Clinical trial NCT04927247: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: 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
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-06-15
Enrollment: 72
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization
timeFrame: Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91])
Secondary outcomes:
measure: Time to First VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization
timeFrame: Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]) or censored day, whichever occurred earlier
measure: Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 30 Days of Randomization
timeFrame: Within 30 days of randomization (randomization happened on Day 1 [Day 1 to Day 31])
measure: Rate of VOCs Leading to a Healthcare Visit That Requires Parenteral Pain Medication or an Increase in Treatment With Oral Narcotics Within 90 Days Following Randomization
timeFrame: Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91])
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Strada Patient Care Center
state: Alabama
city: Mobile
country: United States
facility: University of South Alabama Children's and Women's Hospital
state: Alabama
city: Mobile
country: United States
facility: University of South Alabama Mitchell Cancer Institute
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital, Oakland
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Tampa
country: United States
facility: St. Joseph's Hospital
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan Hospitals - Michigan Medicine
state: Michigan
city: Detroit
country: United States
facility: Functional Fluidics, Inc.
state: Michigan
city: Las Vegas
country: United States
facility: Alliance for Childhood Diseases dba Cure 4 The Kids Foundation
state: Nevada
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Buffalo
country: United States
facility: Erie County Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Durham
country: United States
facility: DUMC Investigational Drug Services Pharmacy
state: North Carolina
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Richmond
country: United States
facility: PPD Bioanalytical
state: Virginia
city: Salvador
country: Brazil
facility: Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael
state: Bahia
city: Recife
country: Brazil
facility: Multihemo Servicos Medicos S/A
state: PE
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre
state: RS
city: São José Do Rio Preto
country: Brazil
facility: Fundação Faculdade Regional de Medicina de São José do Rio Preto
state: SP
city: São Paulo
country: Brazil
facility: Casa de Saude Santa Marcelina
state: SP
city: Rio de Janeiro
country: Brazil
facility: Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO
city: São Paulo
country: Brazil
facility: Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP
city: São Paulo
country: Brazil
facility: CEPEC-Centro de Pesquisa Clinica
city: Barranquilla
country: Colombia
facility: Clinica de la Costa Ltda.
state: Atlantico
city: Barranquilla
country: Colombia
facility: Organizacion Clinica Bonnadona Prevenir S.A.S.
state: Atlántico
city: Bobigny
country: France
facility: Hopital Avicenne
city: Créteil
country: France
facility: Hopital Henri Mondor
city: Regensburg
country: Germany
facility: Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation
city: Genova
country: Italy
facility: Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera
state: Genoa
city: Genova
country: Italy
facility: S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro
city: Napoli
country: Italy
facility: DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitell
city: Napoli
country: Italy
facility: UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria
city: Napoli
country: Italy
facility: UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
city: Napoli
country: Italy
facility: UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"
city: Kisumu
country: Kenya
facility: Kemri/Crdr, Siaya, Kemri Clinical Research Annex,
state: Siaya
city: Eldoret
country: Kenya
facility: International Cancer Institute
city: Nairobi
country: Kenya
facility: Kenya medical Research Institute-centre for Respiratory Disease Research
city: Nairobi
country: Kenya
facility: Strathmore University Medical Center - Center for Research in Therapeutic Sciences(CREATES)
city: Hamra
country: Lebanon
facility: American University of Beirut Medical Center
state: Beirut
city: Tripoli
country: Lebanon
facility: Nini Hospital
state: North Lebanon
city: Calabar
country: Nigeria
facility: University of Calabar Teaching Hospital
state: Cross River State
city: Abuja
country: Nigeria
facility: National Hospital Abuja
state: FCT
city: Gwagwalada
country: Nigeria
facility: University of Abuja Teaching Hospital
state: FCT
city: Zaria
country: Nigeria
facility: Ahmadu Bello University Teaching Hospital (ABUTH)
state: Kaduna
city: Enugu
country: Nigeria
facility: University of Nigeria Teaching Hospital
city: Kaduna
country: Nigeria
facility: Barau Dikko Teaching Hospital/Kaduna State University
city: Kano
country: Nigeria
facility: Aminu Kano Teaching Hospital
city: Lagos
country: Nigeria
facility: Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
city: Muscat
country: Oman
facility: Sultan Qaboos University Hospital
city: Jizan
country: Saudi Arabia
facility: Prince Mohammed bin Nasser Hospital
state: Southern
city: Ankara
country: Turkey
facility: Hacettepe University
state: Altindag/sihhiye
city: Yenischir, Mersin
country: Turkey
facility: Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi
state: Mersin
city: Adana
country: Turkey
facility: Baskent University Hospital
state: Yuregir
city: Adana
country: Turkey
facility: Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
city: London
country: United Kingdom
facility: Guy's & Thomas' NHS Foundation Trust
state: England
city: Lusaka
country: Zambia
facility: Matero Clinical Research Site,
city: Lusaka
country: Zambia
facility: University Teaching Hospital- Children's Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: