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Phase 3 N=241 Randomized Quadruple-blind Treatment

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

Sickle Cell Disease · Vaso-occlusive Pain Episode in Sickle Cell Disease · Vaso-occlusive Crisis

Enrolled (actual)
241
Serious AEs
20.9%
Results posted
Dec 2025
Primary outcome: Primary: Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48 — 1.49; 1.58 Events per 48 weeks — p=0.6967

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Inclacumab (Drug); Placebo (Drug)
Age
Pediatric, Adult, Older Adult · 12+ yrs
Sex
All
Sponsor
Pfizer
Primary completion
Jun 2024

Outcome Measures

OutcomeResultp-value
PRIMARY
Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48
1.49; 1.58 0.6967
SECONDARY
Time to First VOC Through Week 48
28.7; 21.6 0.4298
SECONDARY
Time to Second VOC Through Week 48
NA; NA
SECONDARY
Percentage of Participants With no VOCs Through Week 48
36.0; 25.7 0.0912
SECONDARY
Rate of VOCs Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication [Adjudicated] Through Week 48
0.85; 0.83 0.9246
SECONDARY
Rate of Inpatient Hospitalization Days for a VOC Through Week 48
4.96; 5.37 =0.8263
SECONDARY
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
87; 97

Summary

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.

Eligibility Criteria

Inclusion Criteria

  • Participant has a confirmed diagnosis of SCD (HbSS, HbSC, HbSB0 thalassemia, or HbSB+ thalassemia genotype).

Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing during Screening.

  • Participant is male or female, ≥ 12 years of age at the time of informed consent.
  • Participant has experienced between 2 and 10 VOCs within the 12 months prior to the Screening Visit as determined by documented medical history. A prior VOC is defined as an acute episode of pain which:
  • Has no medically determined cause other than a vaso-occlusive event, and
  • Results in a visit to a medical facility (hospital, 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 nonsteroidal anti- inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.
  • Participants receiving erythropoiesis-stimulating agents (ESA, e.g., erythropoietin [EPO]) must be on a stable dose for at least 90 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.
  • Participants receiving hydroxyurea (HU), L-glutamine, or voxelotor (Oxbryta®) must be on a stable dose for at least 30 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.

Exclusion Criteria

  • Participant is receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion).
  • Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days prior to the Screening Visit
  • Participant weighs > 133 kg (292 lbs.).

Other protocol-defined Inclusion/Exclusion may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04935879). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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