Summary

Eligibility
for people ages 12 years and up (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Elliott Vichinsky
Headshot of Elliott Vichinsky
Elliott Vichinsky

Description

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.

Official Title

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

Details

The study will include approximately 280 adult and adolescent participants (≥ 12 years of age) with SCD. Eligible participants will be administered inclacumab or placebo intravenous (IV) as a single dose. Participants that complete the study through Day 90 will be provided the opportunity to enroll in an open-label extension (OLE) study.

Keywords

Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease, Re-admission, Acute, blood disorders, hemoglobin, red blood cells, RBCs, sickle-like shape, mutation in hemoglobin gene, sickle-cell trait, sickle-cell crisis, SCD, VOC, SCA, Anemia, Sickle Cell, Inclacumab, inclacumab 30 mg/kg

Eligibility

You can join if…

Open to people ages 12 years and up

  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:
  2. A hospital admission, or
  3. An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
  4. 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.
  • 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.
  • 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 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:
  • Has no medically determined cause other than a vaso-occlusive event, and
  • 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
  • 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 Screening 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 Screening and expected to continue with the stabilized regimen throughout the course of the study.

You CAN'T join if...

  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.

Locations

  • UCSF Benioff Children's Hospital accepting new patients
    San Francisco California 94609-1809 United States
  • Children's Hospital of Orange County accepting new patients
    Orange California 92868 United States

Lead Scientist at UCSF

  • Elliott Vichinsky
    I am the Director of Hematology/Oncology at UCSF Benioff Children's Hospital Oakland, Adjunct Professor at University of California San Francisco, as well as the Director of the Northern California Sickle Cell and Thalassemia Centers. I am a board-certified pediatric hematologist/oncologist with a major interest in understanding and improving the care of patients with hemoglobinopathies.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Global Blood Therapeutics
Links
Global Blood Therapeutics (GBT) website
ID
NCT04927247
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 280 study participants
Last Updated