Sickle Cell DiseasePhase 3NCT05348915
A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
A Phase 3 study testing Inclacumab in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by Pfizer at 50 sites in 10 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- What is being tested?
- Inclacumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 242 participants
- Where
- 50 sitesin 10 countries
- Study dates
- 29 Mar 2022to 7 Nov 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Participants meeting any of the following exclusion criteria will not be eligible for study enrollment:
- 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.
Trial locations and contacts
50 sites in 10 countries.
50 sites in 10 countries
United States18 sites
University of Michigan Hospitals - Michigan Medicine
Ann Arbor, Michigan, 48109
Brigham And Woman's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute IDS Pharmacy
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
University of Illinois at Chicago (UIC) Clinical Research Center
Chicago, Illinois, 60612
University of Illinois Hospital and Health Sciences System (UI Health)
Chicago, Illinois, 60612
Duke University Medical Center
Durham, North Carolina, 27705
DUMC Investigational Drug Services Pharmacy
Durham, North Carolina, 27710
UConn-Neag Comprehensive Cancer Center
Farmington, Connecticut, 06030
McGovern Medical School at UTHealth
Houston, Texas, 77030
UT Health Science Center at Houston - Clinical Research Unit at Memorial Hermann Hospital
Houston, Texas, 77030
UT Physicians Comprehensive Sickle Cell Center Houston
Houston, Texas, 77030
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Strada Patient Care Center, Pediatric Hematology
Mobile, Alabama, 36604
University of South Alabama Children's and Women's Hospital
Mobile, Alabama, 36604
South Alabama Medical Science Foundation
Mobile, Alabama, 36606
UC Irvine Medical Center
Orange, California, 92868
Jacobi Medical Center
The Bronx, New York, 10461
Brazil9 sites
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
Multihemo Servicos Medicos S/A
Recife, Pernambuco, 50070-460
Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP
Ribeirão Preto, São Paulo, 14051-140
Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO
Rio de Janeiro, 20211-030
Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael
Salvador, Estado de Bahia, 41253-190
Fundação Faculdade Regional de Medicina de São José do Rio Preto
São José do Rio Preto, São Paulo, 15090-000
Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A
São Paulo, 01232-010
Casa de Saude Santa Marcelina
São Paulo, 08270-070
CEPEC-Centro de Pesquisa Clinica
São Paulo, 08270-120
Nigeria8 sites
National Hospital Abuja
Abuja, Federal Capital Territory, 900211
University of Calabar Teaching Hospital
Calabar, Cross River State, 540242
University of Nigeria Teaching Hospital
Enugu, 460000
University of Abuja Teaching Hospital
Gwagwalada, Federal Capital Territory, 902101
Barau Dikko Teaching Hospital/Kaduna State University
Kaduna, 800212
Aminu Kano Teaching Hospital
Kano, 700223
Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital
Lagos, 100254
Ahmadu Bello University Teaching Hospital
Zaria, Kaduna State, 810105
Kenya5 sites
International Cancer Institute
Eldoret, 30100
KEMRI/CRDR Siaya Clinical Research Annex
Kisumu, Siaya County, 40600
Gertrude's Children's Hospital
Nairobi, 00100
Kenya Medical Research Institute - Centre for Respiratory Disease Research
Nairobi, 00100
Strathmore University CREATES
Nairobi, 00200
Turkey (Türkiye)3 sites
Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji
Adana, 01130
Baskent University Adana Appl. and Research Central, Yuregir Baskent Hospital Hematology
Adana, 01250
Hacettepe University Ihsan Dogramaci Children Hospital
Ankara, 06430
Colombia2 sites
Clinica de la Costa Ltda.
Barranquilla, Atlántico, 080020
Sociedad de Oncología y hematología del Cesar
Valledupar, Cesar Department, 200001
Lebanon2 sites
American University of Beirut Medical Center
Hamra, Beirut
Nini Hospital
Tripoli, North Lebanon
Saudi Arabia1 site
Prince Mohammed bin Nasser Hospital
Jizan, Southern, 82943
Tanzania1 site
National Institute for Medical Research (NIMR)
Dar es Salaam, 11101
About this study
Official title: 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.
Read the 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.
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
- Being tested
- Inclacumab
- 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
- Medical subject headings
- Anemia, Sickle Cell, Vaso-Occlusive Crises, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InclacumabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Inclacumab 30 mg/kg | Experimental | Inclacumab 30 mg/kg administered intravenously (IV) Receives: Drug: Inclacumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs).
Measured: Day 1 through study completion, an estimate of 5 years
Secondary outcomes (7)
Annualized rate of VOCs
Measured: Day 1 through study completion, an estimate of 5 years
Annualized rate of VOCs that require admission to a healthcare facility and treatment.
Measured: Day 1 through study completion, an estimate of 5 years
Annualized number of days of inpatient hospitalization for a VOC.
Measured: 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.
Measured: 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.
Measured: Day 1 through study completion, an estimate of 5 years
Annualized rate of complicated VOCs.
Measured: Day 1 through study completion, an estimate of 5 years
Annualized rate of RBC transfusions.
Measured: Day 1 through study completion, an estimate of 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2022
- Study start29 Mar 2022
- Primary completion7 Nov 2025
- Study completion7 Nov 2025
Why it stopped: Sponsor decision to terminate study based on results of parent study GBT2104-131.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 242 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05348915
- Sponsor's protocol ID
- GBT2104-133
- Other IDs
- 2020-005289-32, C5361003
- Status last verified
- Dec 2025
- First submitted
- 29 Mar 2022
- Last updated
- 17 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05348915
- Acronym / sponsor ID
- GBT2104-133
- 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
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate study based on results of parent study GBT2104-131.
- Enrolment
- 242 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 29 Mar 2022 (actual)
- Primary completion
- 7 Nov 2025 (actual)
- Study completion
- 7 Nov 2025 (actual)
- First posted
- 27 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Inclacumab 30 mg/kg | Experimental | Drug: InclacumabInclacumab 30 mg/kg administered intravenously (IV) |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (6 listed)
- Participants meeting any of the following exclusion criteria will not be eligible for study enrollment:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| InclacumabInclacumab 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. | Drug | Inclacumab 30 mg/kg |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs).
Time frame: Day 1 through study completion, an estimate of 5 years
7 secondary outcomes
Annualized rate of VOCs
Time frame: Day 1 through study completion, an estimate of 5 years
Annualized rate of VOCs that require admission to a healthcare facility and treatment.
Time frame: Day 1 through study completion, an estimate of 5 years
Annualized number of days of inpatient hospitalization for a VOC.
Time frame: 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.
Time frame: 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.
Time frame: Day 1 through study completion, an estimate of 5 years
Annualized rate of complicated VOCs.
Time frame: Day 1 through study completion, an estimate of 5 years
Annualized rate of RBC transfusions.
Time frame: Day 1 through study completion, an estimate of 5 years
Results and safety
The study was stopped early (reason given: Sponsor decision to terminate study based on results of parent study GBT2104-131.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2025.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
50 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Inclacumab in people with Sickle Cell Disease and Vaso-occlusive Crisis, run by Pfizer at 50 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 17 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, Brazil, Nigeria, Kenya, Turkey (Türkiye) and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision to terminate study based on results of parent study GBT2104-131.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs). (time frame: Day 1 through study completion, an estimate of 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Dec 2025.