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

About the study

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.

Study point of contact

Carolyn Hoppe, MD
650-822-8728
[email protected]
Eleanor Sales
[email protected]

Locations

10 United States sites

1 Lebanon site

Age

> 12 Years

Genotypes

HbSS, HbSC, HbSB0 thalassemia

Phase

Phase 3

Study type

Interventional

Gender

All

Interventions

Drug

participation requirements

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.

participation restrictions

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.

Locations

  • Mobile, Alabama, United States, University of South Alabama Children's and Women's Hospital
  • Little Rock, Arkansas, United States, Arkansas Children's Hospital
  • Tampa, Florida, United States, University of South Florida
  • Atlanta, Georgia, United States, Children's Healthcare of Atlanta at Scottish Rite Hospital
  • Chicago, Illinois, United States, University of Illinois at Chicago
  • Boston, Massachusetts, United States, Brigham and Women's Hospital
  • Ann Arbor, Michigan, United States, University of Michigan
  • Bronx, New York, United States, Jacobi Medical Center
  • Durham, North Carolina, United States, Duke University Medical Center
  • Memphis, Tennessee, United States, St Jude Children's Research Hospital
  • Tripoli, Lebanon, Nini Hospital
Last updated 2021-11-04