A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
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|ClinicalTrials.gov Identifier: NCT04935879|
Recruitment Status : Recruiting
First Posted : June 23, 2021
Last Update Posted : November 7, 2022
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease Vaso-occlusive Pain Episode in Sickle Cell Disease Vaso-occlusive Crisis||Drug: Inclacumab Drug: Placebo||Phase 3|
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 (Clinical Trial)|
|Estimated Enrollment :||240 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Double blind study|
|Official Title:||A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises|
|Actual Study Start Date :||October 26, 2021|
|Estimated Primary Completion Date :||March 31, 2023|
|Estimated Study Completion Date :||April 30, 2024|
Experimental: inclacumab, 30 mg/kg
Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks
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 Comparator: placebo
Participants will receive placebo administered IV every 12 weeks.
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 VOCs during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
A VOC is defined as an acute episode of pain that:
- has no medically determined cause other than a vaso-occlusive event, and
- results in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or results in a remote contact with a healthcare provider; and
- requires parenteral narcotic agents, parenteral non-steroidal anti-inflammatoroy drugs (NSAIDS), or an increase in treatment with oral narcotics.
Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, and priapism that meet the requirements listed above will be included in the primary endpoint
- Time to first VOC during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
- Time to second VOC during the 48-week treatment period Efficacy. [ Time Frame: Day 1- Week 48 ]
- Proportion of participants with no VOCs during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
- Rate of VOCs that required admission to a healthcare facility and treatment with parenteral pain medication during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]Admission includes: (a) A hospital admission, or (b) An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or (c) 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period.
- Number of days of inpatient hospitalization for a VOC during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
- Incidence of treatment-emergent adverse events (TEAEs). [ Time Frame: Day 1- Week 48 ]
- PD parameter (P-selectin inhibition) [ Time Frame: Day 1- Week 48 ]To characterize the pharmacodynamics (PD) (P-selectin inhibition) of inclacumab at 30 mg/kg
- PD parameter (Platelet Leukocyte Aggregation) [ Time Frame: Day 1- Week 48 ]To characterize the pharmacodynamics (PD) (PLA) of inclacumab at 30 mg/kg
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04935879
|Contact: Carolyn Hoppe, MD||650-822-8728||THRIVEstudy@gbt.com|