Clinical trial NCT05348915: A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
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
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2022-04-27
Enrollment: 147
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs).
timeFrame: Day 1 through study completion, an estimate of 5 years
Secondary outcomes:
measure: Annualized rate of VOCs
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Annualized rate of VOCs that require admission to a healthcare facility and treatment.
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Annualized number of days of inpatient hospitalization for a VOC.
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Annualized rate of all SCD-related urgent care visits to the clinic, emergency room, and hospital.
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Proportion of total days missed from school or work due to SCD pain symptoms for the first 48 weeks.
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Annualized rate of complicated VOCs.
timeFrame: Day 1 through study completion, an estimate of 5 years
measure: Annualized rate of RBC transfusions.
timeFrame: Day 1 through study completion, an estimate of 5 years
Other outcomes:
measure: Plasma pharmacokinetic (PK) of inclacumab as assessed by population PK analysis using nonlinear mixed-effects modeling.
timeFrame: Day 1 through Week 48
measure: Incidence of anti-drug antibodies (ADA) to inclacumab.
timeFrame: Day 1 through Week 48
measure: PD parameter (P-selectin inhibition)
timeFrame: Day 1 through Week 48
measure: PD parameter (Platelet Leukocyte Aggregation)
timeFrame: Day 1 through Week 48
Locations:
city: Mobile
country: United States
facility: University of South Alabama Children's and Women's Hospital
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Orange
country: United States
facility: UC Irvine Medical Center
state: California
city: Farmington
country: United States
facility: UConn-Neag Comprehensive Cancer Center
state: Connecticut
city: Chicago
country: United States
facility: University of Illinois Hospital and Health Sciences System (UI Health)
state: Illinois
city: Boston
country: United States
facility: Brigham And Woman's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Hospitals - Michigan Medicine
state: Michigan
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Houston
country: United States
facility: McGovern Medical School at UTHealth
state: Texas
city: Sao Jose do Rio Preto
country: Brazil
facility: Fundação Faculdade Regional de Medicina de São José do Rio Preto
state: SAO Paulo
city: São Paulo
country: Brazil
facility: Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A
city: Barranquilla
country: Colombia
facility: Clinica de la Costa Ltda.
state: Atlantico
city: Valledupar
country: Colombia
facility: Sociedad de Oncología y hematología del Cesar
state: Cesar
city: Eldoret
country: Kenya
facility: International Cancer Institute
city: Nairobi
country: Kenya
facility: Strathmore University CREATES
city: Calabar
country: Nigeria
facility: University of Calabar Teaching Hospital
state: Cross River State
city: Abuja
country: Nigeria
facility: National Hospital Abuja
state: FCT
city: Zaria
country: Nigeria
facility: Ahmadu Bello University Teaching Hospital
state: Kaduna
city: Kano
country: Nigeria
facility: Aminu Kano Teaching Hospital
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: