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A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

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ClinicalTrials.gov Identifier: NCT04927247
Recruitment Status : Recruiting
First Posted : June 15, 2021
Last Update Posted : November 7, 2022
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief 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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Vaso-occlusive Crisis Vaso-occlusive Pain Episode in Sickle Cell Disease Drug: Inclacumab Drug: Placebo Phase 3

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 280 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double blind study
Primary Purpose: Treatment
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
Actual Study Start Date : December 13, 2021
Estimated Primary Completion Date : December 11, 2023
Estimated Study Completion Date : March 11, 2024


Arm Intervention/treatment
Experimental: inclacumab 30 mg/kg
Inclacumab 30 mg/kg administered intravenously (IV)
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.

Placebo Comparator: placebo
Placebo administered IV
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 Outcome Measures :
  1. Re-admission for a VOC within 90 days of randomization [ Time Frame: Within 90 days of randomization ]
    Following an index VOC, the proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication


Secondary Outcome Measures :
  1. Time to first re-admission for a VOC [ Time Frame: Within 90 days of randomization ]
    Time to first VOC that required admission to a healthcare facility and treatment with parenteral pain medication

  2. Readmission for a VOC within 30 days [ Time Frame: Within 30 days of randomization ]
    Proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication

  3. Rate of VOCs leading to healthcare visits [ Time Frame: Within 90 days following randomization ]
    Rate of VOCs leading to a healthcare visit that requires parenteral pain medication or an increase in treatment with oral narcotics

  4. Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Through Day 91 ]

Other Outcome Measures:
  1. PD parameter (P-selectin inhibition) [ Time Frame: Through Day 91 ]
    To characterize the pharmacodynamics (PD) (P-selectin inhibition) of inclacumab at 30 mg/kg

  2. PD parameter (Platelet Leukocyte Aggregation) [ Time Frame: Through Day 91 ]
    To characterize the pharmacodynamics (PD) (PLA) of inclacumab at 30 mg/kg



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04927247


Contacts
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Contact: Carolyn Hoppe, MD (510) 289-9097 THRIVEstudy@gbt.com
Contact: Stacy Tsukayama stsukayama@gbt.com

Locations
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Sponsors and Collaborators
Global Blood Therapeutics
Additional Information:
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT04927247    
Other Study ID Numbers: GBT2104-132
2020-005287-60 ( EudraCT Number )
First Posted: June 15, 2021    Key Record Dates
Last Update Posted: November 7, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Global Blood Therapeutics:
Re-admission
Acute
blood disorders
hemoglobin
red blood cells
RBCs
sickle-like shape
mutation in hemoglobin gene
sickle-cell trait
sickle-cell crisis
Sickle Cell Disease
SCD
Vaso-occlusive Crisis
VOC
SCA
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn