A Randomized, Double-blind, Placebo-controlled, Multicenter Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

Official Summary

This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.

Eligibility Requirements

• Minimum Age: 12 Years

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: QUADRUPLE
• Enrollment: 72 participants

Study Arms

• inclacumab 30 mg/kg (EXPERIMENTAL)
  Inclacumab 30 mg/kg administered intravenously (IV)
• placebo (PLACEBO_COMPARATOR)
  Placebo administered IV

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

• Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization (Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]))

Secondary Outcomes

• Time to First VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization (Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]) or censored day, whichever occurred earlier)
• Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 30 Days of Randomization (Within 30 days of randomization (randomization happened on Day 1 [Day 1 to Day 31]))
• Rate of VOCs Leading to a Healthcare Visit That Requires Parenteral Pain Medication or an Increase in Treatment With Oral Narcotics Within 90 Days Following Randomization (Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]))

Eligibility Criteria

Inclusion Criteria:

1. Participant has an index VOC. The index VOC is any VOC that required admission to a healthcare facility and treatment with parenteral pain medication. An admission for the index VOC includes:

   1. A hospital admission, or
   2. An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
   3. 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period

   for an acute episode of pain with no other cause other than a vaso- occlusive event that includes the following:
   * Uncomplicated VOC,
   * Acute chest syndrome (ACS),
   * Acute hepatic sequestration,
   * Acute splenic sequestration, or
   * Priapism.
2. Participant has a confirmed diagnosis of SCD (any genotype). Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing at Baseline.
3. Participant is male or female, ≥ 12 years of age at the time of informed consent.
4. Participant has experienced between 2 and 10 VOCs within the 12 months prior to Screening as determined by documented medical history. The index VOC is not to be considered as one of the 2 to 10 events. A prior VOC is defined as an acute episode of pain that:

   1. Has no medically determined cause other than a vaso-occlusive event, and
   2. Results in a visit to a healthcare facility (hospital, emergency department, urgent care center, outpatient clinic, or infusion center) or results in a remote contact with a healthcare provider; and
   3. Requires parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.
5. Participants receiving erythropoiesis-stimulating agents (ESA, e.g., erythropoietin \[EPO\]) must be on a stable dose for at least 90 days prior to Screening and expected to continue with the stabilized regimen throughout the course of the study.
6. Participants receiving hydroxyurea (HU), L-glutamine, or voxelotor (Oxbryta®) must be on a stable dose for at least 30 days prior to Screening 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 Screening.
3. Participant weighs \> 133 kg (292 lbs.).

Other protocol-defined Inclusion/Exclusion may apply.

Trial Locations

• Strada Patient Care Center, Mobile, Alabama, United States
• 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
• Phoenix Children's Hospital, Phoenix, Arizona, United States
• Arkansas Children's Hospital, Little Rock, Arkansas, United States
• UCSF Benioff Children's Hospital, Oakland, Oakland, California, United States
• Children's Hospital of Orange County, Orange, California, United States
• St. Joseph's Hospital, Tampa, Florida, United States
• University of Michigan Hospitals - Michigan Medicine, Ann Arbor, Michigan, United States
• Functional Fluidics, Inc., Detroit, Michigan, United States
• ...and 10 more locations

Study Officials

• Pfizer CT.gov Call Center — STUDY_DIRECTOR
  Pfizer

More Sickle Cell Disease Trials

View all Sickle Cell Disease clinical trials