This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.
Eligible participants will be administered inclacumab or placebo intravenous (IV) every 12 weeks. The total duration of treatment for each participant will be 48 weeks. Participants that complete the study through Week 48 will be provided the opportunity to enroll in an open-label extension (OLE) study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
241
Inclacumab will be supplied in single use 10 mL vials at a concentration of 50 mg/mL. One vial contains 500 mg of inclacumab. This is a liquid concentrate for IV infusion.
Placebo will be supplied in single use 10 mL vials containing the same ingredients without the active drug. Placebo will be prepared as a liquid concentrate for IV infusion and administered in the same manner as active study drug
Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. The rate of VOC was defined as number of VOC events per 48 weeks and presented in this outcome measure.
Time frame: Randomization (Day 1) up to Week 48
Time to First VOC Through Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral NSAIDs, or an increase in treatment with oral narcotics. Time to first VOC was the time between randomization date and onset date of first VOC event during 48 weeks. Kaplan-Meier method was used for estimation.
Time frame: Randomization (Day 1) up to Week 48
Time to Second VOC Through Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral NSAIDs, or an increase in treatment with oral narcotics. Time to second VOC was the time between randomization date and onset date of second VOC event during 48 weeks. Kaplan-Meier method was used for estimation.
Time frame: Randomization (Day 1) up to Week 48
Percentage of Participants With no VOCs Through Week 48
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University of South Alabama Children's and Women's Hospital
Mobile, Alabama, United States
University of South Alabama Mitchell Cancer Institute
Mobile, Alabama, United States
University of South Alabama Strada Patient Care Center
Mobile, Alabama, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UC Irvine Health
Orange, California, United States
UCI Center for clinical research
Orange, California, United States
Uconn Health/Uconn John Dempsey Hospital/Neag Comprehensive Cancer Center/New England Sickle Cell
Farmington, Connecticut, United States
Hospital Pharmacy Services- Investigational Drug Services
Chicago, Illinois, United States
Rush University Medical Center Investigator Pharmacy
Chicago, Illinois, United States
Rush University Medical Center
Chicago, Illinois, United States
...and 48 more locations
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral NSAIDs, or an increase in treatment with oral narcotics. Participants without an observed VOC who discontinued the study prior to the end of the 48-week treatment period were assumed to had experienced at least one VOC.
Time frame: Randomization (Day 1) up to Week 48
Rate of VOCs Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication [Adjudicated] Through Week 48
A VOC that required admission to a healthcare facility and treatment with parenteral pain medication where admission included: a hospital admission or an admission to an emergency room, observation unit, or infusion center for \>= 12 hours, or 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period. The rate of VOC was defined as number of VOC events per 48 weeks; rate of VOCs which required admission to a healthcare facility and treatment with parenteral pain medication is presented in this outcome measure.
Time frame: Randomization (Day 1) up to Week 48
Rate of Inpatient Hospitalization Days for a VOC Through Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility hospitalization. For each VOC event requiring inpatient hospitalization (regardless of treatment received) during the 48-week, the number of days hospitalized were determined based on the hospital admission and discharge dates. The rate of inpatient hospitalization days was defined as number of inpatient hospitalization days for a VOC per 48 weeks and presented in this outcome measure.
Time frame: Randomization (Day 1) up to Week 48
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product during the course of a clinical investigation. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product, whether or not thought to be related to the investigational product. A TEAE was defined as an AE with an onset after the initiation of dosing for the first dose of study drug. A serious adverse events (SAE) or serious suspected adverse reaction is an AE or suspected adverse reaction that, at any dose, in the view of the either the investigator or sponsor, results in any of the following outcomes: death, life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization and congenital anomaly/birth defect. AEs included both serious and all non-SAEs.
Time frame: Day 1 up to Week 60 (12 week of follow-up post Week 48)