Back to resultsA Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
Primary Purpose
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Inclacumab
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring 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
Eligibility Criteria
Inclusion Criteria:
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:
- A hospital admission, or
- An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
- 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.
Exclusion Criteria:
- 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 Screening.
- Participant weighs > 133 kg (292 lbs.).
Other protocol-defined Inclusion/Exclusion may apply.
Sites / Locations
- Strada Patient Care Center
- University of South Alabama Children's and Women's Hospital
- University of South Alabama Mitchell Cancer Institute
- Phoenix Children's Hospital
- Arkansas Children's Hospital
- UCSF Benioff Children's Hospital, Oakland
- Children's Hospital of Orange County
- St. Joseph's Hospital
- University of Michigan Hospitals - Michigan Medicine
- Functional Fluidics, Inc.
- Alliance for Childhood Diseases dba Cure 4 The Kids Foundation
- Jacobi Medical Center
- Erie County Medical Center
- Duke University Medical Center
- DUMC Investigational Drug Services Pharmacy
- St. Jude Children's Research Hospital
- PPD Bioanalytical
- Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael
- Multihemo Servicos Medicos S/A
- Hospital de Clinicas de Porto Alegre
- Fundação Faculdade Regional de Medicina de São José do Rio Preto
- Casa de Saude Santa Marcelina
- Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
- Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP
- CEPEC-Centro de Pesquisa Clinica
- Clinica de la Costa Ltda.
- Organizacion Clinica Bonnadona Prevenir S.A.S.
- Hopital Avicenne
- Hopital Henri Mondor
- Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation
- Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera
- S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro
- DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitell
- UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria
- UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
- UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
- Kemri/Crdr, Siaya, Kemri Clinical Research Annex,
- International Cancer Institute
- Kenya medical Research Institute-centre for Respiratory Disease Research
- Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES)
- American University of Beirut Medical Center
- Nini Hospital
- University of Calabar Teaching Hospital
- National Hospital Abuja
- University of Abuja Teaching Hospital
- Ahmadu Bello University Teaching Hospital (ABUTH)
- University of Nigeria Teaching Hospital
- Barau Dikko Teaching Hospital/Kaduna State University
- Aminu Kano Teaching Hospital
- Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
- Sultan Qaboos University Hospital
- Prince Mohammed bin Nasser Hospital
- Hacettepe University
- Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi
- Baskent University Hospital
- Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
- Guy's & Thomas' NHS Foundation Trust
- Matero Clinical Research Site,
- University Teaching Hospital- Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
inclacumab 30 mg/kg
placebo
Arm Description
Inclacumab 30 mg/kg administered intravenously (IV)
Placebo administered IV
Outcomes
Primary Outcome Measures
Re-admission for a VOC 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
Time to first re-admission for a VOC
Time to first VOC that required admission to a healthcare facility and treatment with parenteral pain medication
Readmission for a VOC within 30 days
Proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication
Rate of VOCs leading to healthcare visits
Rate of VOCs leading to a healthcare visit that requires parenteral pain medication or an increase in treatment with oral narcotics
Incidence of treatment-emergent adverse events (TEAEs)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04927247
Brief Title
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 13, 2021 (Actual)
Primary Completion Date
October 3, 2023 (Anticipated)
Study Completion Date
December 8, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Keywords
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
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
Double blind study
Allocation
Randomized
Enrollment
67 (Actual)
8. Arms, Groups, and Interventions
Arm Title
inclacumab 30 mg/kg
Arm Type
Experimental
Arm Description
Inclacumab 30 mg/kg administered intravenously (IV)
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered IV
Intervention Type
Drug
Intervention Name(s)
Inclacumab
Intervention Description
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
Intervention Name(s)
Placebo
Intervention Description
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 Measure Information:
Title
Re-admission for a VOC within 90 days of randomization
Description
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
Time Frame
Within 90 days of randomization
Secondary Outcome Measure Information:
Title
Time to first re-admission for a VOC
Description
Time to first VOC that required admission to a healthcare facility and treatment with parenteral pain medication
Time Frame
Within 90 days of randomization
Title
Readmission for a VOC within 30 days
Description
Proportion of participants with at least 1 VOC that required admission to a healthcare facility and treatment with parenteral pain medication
Time Frame
Within 30 days of randomization
Title
Rate of VOCs leading to healthcare visits
Description
Rate of VOCs leading to a healthcare visit that requires parenteral pain medication or an increase in treatment with oral narcotics
Time Frame
Within 90 days following randomization
Title
Incidence of treatment-emergent adverse events (TEAEs)
Time Frame
Through Day 91
Other Pre-specified Outcome Measures:
Title
PD parameter (P-selectin inhibition)
Description
To characterize the pharmacodynamics (PD) (P-selectin inhibition) of inclacumab at 30 mg/kg
Time Frame
Through Day 91
Title
PD parameter (Platelet Leukocyte Aggregation)
Description
To characterize the pharmacodynamics (PD) (PLA) of inclacumab at 30 mg/kg
Time Frame
Through Day 91
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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:
A hospital admission, or
An admission to an emergency room, observation unit, or infusion center for ≥ 12 hours, or
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.
Exclusion Criteria:
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 Screening.
Participant weighs > 133 kg (292 lbs.).
Other protocol-defined Inclusion/Exclusion may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Strada Patient Care Center
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36604
Country
United States
Facility Name
University of South Alabama Children's and Women's Hospital
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36604
Country
United States
Facility Name
University of South Alabama Mitchell Cancer Institute
City
Mobile
State/Province
Alabama
ZIP/Postal Code
36604
Country
United States
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
UCSF Benioff Children's Hospital, Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
St. Joseph's Hospital
City
Tampa
State/Province
Florida
ZIP/Postal Code
33607
Country
United States
Facility Name
University of Michigan Hospitals - Michigan Medicine
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Functional Fluidics, Inc.
