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

NCT04927247 | Terminated Phase 3 Results DMC FDA Drug

• 3 other identifiers: GBT2104-132C53610022020-005287-60
• Study Type: interventional
• Target: 72
• Locations: 14 countries
• Sites: 59

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.

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

Outcome Measures

Primary Outcomes (1)

• 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

  A VOC was a complication of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. An admission for a VOC included 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. An acute episode of pain with no other cause other than a vaso-occlusive event included: uncomplicated VOC, acute chest syndrome, hepatic sequestration, splenic sequestration, or priapism. All on-study VOCs were adjudicated by an independent, blinded VOC Adjudication Committee comprised of experts in SCD.

  Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91])

Secondary Outcomes (3)

• Time to First VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization

  Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91]) or censored day, whichever occurred earlier

• 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

  Within 30 days of randomization (randomization happened on Day 1 [Day 1 to Day 31])

• 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

  Within 90 days of randomization (randomization happened on Day 1 [Day 1 to Day 91])

Study Arms (2)

inclacumab 30 mg/kg

EXPERIMENTAL

Inclacumab 30 mg/kg administered intravenously (IV)

Drug: Inclacumab

placebo

PLACEBO COMPARATOR

Placebo administered IV

Drug: Placebo

Interventions

Inclacumab 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.

inclacumab 30 mg/kg

Placebo 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.

placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.).

Sponsors & Collaborators

• Pfizer: lead

Study Sites (59)

Strada Patient Care Center

Mobile, Alabama, 36604, United States

Location

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

Mobile, Alabama, 36604, United States

Location

University of South Alabama Mitchell Cancer Institute

Mobile, Alabama, 36604, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UCSF Benioff Children's Hospital, Oakland

Oakland, California, 94609, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

St. Joseph's Hospital

Tampa, Florida, 33607, United States

Location

University of Michigan Hospitals - Michigan Medicine

Ann Arbor, Michigan, 48109, United States

Location

Functional Fluidics, Inc.

Detroit, Michigan, 48202, United States

Location

Alliance for Childhood Diseases dba Cure 4 The Kids Foundation

Las Vegas, Nevada, 89135, United States

Location

Erie County Medical Center

Buffalo, New York, 14215, United States

Location

Jacobi Medical Center

The Bronx, New York, 10461, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

DUMC Investigational Drug Services Pharmacy

Durham, North Carolina, 27710, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

PPD Bioanalytical

Richmond, Virginia, 23230, United States

Location

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

Salvador, Estado de Bahia, 41253-190, Brazil

Location

Multihemo Servicos Medicos S/A

Recife, Pernambuco, 50070-460, Brazil

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Fundação Faculdade Regional de Medicina de São José do Rio Preto

São José do Rio Preto, São Paulo, 15090-000, Brazil

Location

Casa de Saude Santa Marcelina

São Paulo, São Paulo, 08270-070, Brazil

Location

Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, 20211-030, Brazil

Location

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

São Paulo, 05403-000, Brazil

Location

CEPEC-Centro de Pesquisa Clinica

São Paulo, 08270-120, Brazil

Location

Clinica de la Costa Ltda.

Barranquilla, Atlántico, 080020, Colombia

Location

Organizacion Clinica Bonnadona Prevenir S.A.S.

Barranquilla, Atlántico, 080020, Colombia

Location

Hopital Avicenne

Bobigny, 93000, France

Location

Hopital Henri Mondor

Créteil, 94010, France

Location

Universitatsklinikum Regensburg Padiatrische Hamatologie, Onkologie und Stammzelltransplantation

Regensburg, 93053, Germany

Location

Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera

Genova, Genoa, 16128, Italy

Location

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

Genova, 16128, Italy

Location

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

Napoli, 80138, Italy

Location

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

Napoli, 80138, Italy

Location

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

Napoli, 80138, Italy

Location

UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"

Napoli, 80138, Italy

Location

Kemri/Crdr, Siaya, Kemri Clinical Research Annex,

Kisumu, Siaya County, 40600, Kenya

Location

International Cancer Institute

Eldoret, 30100, Kenya

Location

Kenya medical Research Institute-centre for Respiratory Disease Research

Nairobi, 00100, Kenya

Location

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

Nairobi, 00200, Kenya

Location

American University of Beirut Medical Center

Hamra, Beyrouth, Lebanon

Location

Nini Hospital

Tripoli, North Lebanon, Lebanon

Location

University of Calabar Teaching Hospital

Calabar, Cross River State, 540242, Nigeria

Location

National Hospital Abuja

Abuja, Federal Capital Territory, 900211, Nigeria

Location

University of Abuja Teaching Hospital

Gwagwalada, Federal Capital Territory, 902101, Nigeria

Location

Ahmadu Bello University Teaching Hospital (ABUTH)

Zaria, Kaduna State, 810107, Nigeria

Location

University of Nigeria Teaching Hospital

Enugu, 460000, Nigeria

Location

Barau Dikko Teaching Hospital/Kaduna State University

Kaduna, 800212, Nigeria

Location

Aminu Kano Teaching Hospital

Kano, 700223, Nigeria

Location

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

Lagos, 100254, Nigeria

Location

Sultan Qaboos University Hospital

Muscat, 123, Oman

Location

Prince Mohammed bin Nasser Hospital

Jizan, Southern, 82943, Saudi Arabia

Location

Hacettepe University

Ankara, Altindag/sihhiye, 06230, Turkey (Türkiye)

Location

Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi

Yenischir, Mersin, Mersin, 33343, Turkey (Türkiye)

Location

Baskent University Hospital

Adana, Yuregir, 01250, Turkey (Türkiye)

Location

Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji

Adana, 01130, Turkey (Türkiye)

Location

Guy's & Thomas' NHS Foundation Trust

London, England, SE1 9RT, United Kingdom

Location

Matero Clinical Research Site,

Lusaka, 10101, Zambia

Location

University Teaching Hospital- Children's Hospital

Lusaka, 10101, Zambia

Location

Related Links

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

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises; Hematologic Diseases; Sickle Cell Trait

Interventions

inclacumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double blind study
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2021
• First Posted: June 15, 2021
• Study Start: December 9, 2021
• Primary Completion: November 24, 2023
• Study Completion: November 24, 2023
• Last Updated: December 11, 2024
• Results First Posted: December 11, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will share

Locations

NG (8); BR (8); OM (1); CO (2); SA (1); IT (6); US (17); LE (2); KE (4); TU (4); GB (1); FR (2); DE (1); ZA (2)