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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05348915
• Other study ID #: GBT2104-133
• Secondary ID: C53610032020-005
• Status: Recruiting
• Phase: Phase 3
• Start date: March 29, 2022
• Est. completion date: November 15, 2028

Study information

• Verified date: March 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 520
• Est. completion date: November 15, 2028
• Est. primary completion date: November 15, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease
• Vaso-occlusive Crisis
• Vaso-occlusive Pain Episode in Sickle Cell Disease

Intervention

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.

Locations

Country	Name	City	State
Brazil	Hemocentro de Belo Horizonte - Fundacao Hemominas	Belo Horizonte	MG
Brazil	Hospital das Clinicas da Universidade Federal de Minas Gerais	Belo Horizonte	Minas Gerais
Brazil	Hospital de Clínicas de Porto Alegre	Porto Alegre	RIO Grande DO SUL
Brazil	Multihemo Servicos Medicos S/A	Recife	PE
Brazil	Hospital Das Clinicas da Faculdade de Medicina de Ribeirão Preto - USP	Ribeirão Preto	SÃO Paulo
Brazil	Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO	Rio de Janeiro
Brazil	Instituto D'Or de Pesquisa e Ensino - Hospital São Rafael	Salvador	Bahia
Brazil	Fundação Faculdade Regional de Medicina de São José do Rio Preto	Sao Jose do Rio Preto	SAO Paulo
Brazil	Casa de Saude Santa Marcelina	São Paulo
Brazil	CEPEC-Centro de Pesquisa Clinica	São Paulo
Brazil	Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo -HCFMUSP	São Paulo
Brazil	Hospital Samaritano Higienópolis/Esho Empresa De Servicos Hospitalares S.A	São Paulo
Colombia	Clinica de la Costa Ltda.	Barranquilla	Atlantico
Colombia	Sociedad de Oncología y hematología del Cesar	Valledupar	Cesar
Germany	Universitätsklinikum Regensburg Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation	Regensburg
Italy	Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera	Genova
Italy	S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro	Genova
Italy	DAI Materno-Infantile,- UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitel	Napoli
Italy	UO di Farmacia Clinica,Dipartimento di Medicina Sperimentale	Napoli
Italy	UO di Farmacia Clinica,Dipartimento di Medicina Sperimentale	Napoli
Italy	UOC Patologia Clinica e Molecolare Azienda Ospedaliera Universitaria "Luigi Vanvitelli"	Napoli
Italy	UOC Radiologia Azienda Ospedaliera Universitaria "Luigi Vanvitelli"	Napoli
Italy	Dipartimento Strutturale Aziendale Salute della Donna e del Bambino Clinica Ginecologica	Padova
Italy	Farmacia Azienda Ospedale Universita Padova	Padova
Italy	U.O.C. Farmacia Istituto Oncologico Veneto	Padova
Kenya	International Cancer Institute	Eldoret
Kenya	KEMRI/CRDR Siaya Clinical Research Annex	Kisumu	Siaya
Kenya	Kenya Medical Research Institute - Centre for Respiratory Disease Research	Nairobi
Kenya	Strathmore University CREATES	Nairobi
Lebanon	American University of Beirut Medical Center	Hamra	Beirut
Lebanon	Nini Hospital	Tripoli	North Lebanon
Nigeria	National Hospital Abuja	Abuja	FCT
Nigeria	University of Calabar Teaching Hospital	Calabar	Cross River State
Nigeria	University of Nigeria Teaching Hospital	Enugu
Nigeria	University of Abuja Teaching Hospital	Gwagwalada	FCT
Nigeria	Barau Dikko Teaching Hospital/Kaduna State University	Kaduna
Nigeria	Aminu Kano Teaching Hospital	Kano
Nigeria	Department of Pediatrics, College of Medicine, Lagos University Teaching Hospital	Lagos
Nigeria	Ahmadu Bello University Teaching Hospital	Zaria	Kaduna
Oman	Sultan Qaboos University Hospital	Muscat
Saudi Arabia	Prince Mohammed bin Nasser Hospital	Jizan	Southern
Tanzania	NIMR-Mbeya Medical Research Center	Mbeya
Turkey	Acibadem Adana Hastanesi Cocuk Hematoloji Onkoloji	Adana
Turkey	Baskent University Adana Appl. and Research Central, Yuregir Baskent Hospital Hematology	Adana
Turkey	Hacettepe University Ihsan Dogramaci Children Hospital	Ankara
Turkey	Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi	Mersin
United States	University of Michigan Hospitals - Michigan Medicine	Ann Arbor	Michigan
United States	Brigham And Woman's Hospital	Boston	Massachusetts
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	Dana-Farber Cancer Institute IDS Pharmacy	Boston	Massachusetts
United States	Jacobi Medical Center	Bronx	New York
United States	Erie Country Medical Center	Buffalo	New York
United States	University of Illinois at Chicago	Chicago	Illinois
United States	University of Illinois Hospital and Health Sciences System (UI Health)	Chicago	Illinois
United States	University of Illinois Hospital and Health Systems	Chicago	Illinois
United States	Duke University Medical Center	Durham	North Carolina
United States	DUMC Investigational Drug Services Pharmacy	Durham	North Carolina
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Strada Patient Care Center, Pediatric Hematology	Mobile	Alabama
United States	University of South Alabama Children's and Women's Hospital	Mobile	Alabama
United States	Children's Hospital and Research Center at Oakland	Oakland	California
United States	UC Irvine Medical Center	Orange	California
United States	UC Irvine Medical Center	Orange	California
United States	John S Curran,MD, Children's Health Center/Children's Medical Center (CMS)	Tampa	Florida
United States	St. Joseph's Hospital	Tampa	Florida
United States	University of South Florida, Department of Pediatrics	Tampa	Florida
United States	USF Health Carol and Frank Morsani Center for Advanced Healthcare	Tampa	Florida
United States	USF Health South Tampa Center for Advanced Healthcare (STC)	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Brazil,  Colombia,  Germany,  Italy,  Kenya,  Lebanon,  Nigeria,  Oman,  Saudi Arabia,  Tanzania,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Other	Incidence of anti-drug antibodies (ADA) to inclacumab.		Day 1 through Week 48
Other	PD parameter (P-selectin inhibition)		Day 1 through Week 48
Other	PD parameter (Platelet Leukocyte Aggregation)		Day 1 through Week 48
Primary	Incidence of treatment-emergent adverse events (TEAEs).		Day 1 through study completion, an estimate of 5 years
Secondary	Annualized rate of VOCs		Day 1 through study completion, an estimate of 5 years
Secondary	Annualized rate of VOCs that require admission to a healthcare facility and treatment.		Day 1 through study completion, an estimate of 5 years
Secondary	Annualized number of days of inpatient hospitalization for a VOC.		Day 1 through study completion, an estimate of 5 years
Secondary	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
Secondary	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
Secondary	Annualized rate of complicated VOCs.		Day 1 through study completion, an estimate of 5 years
Secondary	Annualized rate of RBC transfusions.		Day 1 through study completion, an estimate of 5 years

External Links

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