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Alliance for Childhood Diseases dba Cure 4 The Kids Foundation
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89135
Country
United States
Facility Name
Jacobi Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Facility Name
Erie County Medical Center
City
Buffalo
State/Province
New York
ZIP/Postal Code
14215
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
DUMC Investigational Drug Services Pharmacy
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
St. Jude Children's Research Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38105
Country
United States
Facility Name
PPD Bioanalytical
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23230
Country
United States
Facility Name
Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael
City
Salvador
State/Province
Bahia
ZIP/Postal Code
41253-190
Country
Brazil
Facility Name
Multihemo Servicos Medicos S/A
City
Recife
State/Province
PE
ZIP/Postal Code
50070-460
Country
Brazil
Facility Name
Hospital de Clinicas de Porto Alegre
City
Porto Alegre
State/Province
RS
ZIP/Postal Code
90035-903
Country
Brazil
Facility Name
Fundação Faculdade Regional de Medicina de São José do Rio Preto
City
São José Do Rio Preto
State/Province
SP
ZIP/Postal Code
15090-000
Country
Brazil
Facility Name
Casa de Saude Santa Marcelina
City
São Paulo
State/Province
SP
ZIP/Postal Code
08270-070
Country
Brazil
Facility Name
Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
City
Rio de Janeiro
ZIP/Postal Code
20211-030
Country
Brazil
Facility Name
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP
City
São Paulo
ZIP/Postal Code
05403-000
Country
Brazil
Facility Name
CEPEC-Centro de Pesquisa Clinica
City
São Paulo
ZIP/Postal Code
08270-120
Country
Brazil
Facility Name
Clinica de la Costa Ltda.
City
Barranquilla
State/Province
Atlantico
ZIP/Postal Code
080020
Country
Colombia
Facility Name
Organizacion Clinica Bonnadona Prevenir S.A.S.
City
Barranquilla
State/Province
Atlántico
ZIP/Postal Code
080020
Country
Colombia
Facility Name
Hopital Avicenne
City
Bobigny
ZIP/Postal Code
93000
Country
France
Facility Name
Hopital Henri Mondor
City
Créteil
ZIP/Postal Code
94010
Country
France
Facility Name
Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation
City
Regensburg
ZIP/Postal Code
93053
Country
Germany
Facility Name
Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera
City
Genova
State/Province
Genoa
ZIP/Postal Code
16128
Country
Italy
Facility Name
S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro
City
Genova
ZIP/Postal Code
16128
Country
Italy
Facility Name
DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitell
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Facility Name
UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Facility Name
UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Facility Name
UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Facility Name
Kemri/Crdr, Siaya, Kemri Clinical Research Annex,
City
Kisumu
State/Province
Siaya
ZIP/Postal Code
40600
Country
Kenya
Facility Name
International Cancer Institute
City
Eldoret
ZIP/Postal Code
30100
Country
Kenya
Facility Name
Kenya medical Research Institute-centre for Respiratory Disease Research
City
Nairobi
ZIP/Postal Code
00100
Country
Kenya
Facility Name
Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES)
City
Nairobi
ZIP/Postal Code
00200
Country
Kenya
Facility Name
American University of Beirut Medical Center
City
Hamra
State/Province
Beirut
Country
Lebanon
Facility Name
Nini Hospital
City
Tripoli
State/Province
North Lebanon
Country
Lebanon
Facility Name
University of Calabar Teaching Hospital
City
Calabar
State/Province
Cross River State
ZIP/Postal Code
540242
Country
Nigeria
Facility Name
National Hospital Abuja
City
Abuja
State/Province
FCT
ZIP/Postal Code
900211
Country
Nigeria
Facility Name
University of Abuja Teaching Hospital
City
Gwagwalada
State/Province
FCT
ZIP/Postal Code
902101
Country
Nigeria
Facility Name
Ahmadu Bello University Teaching Hospital (ABUTH)
City
Zaria
State/Province
Kaduna
ZIP/Postal Code
810107
Country
Nigeria
Facility Name
University of Nigeria Teaching Hospital
City
Enugu
ZIP/Postal Code
460000
Country
Nigeria
Facility Name
Barau Dikko Teaching Hospital/Kaduna State University
City
Kaduna
ZIP/Postal Code
800212
Country
Nigeria
Facility Name
Aminu Kano Teaching Hospital
City
Kano
ZIP/Postal Code
700223
Country
Nigeria
Facility Name
Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
City
Lagos
ZIP/Postal Code
100254
Country
Nigeria
Facility Name
Sultan Qaboos University Hospital
City
Muscat
ZIP/Postal Code
123
Country
Oman
Facility Name
Prince Mohammed bin Nasser Hospital
City
Jizan
State/Province
Southern
ZIP/Postal Code
82943
Country
Saudi Arabia
Facility Name
Hacettepe University
City
Ankara
State/Province
Altindag/sihhiye
ZIP/Postal Code
06230
Country
Turkey
Facility Name
Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi
City
Yenischir, Mersin
State/Province
Mersin
ZIP/Postal Code
33343
Country
Turkey
Facility Name
Baskent University Hospital
City
Adana
State/Province
Yuregir
ZIP/Postal Code
01250
Country
Turkey
Facility Name
Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Facility Name
Guy's & Thomas' NHS Foundation Trust
City
London
State/Province
England
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
Facility Name
Matero Clinical Research Site,
City
Lusaka
ZIP/Postal Code
10101
Country
Zambia
Facility Name
University Teaching Hospital- Children's Hospital
City
Lusaka
ZIP/Postal Code
10101
Country
Zambia
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=GBT2104-132
Description
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Learn more about this trial
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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