A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism

Study Overview
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Brief Summary
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The goal of the study is to evaluate the efficacy and safety of crizanlizumab in SCD patients with priapism.

Official Title
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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)

Conditions
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Priapism

Intervention / Treatment
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* Drug: crizanlizumab


Participation Criteria
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Eligibility Criteria
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Inclusion criteria Male patients aged 16 years and above Confirmed diagnosis of SCD by hemoglobin electrophoresis or high performance liquid chromatography. All SCD genotypes are 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 plan 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 plan to continue taking the drug at the same dose and schedule during the trial Written informed consent (or assent/ parental consent for minor subjects) 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 are not allowed Took drugs/medications that may induce priapism over the 14 weeks preceding study entry Received leuprolide acetate (Lupron) within 3 months before pre-screening. Had an erection lasting more than 12 hours over the 14 week preceding study entry Had an erection lasting more than 12 hours during the 12 weeks of the screening period Other protocol-defined inclusion/exclusion criteria may apply

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Crizanlizumab<br>5 mg/kg by intravenous infusion at Week 1 Day 1, Week 3 Day 1 and Day 1 of every 4-week cycle until Week 51 | Drug: crizanlizumab<br>* Crizanlizumab is a concentrate for solution for infusion, i.v. use. Supplied in single use 10 mL vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab<br>* Other names: SEG101;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percent change in priapic events from baseline to 26 weeks | Priapism is defined as an unwanted or painful penile erection lasting at least 60 minutes. The end of the priapic event will be the duration when the unwanted erection has resolved. This event will be self-reported via an electronic reporting system, and this data should be collected throughout the study period. | Baseline up to 26 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of priapic events | The rate of priapic events is defined as the total number of priapic events for a subject occurring from the date of initial infusion to the last contact date of the Treatment Phase | Baseline up to 26 and 52 weeks | 
| Percent change in acute priapic events from baseline to 26 weeks | An acute priapic event is defined as an unwanted, painful erection that lasts more than 4 hours and need a visit to emergency room. | Baseline up to 26 and 52 weeks | 
| Rate of uncomplicated vaso-occlusive crises | The number of uncomplicated VOC events (defined as an acute event of pain with no known cause for pain other than a vaso occlusive event; and requiring treatment with a parenteral or oral opioids or other parenteral analgesic; but is NOT classified as an acute chest syndrome, hepatic sequestration, splenic sequestration or priapism). Events include both healthcare and self-reported events. | Baseline up to 26 and 52 weeks | 
| Rate of complicated vaso-occlusive crises | The number of complicated VOCs (defined as acute chest syndrome, hepatic sequestration, splenic sequestration, and acute priapism) recorded by healthcare visit. | Baseline up to 26 and 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Priapism, sickle cell disease, SCD, crizanlizumab, P-selectin, SEG101, monoclonal antibody, prolonged erection, painful erection