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

NCT05348915 | Terminated Phase 3 DMC FDA Drug

• 3 other identifiers: GBT2104-1332020-005289-32C5361003
• Study Type: interventional
• Target: 242
• Locations: 10 countries
• Sites: 50

Brief Summary

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.

Conditions

Sickle Cell Disease; Vaso-occlusive Crisis; Vaso-occlusive Pain Episode in Sickle Cell Disease

Keywords

blood disorders; hemoglobin; red blood cells; RBCs; sickle-like shape; mutation in hemoglobin gene; Sickle Cell Disease; SCD; Vaso-occlusive Crisis; VOC; SCA; Open Label

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs).

  Day 1 through study completion, an estimate of 5 years

Secondary Outcomes (7)

• 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

Other Outcomes (4)

• Plasma pharmacokinetic (PK) of inclacumab as assessed by population PK analysis using nonlinear mixed-effects modeling.

  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

Study Arms (1)

Inclacumab 30 mg/kg

EXPERIMENTAL

Inclacumab 30 mg/kg administered intravenously (IV)

Drug: Inclacumab

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

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:

• Participants who meet all the following criteria will be eligible for study enrollment:
• Male or female participant with SCD who participated and received study drug in a GBT-Sponsored inclacumab clinical study.
• 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.
• 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.
• 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.
• 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.

You may not qualify if:

• Female participant who is breastfeeding or pregnant.
• Participant had an infusions-related reaction (IRR) in the originating inclacumab clinical study.
• Participant withdrew consent from the originating inclacumab clinical study.
• Participant was lost to follow-up from the originating inclacumab clinical study.
• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (50)

Strada Patient Care Center, Pediatric Hematology

Mobile, Alabama, 36604, United States

Location

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

Mobile, Alabama, 36604, United States

Location

South Alabama Medical Science Foundation

Mobile, Alabama, 36606, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UC Irvine Medical Center

Orange, California, 92868, United States

Location

UConn-Neag Comprehensive Cancer Center

Farmington, Connecticut, 06030, United States

Location

University of Illinois at Chicago (UIC) Clinical Research Center

Chicago, Illinois, 60612, United States

Location

University of Illinois Hospital and Health Sciences System (UI Health)

Chicago, Illinois, 60612, United States

Location

Brigham And Woman's Hospital

Boston, Massachusetts, 02115, United States

Location

Dana-Farber Cancer Institute IDS Pharmacy

Boston, Massachusetts, 02215, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan Hospitals - Michigan Medicine

Ann Arbor, Michigan, 48109, 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

McGovern Medical School at UTHealth

Houston, Texas, 77030, United States

Location

UT Health Science Center at Houston - Clinical Research Unit at Memorial Hermann Hospital

Houston, Texas, 77030, United States

Location

UT Physicians Comprehensive Sickle Cell Center Houston

Houston, Texas, 77030, 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 Clínicas de Porto Alegre

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

Location

Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP

Ribeirão Preto, São Paulo, 14051-140, Brazil

Location

Fundação Faculdade Regional de Medicina de São José do Rio Preto

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

Location

Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, 20211-030, Brazil

Location

Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A

São Paulo, 01232-010, Brazil

Location

Casa de Saude Santa Marcelina

São Paulo, 08270-070, Brazil

Location

CEPEC-Centro de Pesquisa Clinica

São Paulo, 08270-120, Brazil

Location

Clinica de la Costa Ltda.

Barranquilla, Atlántico, 080020, Colombia

Location

Sociedad de Oncología y hematología del Cesar

Valledupar, Cesar Department, 200001, Colombia

Location

KEMRI/CRDR Siaya Clinical Research Annex

Kisumu, Siaya County, 40600, Kenya

Location

International Cancer Institute

Eldoret, 30100, Kenya

Location

Gertrude's Children's Hospital

Nairobi, 00100, Kenya

Location

Kenya Medical Research Institute - Centre for Respiratory Disease Research

Nairobi, 00100, Kenya

Location

Strathmore University 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

Zaria, Kaduna State, 810105, 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

National Institute for Medical Research (NIMR)

Dar es Salaam, 11101, Tanzania

Location

Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji

Adana, 01130, Turkey (Türkiye)

Location

Baskent University Adana Appl. and Research Central, Yuregir Baskent Hospital Hematology

Adana, 01250, Turkey (Türkiye)

Location

Hacettepe University Ihsan Dogramaci Children Hospital

Ankara, 06430, Turkey (Türkiye)

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

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

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 29, 2022
• First Posted: April 27, 2022
• Study Start: March 29, 2022
• Primary Completion: November 7, 2025
• Study Completion: November 7, 2025
• Last Updated: December 17, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

LE (2); NG (8); TU (3); CO (2); OM (1); BR (9); KE (5); SA (1); US (18); TA (1)