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

Official 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 Requirements

• Minimum Age: 12 Years

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 241 participants

Study Arms

• inclacumab, 30 mg/kg (EXPERIMENTAL)
  Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks
• placebo (PLACEBO_COMPARATOR)
  Participants will receive placebo administered IV every 12 weeks.

Interventions

• DRUG: Inclacumab — 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.
• DRUG: Placebo — 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

Primary Outcomes

• Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48 (Randomization (Day 1) up to Week 48)

Secondary Outcomes

• Time to First VOC Through Week 48 (Randomization (Day 1) up to Week 48)
• Time to Second VOC Through Week 48 (Randomization (Day 1) up to Week 48)
• Percentage of Participants With no VOCs Through Week 48 (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 (Randomization (Day 1) up to Week 48)
• Rate of Inpatient Hospitalization Days for a VOC Through Week 48 (Randomization (Day 1) up to Week 48)

Eligibility Criteria

Inclusion Criteria:

1. 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.
2. Participant is male or female, ≥ 12 years of age at the time of informed consent.
3. 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.
4. 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.
5. 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:

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

Other protocol-defined Inclusion/Exclusion may apply.

Trial Locations

• 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 10 more locations

Study Officials

• Pfizer CT.gov Call Center — STUDY_DIRECTOR
  Pfizer

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