NCT04935879|CompletedPhase 3ResultsDMCFDA Drug

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

Pfizer ClinicalTrials.gov

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3 other identifiers

GBT2104-1312020-005286-13C5361001

Study Type

interventional

Target

241

Locations

12 countries

Sites

Timeline

RegisteredJun 2021

StartedOct 2021

CompletionJun 2024

Brief Summary

This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

241

participants targeted

Target at P50-P75 for phase_3

Timeline

Completed

Started Oct 2021

Typical duration for phase_3

Geographic Reach

12 countries

58 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.7 years study duration

First Submitted

Initial submission to the registry

May 26, 2021

Completed

28 days until next milestone

First Posted

Study publicly available on registry

June 23, 2021

Completed

3 months until next milestone

Study Start

First participant enrolled

October 4, 2021

Completed

2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 6, 2024

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 6, 2024

Completed

1.5 years until next milestone

Results Posted

Study results publicly available

December 2, 2025

Completed

Last Updated

December 2, 2025

Status Verified

November 1, 2025

Enrollment Period

2.7 years

First QC Date

May 26, 2021

Results QC Date

October 30, 2025

Last Update Submit

November 17, 2025

Conditions

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

Keywords

blood disordershemoglobinred blood cellssickle-like shapemutation in hemoglobin genesickle-cell traitsickle-cell crisisSickle Cell DiseaseSCDVaso-occlusive CrisesVOCSCARBCs

Outcome Measures

Primary Outcomes (1)

Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48
A VOC was defined as an acute episode of pain that: had no medically determined cause other than a vaso-occlusive event; resulted in a visit to a medical facility (hospitalization, emergency department, urgent care center, outpatient clinic, or infusion center), or resulted in a remote contact with a healthcare provider and required parenteral narcotic agents, parenteral nonsteroidal anti-inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics. The rate of VOC was defined as number of VOC events per 48 weeks and presented in this outcome measure.
Randomization (Day 1) up to Week 48

Secondary Outcomes (6)

Time to First VOC Through Week 48
Randomization (Day 1) up to Week 48
Time to Second VOC Through Week 48
Randomization (Day 1) up to Week 48
Percentage of Participants With no VOCs Through Week 48
Randomization (Day 1) up to Week 48
Rate of VOCs Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication [Adjudicated] Through Week 48
Randomization (Day 1) up to Week 48
Rate of Inpatient Hospitalization Days for a VOC Through Week 48
Randomization (Day 1) up to Week 48
+1 more secondary outcomes

Study Arms (2)

inclacumab, 30 mg/kg

EXPERIMENTAL

Participants will receive inclacumab 30 mg/kg administered IV every 12 weeks

Drug: Inclacumab

placebo

PLACEBO COMPARATOR

Participants will receive placebo administered IV every 12 weeks.

Drug: Placebo

Interventions

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.

inclacumab, 30 mg/kg

PlaceboDRUG

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

Age12 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Participant has a confirmed diagnosis of SCD (HbSS, HbSC, HbSB0 thalassemia, or HbSB+ thalassemia genotype).
Documentation of SCD genotype is required and may be based on documented history of laboratory testing or confirmed by laboratory testing during Screening.
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 the Screening Visit as determined by documented medical history. A prior VOC is defined as an acute episode of pain which:
Has no medically determined cause other than a vaso-occlusive event, and
Results in a visit to a medical facility (hospital, emergency department, urgent care center, outpatient clinic, or infusion center) or results in a remote contact with a healthcare provider; and
Requires parenteral narcotic agents, parenteral nonsteroidal anti- inflammatory drugs (NSAIDs), or an increase in treatment with oral narcotics.
Participants receiving erythropoiesis-stimulating agents (ESA, e.g., erythropoietin \[EPO\]) must be on a stable dose for at least 90 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.
Participants receiving hydroxyurea (HU), L-glutamine, or voxelotor (Oxbryta®) must be on a stable dose for at least 30 days prior to the Screening Visit and expected to continue with the stabilized regimen throughout the course of the study.

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 the Screening Visit
Participant weighs \> 133 kg (292 lbs.).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pfizerlead

Study Sites (58)

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

University of South Alabama Strada Patient Care Center

Mobile, Alabama, 36604, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

UC Irvine Health

Orange, California, 92868, United States

Location

UCI Center for clinical research

Orange, California, 92868, United States

Location

Uconn Health/Uconn John Dempsey Hospital/Neag Comprehensive Cancer Center/New England Sickle Cell

Farmington, Connecticut, 06030, United States

Location

Hospital Pharmacy Services- Investigational Drug Services