* Had an erection lasting more than 12 hours over the 14 weeks preceding study entry
* Had an erection lasting more than 12 hours during the 12 weeks of the Screening period
* Male patients aged 12 years and above
* Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
* Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
* Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
* If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures
Exclusion criteria:
* Had penile prosthetic implants or shunts or any other surgical procedure on the penis performed within 12 months prior to consenting was not allowed
* Took drugs/medications that may induce priapism over the 14 weeks preceding study entry
* Received leuprolide acetate (Lupron) or any other gonadotropin releasing hormone receptor agonist agent within 3 months before pre-screening
* Had an erection lasting more than 12 hours over the 14 weeks preceding study entry
* Had an erection lasting more than 12 hours during the 12 weeks of the Screening period
Inclusion Criteria
Inclusion criteria
* Male patients aged 12 years and above
* Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
* Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
* Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
* If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures
* Male patients aged 12 years and above
* Confirmed diagnosis of SCD by hemoglobin electrophoresis or high-performance liquid chromatography. All SCD genotypes were eligible (HbSS, HbSβ0, HbSC, HbSβ+, and others)
* Experienced 4 or more priapic events (unwanted erection lasting at least 60 minutes) over the 14 weeks preceding study participation
* Experienced at least 3 priapic events (unwanted erection lasting at least 60 minutes) during the 12-week Screening period with at least 1 event occurring within 4 weeks prior to the first treatment
* If receiving hydroxyurea/hydroxycarbamide or L-glutamine or erythropoietin stimulating agent or voxelotor, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* If receiving prophylactic treatment for priapism, must have been receiving the drug for at least 14 weeks prior to screening and planned to continue taking the drug at the same dose and schedule during the trial
* Written informed consent (or assent/parental consent for minor participants) prior to any screening procedures
Gender
Male
Gender Based
false
Keywords
Priapism
sickle cell disease
SCD
crizanlizumab
P-selectin
SEG101
monoclonal antibody
prolonged erection
painful erection
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
100 Years
Minimum Age
12 Years
NCT Id
NCT03938454
Org Class
Industry
Org Full Name
Novartis
Org Study Id
CSEG101AUS05
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Prospective Phase II, Open-Label, Single-arm, Multicenter, Study to Assess Efficacy and Safety of SEG101 (Crizanlizumab), in Sickle Cell Disease Patients With Priapism (SPARTAN)
Primary Outcomes
Outcome Description
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 Measure
Percent Change in Priapic Events From Baseline to 26 Weeks
Outcome Time Frame
Baseline up to 26 weeks
Secondary Outcomes
Outcome Description
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.
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 Time Frame
Baseline up to 26 and 52 weeks
Outcome Measure
Annualized Rate of Priapic Events
Outcome Description
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 Time Frame
Baseline up to 26 and 52 weeks
Outcome Measure
Number of Acute Priapic Events From Baseline to 26 and 52 Weeks
Outcome Description
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.
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 Time Frame
Baseline up to 26 and 52 weeks
Outcome Measure
Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)
Outcome Description
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.
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 Time Frame
Baseline up to 26 and 52 weeks
Outcome Measure
Annualized Rate of Complicated VOCs
See Also Links
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
100
Minimum Age Number (converted to Years and rounded down)
12
Investigators
Investigator Type
Principal Investigator
Investigator Name
Henny Billett
Investigator Email
hbillett@montefiore.org
Investigator Phone
718-920-6310