Clinical trial NCT03938454: A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Priapism
Keywords: Priapism
sickle cell disease
SCD
crizanlizumab
P-selectin
SEG101
monoclonal antibody
prolonged erection
painful erection
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2019-05-06
Enrollment: 36
Sex: MALE
Minimum age (years): 12
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with 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.

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

Primary outcomes:
measure: Percent Change in Priapic Events From Baseline to 26 Weeks
timeFrame: Baseline up to 26 weeks
Secondary outcomes:
measure: Annualized Rate of Priapic Events
timeFrame: Baseline up to 26 and 52 weeks
measure: Number of Acute Priapic Events From Baseline to 26 and 52 Weeks
timeFrame: Baseline up to 26 and 52 weeks
measure: Annualized Rate of Uncomplicated Vaso-occlusive Crises (VOCs)
timeFrame: Baseline up to 26 and 52 weeks
measure: Annualized Rate of Complicated VOCs
timeFrame: Baseline up to 26 and 52 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University Of Alabama
state: Alabama
city: Farmington
country: United States
facility: University of Connecticut Health Center
state: Connecticut
city: Washington
country: United States
facility: Childrens National Hospital
state: District of Columbia
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Shreveport
country: United States
facility: LSU Medical Center
state: Louisiana
city: Boston
country: United States
facility: Childrens Hosp Boston Dept of Hematology
state: Massachusetts
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: Brody School of Medicine
state: North Carolina
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Greenville
country: United States
facility: Prisma Health Upstate
state: South Carolina
city: Houston
country: United States
facility: University of Texas Medical School
state: Texas
Study officials:
References: