A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
- Conditions
- Sickle Cell DiseaseVaso-occlusive Pain Episode in Sickle Cell DiseaseVaso-occlusive Crisis
- Interventions
- Drug: PlaceboDrug: Inclacumab
- Registration Number
- NCT04935879
- Lead Sponsor
- Pfizer
- 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.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 243
-
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.
-
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 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.
-
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.
-
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.
- 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 the Screening Visit
- Participant weighs > 133 kg (292 lbs.).
Other protocol-defined Inclusion/Exclusion may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description placebo Placebo Participants will receive placebo administered IV every 12 weeks. inclacumab, 30 mg/kg Inclacumab Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks
- Primary Outcome Measures
Name Time Method Rate of VOCs during the 48-week treatment period. 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
Name Time Method Incidence of treatment-emergent adverse events (TEAEs). Day 1- Week 48 Time to second VOC during the 48-week treatment period Efficacy. Day 1- Week 48 Number of days of inpatient hospitalization for a VOC during the 48-week treatment period. Day 1- Week 48 Time to first VOC during the 48-week treatment period. Day 1- Week 48 Proportion of participants with no VOCs during the 48-week treatment period. Day 1- Week 48 Rate of VOCs that required admission to a healthcare facility and treatment with parenteral pain medication during the 48-week treatment period. 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.
Trial Locations
- Locations (97)
UCI Center for clinical research
🇺🇸Orange, California, United States
University of South Alabama Children's and Women's Hospital
🇺🇸Mobile, Alabama, United States
University of South Alabama Mitchell Cancer Institute
🇺🇸Mobile, Alabama, United States
University of South Alabama Strada Patient Care Center
🇺🇸Mobile, Alabama, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital, Oakland
🇺🇸Oakland, California, United States
UC Irvine Health
🇺🇸Orange, California, United States
Uconn Health/Uconn John Dempsey Hospital/Neag Comprehensive Cancer Center/New England Sickle Cell
🇺🇸Farmington, Connecticut, United States
University of South Florida
🇺🇸Tampa, Florida, United States
USF Health South Tampa Center for Advanced Healthcare
🇺🇸Tampa, Florida, United States
John S. Curran, MD.,Children's Health Center
🇺🇸Tampa, Florida, United States
USF Clinical Investigational Research Pharmacy
🇺🇸Tampa, Florida, United States
USF Health Carol & Frank Morsani Center for Advanced Healthcare
🇺🇸Tampa, Florida, United States
Children's Healthcare of Atlanta - Egleston
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta - Scottish Rite
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Hospital Pharmacy Services- Investigational Drug Services
🇺🇸Chicago, Illinois, United States
Rush University Medical Center Investigator Pharmacy
🇺🇸Chicago, Illinois, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
University of Illinois Clinical Research Center (CRC)
🇺🇸Chicago, Illinois, United States
University of Illinois Hospital and Health Sciences System(UI Health)
🇺🇸Chicago, Illinois, United States
Dana-Farber Cancer Institute IDS Pharmacy
🇺🇸Boston, Massachusetts, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
University of Michigan Hospitals - Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Jacobi Medical Center
🇺🇸Bronx, New York, United States
Erie County Medical Center
🇺🇸Buffalo, New York, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
DUMC Investigational Drug Services Pharmacy
🇺🇸Durham, North Carolina, United States
St. Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States
UT Physicians Comprehensive Sickle Cell Clinic
🇺🇸Houston, Texas, United States
McGovern Medical School/Health Science Center Houston
🇺🇸Houston, Texas, United States
Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy
🇺🇸Houston, Texas, United States
Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU)
🇺🇸Houston, Texas, United States
Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael
🇧🇷Salvador, Bahia, Brazil
Hospital das Clinicas da Universidade Federal de Minas Gerais
🇧🇷Belo Horizonte, Minas Gerais, Brazil
Hemocentro de Belo Horizonte - Fundacao Hemominas
🇧🇷Belo Horizonte, MG, Brazil
Multihemo Serviços Médicos S/A
🇧🇷Recife, Pernambuco, Brazil
Hospital de Clínicas de Porto Alegre
🇧🇷Porto Alegre, RS, Brazil
Fundação Faculdade Regional de Medicina de São José do Rio Preto
🇧🇷Sao Jose do Rio Preto, SAO Paulo, Brazil
Comite de Etica em Pesquisa - CEP do Hospital Alemao Oswaldo Cruz/ SP
🇧🇷Sao Paulo, SP, Brazil
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
🇧🇷Ribeirão Preto, SÃO Paulo, Brazil
Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO
🇧🇷Rio de Janeiro, Brazil
Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A
🇧🇷São Paulo, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo-HCFMUSP
🇧🇷São Paulo, Brazil
Casa de Saúde Santa Marcelina
🇧🇷São Paulo, Brazil
CEPEC-Centro de Pesquisa Clinica
🇧🇷São Paulo, Brazil
Clinica de la Costa Ltda.
🇨🇴Barranquilla, Atlantico, Colombia
Organizacion Clinica Bonnadona Prevenir S.A.S.
🇨🇴Barranquilla, Atlántico, Colombia
Sociedad de Oncologia y hematologia del Cesar S.A.S.
🇨🇴Valledupar, Cesar, Colombia
Zagazig University Hospital
🇪🇬Zagazig, ASH Sharqia, Egypt
Al Kasr Al Ainy Cairo University Hospital
🇪🇬Elmanial, Cairo Governorate, Egypt
Cairo University Paediatric Hospital - abou el Reesh University Hospital
🇪🇬El-Rashidy Street, ElSayeda Zeinb, Cairo, Egypt
Faculty of Medicine Cairo University
🇪🇬Cairo, Egypt
AinShams University Hospital
🇪🇬Cairo, Egypt
Hôpital Avicenne
🇫🇷Bobigny, France
Hôpital Henri Mondor
🇫🇷Créteil, France
Institut Universitaire du Cancer de Toulouse-Oncopole
🇫🇷Toulouse Cedex 9, France
Universitätsklinikum Regensburg Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
🇩🇪Regensburg, Germany
Sickle-Cell Office, Directorate of Child Health, Komfo Anokye Teaching Hospital
🇬🇭Kumasi, Ashanti, Ghana
Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera
🇮🇹Genova, Italy
S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro Azienda
🇮🇹Genova, Italy
DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria
🇮🇹Napoli, Italy
DAI Materno-Infantile,- UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitel
🇮🇹Napoli, Italy
UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria
🇮🇹Napoli, Italy
UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
🇮🇹Napoli, Italy
UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
🇮🇹Napoli, Italy
Dipartimento Strutturale Aziendale Salute della Donna e del Bambino Clinica Ginecologica
🇮🇹Padova, Italy
Farmacia Azienda Ospedale Universita Padova
🇮🇹Padova, Italy
U.O.C. Farmacia Istituto Oncologico Veneto
🇮🇹Padova, Italy
KEMRI/CRDR, Siaya, KEMRI Clinical Research Annex
🇰🇪Kisumu, Siaya, Kenya
International Cancer Institute
🇰🇪Eldoret, Kenya
Gertrude's Children Hospital
🇰🇪Nairobi, Kenya
Kenya Medical Research Institute- Center for Respiratory Disease Research
🇰🇪Nairobi, Kenya
Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES)
🇰🇪Nairobi, Kenya
American University of Beirut Medical Center
🇱🇧Hamra, Beirut, Lebanon
Nini Hospital
🇱🇧Tripoli, North Lebanon, Lebanon
University of Calabar Teaching Hospital
🇳🇬Calabar, Cross River State, Nigeria
National Hospital Abuja
🇳🇬Abuja, FCT, Nigeria
University of Abuja Teaching Hospital
🇳🇬Gwagwalada, FCT, Nigeria
Ahmadu Bello University Teaching Hospital
🇳🇬Zaria, Kaduna, Nigeria
University of Nigeria Teaching Hospital
🇳🇬Enugu, Nigeria
Barau Dikko Teaching Hospital/Kaduna State University
🇳🇬Kaduna, Nigeria
Aminu Kano Teaching Hospital
🇳🇬Kano, Nigeria
Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
🇳🇬Lagos, Nigeria
Sultan َQaboos University Hospital
🇴🇲Muscat, Oman
Prince Mohammed bin Nasser Hospital
🇸🇦Jizan, Southern, Saudi Arabia
NIMR-Mbeya Medical Research Center
🇹🇿Mbeya, Tanzania
Hacettepe University
🇹🇷Ankara, Altindag/sihhiye, Turkey
Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi
🇹🇷Yenisehir, Mersin, Turkey
Baskent University Hospital
🇹🇷Adana, Yuregir, Turkey
Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
🇹🇷Adana, Turkey
Guy's and St Thomas' NHS Foundation Trust
🇬🇧London, London CITY OF, United Kingdom
King's College Hospital NHS Foundation Trust
🇬🇧London, London CITY OF, United Kingdom
Arthur Davison Childrens's Hospital
🇿🇲Ndola, Copperbelt, Zambia
Matero Clinical Research Site,
🇿🇲Lusaka, Zambia
University Teaching Hospital- Children's Hospital
🇿🇲Lusaka, Zambia