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

NCT ID: NCT04927247

Last Updated: 2024-12-11

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

72 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-09

Study Completion Date

2023-11-24

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Sickle Cell Disease Vaso-occlusive Crisis Vaso-occlusive Pain Episode in Sickle Cell Disease

Keywords

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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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double blind study

Study Groups

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inclacumab 30 mg/kg

Inclacumab 30 mg/kg administered intravenously (IV)

Group Type EXPERIMENTAL

Inclacumab

Intervention Type DRUG

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

Placebo administered IV

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Strada Patient Care Center

Mobile, Alabama, United States

Site Status

University of South Alabama Children's and Women's Hospital

Mobile, Alabama, United States

Site Status

University of South Alabama Mitchell Cancer Institute

Mobile, Alabama, United States

Site Status

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status

UCSF Benioff Children's Hospital, Oakland

Oakland, California, United States

Site Status

Children's Hospital of Orange County

Orange, California, United States

Site Status

St. Joseph's Hospital

Tampa, Florida, United States

Site Status

University of Michigan Hospitals - Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

Functional Fluidics, Inc.

Detroit, Michigan, United States

Site Status

Alliance for Childhood Diseases dba Cure 4 The Kids Foundation

Las Vegas, Nevada, United States

Site Status

Erie County Medical Center

Buffalo, New York, United States

Site Status

Jacobi Medical Center

The Bronx, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

DUMC Investigational Drug Services Pharmacy

Durham, North Carolina, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

PPD Bioanalytical

Richmond, Virginia, United States

Site Status

Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael

Salvador, Estado de Bahia, Brazil

Site Status

Multihemo Servicos Medicos S/A

Recife, Pernambuco, Brazil

Site Status

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

Fundação Faculdade Regional de Medicina de São José do Rio Preto

São José do Rio Preto, São Paulo, Brazil

Site Status

Casa de Saude Santa Marcelina

São Paulo, São Paulo, Brazil

Site Status

Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, , Brazil

Site Status

Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP

São Paulo, , Brazil

Site Status

CEPEC-Centro de Pesquisa Clinica

São Paulo, , Brazil

Site Status

Clinica de la Costa Ltda.

Barranquilla, Atlántico, Colombia

Site Status

Organizacion Clinica Bonnadona Prevenir S.A.S.

Barranquilla, Atlántico, Colombia

Site Status

Hopital Avicenne

Bobigny, , France

Site Status

Hopital Henri Mondor

Créteil, , France

Site Status

Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation

Regensburg, , Germany

Site Status

Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera

Genova, Genoa, Italy

Site Status

S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro

Genova, , Italy

Site Status

DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitell

Napoli, , Italy

Site Status

UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria

Napoli, , Italy

Site Status

UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"

Napoli, , Italy

Site Status

UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"

Napoli, , Italy

Site Status

Kemri/Crdr, Siaya, Kemri Clinical Research Annex,

Kisumu, Siaya County, Kenya

Site Status

International Cancer Institute

Eldoret, , Kenya

Site Status

Kenya medical Research Institute-centre for Respiratory Disease Research

Nairobi, , Kenya

Site Status

Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES)

Nairobi, , Kenya

Site Status

American University of Beirut Medical Center

Hamra, Beyrouth, Lebanon

Site Status

Nini Hospital

Tripoli, North Lebanon, Lebanon

Site Status

University of Calabar Teaching Hospital

Calabar, Cross River State, Nigeria

Site Status

National Hospital Abuja

Abuja, Federal Capital Territory, Nigeria

Site Status

University of Abuja Teaching Hospital

Gwagwalada, Federal Capital Territory, Nigeria

Site Status

Ahmadu Bello University Teaching Hospital (ABUTH)

Zaria, Kaduna State, Nigeria

Site Status

University of Nigeria Teaching Hospital

Enugu, , Nigeria

Site Status

Barau Dikko Teaching Hospital/Kaduna State University

Kaduna, , Nigeria

Site Status

Aminu Kano Teaching Hospital

Kano, , Nigeria

Site Status

Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital

Lagos, , Nigeria

Site Status

Sultan Qaboos University Hospital

Muscat, , Oman

Site Status

Prince Mohammed bin Nasser Hospital

Jizan, Southern, Saudi Arabia

Site Status

Hacettepe University

Ankara, Altindag/sihhiye, Turkey (Türkiye)

Site Status

Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi

Yenischir, Mersin, Mersin, Turkey (Türkiye)

Site Status

Baskent University Hospital

Adana, Yuregir, Turkey (Türkiye)

Site Status

Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji

Adana, , Turkey (Türkiye)

Site Status

Guy's & Thomas' NHS Foundation Trust

London, England, United Kingdom

Site Status

Matero Clinical Research Site,

Lusaka, , Zambia

Site Status

University Teaching Hospital- Children's Hospital

Lusaka, , Zambia

Site Status

Countries

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United States Brazil Colombia France Germany Italy Kenya Lebanon Nigeria Oman Saudi Arabia Turkey (Türkiye) United Kingdom Zambia

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://pmiform.com/clinical-trial-info-request?StudyID=GBT2104-132

To obtain contact information for a study center near you, click here.

Other Identifiers

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C5361002

Identifier Type: OTHER

Identifier Source: secondary_id

2020-005287-60

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2020-005287-60

Identifier Type: REGISTRY

Identifier Source: secondary_id

GBT2104-132

Identifier Type: -

Identifier Source: org_study_id