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A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

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ClinicalTrials.gov Identifier: NCT04935879
Recruitment Status : Recruiting
First Posted : June 23, 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 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.

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

Detailed Description:

Eligible participants will be administered inclacumab or placebo intravenous (IV) every 12 weeks.

The total duration of treatment for each participant will be 48 weeks.

Participants that complete the study through Week 48 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 : 240 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 to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
Actual Study Start Date : October 26, 2021
Estimated Primary Completion Date : March 31, 2023
Estimated Study Completion Date : April 30, 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: inclacumab, 30 mg/kg
Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks
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
Participants will receive placebo administered IV every 12 weeks.
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. Rate of VOCs during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]

    A 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 medical facility (hospitalization, 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 non-steroidal anti-inflammatoroy drugs (NSAIDS), or an increase in treatment with oral narcotics.

    Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, and priapism that meet the requirements listed above will be included in the primary endpoint



Secondary Outcome Measures :
  1. Time to first VOC during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
  2. Time to second VOC during the 48-week treatment period Efficacy. [ Time Frame: Day 1- Week 48 ]
  3. Proportion of participants with no VOCs during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
  4. Rate of VOCs that required admission to a healthcare facility and treatment with parenteral pain medication during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
    Admission includes: (a) A hospital admission, or (b) An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or (c) 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period.

  5. Number of days of inpatient hospitalization for a VOC during the 48-week treatment period. [ Time Frame: Day 1- Week 48 ]
  6. Incidence of treatment-emergent adverse events (TEAEs). [ Time Frame: Day 1- Week 48 ]

Other Outcome Measures:
  1. PD parameter (P-selectin inhibition) [ Time Frame: Day 1- Week 48 ]
    To characterize the pharmacodynamics (PD) (P-selectin inhibition) of inclacumab at 30 mg/kg

  2. PD parameter (Platelet Leukocyte Aggregation) [ Time Frame: Day 1- Week 48 ]
    To characterize the pharmacodynamics (PD) (PLA) of inclacumab at 30 mg/kg



Information from the National Library of Medicine

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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 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.


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): NCT04935879


Contacts
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Contact: Carolyn Hoppe, MD 650-822-8728 THRIVEstudy@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: NCT04935879    
Other Study ID Numbers: GBT2104-131
2020-005286-13 ( EudraCT Number )
LBCTR2021054791 ( Registry Identifier: Lebanon Clinical Trials Registry )
First Posted: June 23, 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:
blood disorders
hemoglobin
red blood cells
sickle-like shape
mutation in hemoglobin gene
sickle-cell trait
sickle-cell crisis
Sickle Cell Disease
SCD
Vaso-occlusive Crises
VOC
SCA
RBCs
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn