A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

An Open-label Extension Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease Who Have Participated in an Inclacumab Clinical Trial

This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.

Study Overview

• Status: Terminated
• Conditions: Sickle Cell Disease; Vaso-occlusive Pain Episode in Sickle Cell Disease; Vaso-occlusive Crisis
• Intervention / Treatment: Drug: Inclacumab

Detailed Description

The study will include approximately 520 adult and adolescent participants (≥ 12 years of age) with SCD.

All participants will receive inclacumab 30 mg/kg administered intravenously every 12 weeks.

• Study Type: Interventional
• Enrollment (Actual): 242
• Phase: Phase 3

Contacts and Locations

Study Locations

• Brazil
  • Rio de Janeiro, Brazil, 20211-030
    • Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO
  • São Paulo, Brazil, 08270-070
    • Casa de Saude Santa Marcelina
  • São Paulo, Brazil, 01232-010
    • Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A
  • São Paulo, Brazil, 08270-120
    • CEPEC-Centro de Pesquisa Clinica
  • Estado de Bahia
    • Salvador, Estado de Bahia, Brazil, 41253-190
      • Instituto D'Or de Pesquisa e Ensino - Hospital Sao Rafael
  • Pernambuco
    • Recife, Pernambuco, Brazil, 50070-460
      • Multihemo Servicos Medicos S/A
  • Rio Grande do Sul
    • Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
      • Hospital De Clinicas De Porto Alegre
  • São Paulo
    • Ribeirão Preto, São Paulo, Brazil, 14051-140
      • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
    • São José do Rio Preto, São Paulo, Brazil, 15090-000
      • Fundacao Faculdade Regional de Medicina de Sao Jose Do Rio Preto
• Colombia
  • Atlántico
    • Barranquilla, Atlántico, Colombia, 080020
      • Clínica de la costa Ltda.
  • Cesar Department
    • Valledupar, Cesar Department, Colombia, 200001
      • Sociedad de Oncologia y Hematologia del Cesar
• Kenya
  • Eldoret, Kenya, 30100
    • International Cancer Institute
  • Nairobi, Kenya, 00100
    • Gertrude's Children's Hospital
  • Nairobi, Kenya, 00100
    • Kenya Medical Research Institute - Centre for Respiratory Disease Research
  • Nairobi, Kenya, 00200
    • Strathmore University CREATES
  • Siaya County
    • Kisumu, Siaya County, Kenya, 40600
      • KEMRI/CRDR Siaya Clinical Research Annex
• Lebanon
  • Beyrouth
    • Hamra, Beyrouth, Lebanon
      • American University of Beirut Medical Center
  • North Lebanon
    • Tripoli, North Lebanon, Lebanon
      • Nini Hospital
• Nigeria
  • Enugu, Nigeria, 460000
    • University of Nigeria Teaching Hospital
  • Kaduna, Nigeria, 800212
    • Barau Dikko Teaching Hospital/Kaduna State University
  • Kano, Nigeria, 700223
    • Aminu Kano Teaching Hospital
  • Lagos, Nigeria, 100254
    • Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
  • Cross River State
    • Calabar, Cross River State, Nigeria, 540242
      • University of Calabar Teaching Hospital
  • Federal Capital Territory
    • Abuja, Federal Capital Territory, Nigeria, 900211
      • National Hospital Abuja
    • Gwagwalada, Federal Capital Territory, Nigeria, 902101
      • University of Abuja Teaching Hospital
  • Kaduna State
    • Zaria, Kaduna State, Nigeria, 810105
      • Ahmadu Bello University Teaching Hospital
• Oman
  • Muscat, Oman, 123
    • Sultan Qaboos University Hospital
• Saudi Arabia
  • Southern
    • Jizan, Southern, Saudi Arabia, 82943
      • Prince Mohammed bin Nasser Hospital
• Tanzania
  • Dar es Salaam, Tanzania, 11101
    • National Institute for Medical Research (NIMR)
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01130
    • Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
  • Adana, Turkey (Türkiye), 01250
    • Baskent University Adana Appl. and Research Central, Yuregir Baskent Hospital Hematology
  • Ankara, Turkey (Türkiye), 06430
    • Hacettepe University Ihsan Dogramaci Children Hospital
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • University of South Alabama Children's and Women's Hospital
    • Mobile, Alabama, United States, 36604
      • Strada Patient Care Center, Pediatric Hematology
    • Mobile, Alabama, United States, 36606
      • South Alabama Medical Science Foundation
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Orange, California, United States, 92868
      • UC Irvine Medical Center
  • Connecticut
    • Farmington, Connecticut, United States, 06030
      • UConn-Neag Comprehensive Cancer Center
  • Illinois
    • Chicago, Illinois, United States, 60612
      • University of Illinois Hospital and Health Sciences System (UI Health)
    • Chicago, Illinois, United States, 60612
      • University of Illinois at Chicago (UIC) Clinical Research Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
    • Boston, Massachusetts, United States, 02115
      • Brigham and Woman's Hospital
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute IDS Pharmacy
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Hospitals - Michigan Medicine
  • New York
    • The Bronx, New York, United States, 10461
      • Jacobi Medical Center
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Duke University Medical Center
    • Durham, North Carolina, United States, 27710
      • DUMC Investigational Drug Services Pharmacy
  • Texas
    • Houston, Texas, United States, 77030
      • McGovern Medical School at UTHealth
    • Houston, Texas, United States, 77030
      • UT Physicians Comprehensive Sickle Cell Center Houston
    • Houston, Texas, United States, 77030
      • UT Health Science Center at Houston - Clinical Research Unit at Memorial Hermann Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Participants who meet all the following criteria will be eligible for study enrollment:

1. Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study.
2. Participant has completed the originating inclacumab study within 30 calendar days of the Day 1 Visit. Participants who discontinued study drug in the originating study due to a non-study drug-related AE, but who remained on study, may be eligible for treatment in this study provided the AE does not pose a risk for treatment with inclacumab.

3. Female participants of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1.

   Note: Female participants who become of childbearing potential during the study must be willing to have a negative urine pregnancy test to remain in the study.

4. If sexually active, female participants of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 165 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 165 days after the last dose of study drug.
5. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirement.

Exclusion Criteria:

Participants meeting any of the following exclusion criteria will not be eligible for study enrollment:

1. Female participant who is breastfeeding or pregnant.
2. Participant had an infusions-related reaction (IRR) in the originating inclacumab clinical study.
3. Participant withdrew consent from the originating inclacumab clinical study.
4. Participant was lost to follow-up from the originating inclacumab clinical study.
5. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Inclacumab 30 mg/kg; Inclacumab 30 mg/kg administered intravenously (IV)	Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-emergent adverse events (TEAEs).	Day 1 through study completion, an estimate of 5 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Annualized rate of VOCs	Day 1 through study completion, an estimate of 5 years
Annualized rate of VOCs that require admission to a healthcare facility and treatment.	Day 1 through study completion, an estimate of 5 years
Annualized number of days of inpatient hospitalization for a VOC.	Day 1 through study completion, an estimate of 5 years
Annualized rate of all SCD-related urgent care visits to the clinic, emergency room, and hospital.	Day 1 through study completion, an estimate of 5 years
Proportion of total days missed from school or work due to SCD pain symptoms for the first 48 weeks.	Day 1 through study completion, an estimate of 5 years
Annualized rate of complicated VOCs.	Day 1 through study completion, an estimate of 5 years
Annualized rate of RBC transfusions.	Day 1 through study completion, an estimate of 5 years

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma pharmacokinetic (PK) of inclacumab as assessed by population PK analysis using nonlinear mixed-effects modeling.	Inclacumab concentrations will be measured from plasma samples. Population PK analysis using nonlinear mixed effects modeling will be performed to characterize inclacumab PK in plasma.	Day 1 through Week 48
Incidence of anti-drug antibodies (ADA) to inclacumab.		Day 1 through Week 48
PD parameter (P-selectin inhibition)		Day 1 through Week 48
PD parameter (Platelet Leukocyte Aggregation)		Day 1 through Week 48

Collaborators and Investigators

• Sponsor: Pfizer
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): March 29, 2022
• Primary Completion (Actual): November 7, 2025
• Study Completion (Actual): November 7, 2025

Study Registration Dates

• First Submitted: March 29, 2022
• First Submitted That Met QC Criteria: April 21, 2022
• First Posted (Actual): April 27, 2022

Study Record Updates

• Last Update Posted (Estimated): December 17, 2025
• Last Update Submitted That Met QC Criteria: December 11, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: hemoglobin; Sickle Cell Disease; SCD; blood disorders; red blood cells; sickle-like shape; mutation in hemoglobin gene; VOC; SCA; RBCs; Open Label; Vaso-occlusive Crisis
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; Hematologic Diseases; Vaso-Occlusive Crises; inclacumab
• Other Study ID Numbers: GBT2104-133; 2020-005289-32 (EudraCT Number); C5361003 (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No