Sickle Cell Disease Clinical Trial
Official title:
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
Verified date | March 2024 |
Source | Pfizer |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 240 |
Est. completion date | August 15, 2024 |
Est. primary completion date | August 15, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years and older |
Eligibility | Inclusion Criteria: 1. 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. 2. Participant is male or female, = 12 years of age at the time of informed consent. 3. 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. 4. 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. 5. 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. Exclusion Criteria: 1. Participant is receiving regularly scheduled red blood cell (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion). 2. Participant is taking or has received crizanlizumab (ADAKVEO®) within 90 days prior to the Screening Visit 3. Participant weighs > 133 kg (292 lbs.). Other protocol-defined Inclusion/Exclusion may apply. |
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 das Clinicas da Universidade Federal de Minas Gerais | Belo Horizonte | MG |
Brazil | Hospital de Clínicas de Porto Alegre | Porto Alegre | RS |
Brazil | Multihemo Serviços Médicos S/A | Recife | Pernambuco |
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 | Comite de Etica em Pesquisa - CEP do Hospital Alemao Oswaldo Cruz/ SP | Sao Paulo | SP |
Brazil | Casa de Saúde Santa Marcelina | São Paulo | |
Brazil | CEPEC-Centro de Pesquisa Clinica | São Paulo | |
Brazil | Hospital das Clinicas 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 | Organizacion Clinica Bonnadona Prevenir S.A.S. | Barranquilla | Atlántico |
Colombia | Sociedad de Oncologia y hematologia del Cesar S.A.S. | Valledupar | Cesar |
Egypt | AinShams University Hospital | Cairo | |
Egypt | Faculty of Medicine Cairo University | Cairo | |
Egypt | Cairo University Paediatric Hospital - abou el Reesh University Hospital | El-Rashidy Street, ElSayeda Zeinb | Cairo |
Egypt | Al Kasr Al Ainy Cairo University Hospital | Elmanial | Cairo Governorate |
Egypt | Zagazig University Hospital | Zagazig | ASH Sharqia |
France | Hôpital Avicenne | Bobigny | |
France | Hôpital Henri Mondor | Créteil | |
France | Institut Universitaire du Cancer de Toulouse-Oncopole | Toulouse Cedex 9 | |
Germany | Universitätsklinikum Regensburg Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation | Regensburg | |
Ghana | Sickle-Cell Office, Directorate of Child Health, Komfo Anokye Teaching Hospital | Kumasi | Ashanti |
Italy | Farmacia Interna Azienda Ospedaliero-Ente Ospedaliero Ospedali Galliera | Genova | |
Italy | S.S.D. Microcitemia, anemie congenite e dismetabolismo del ferro Azienda | Genova | |
Italy | DAI Materno-Infantile, UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria | Napoli | |
Italy | DAI Materno-Infantile,- UOC Clinica Pediatrica 1 Azienda Ospedaliera Universitaria "Luigi Vanvitel | Napoli | |
Italy | UO di Farmacia Clinica, Dipartimento di Medicina Sperimentale Azienda Ospedaliera Universitaria | 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, KEMRI Clinical Research Annex | Kisumu | Siaya |
Kenya | Gertrude's Children Hospital | Nairobi | |
Kenya | Kenya Medical Research Institute- Center for Respiratory Disease Research | Nairobi | |
Kenya | Strathmore University Medical Center - Center for Research in Therapeutic Sciences(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 Hospital | Adana | Yuregir |
Turkey | Hacettepe University | Ankara | Altindag/sihhiye |
Turkey | Mersin Universitesi Tip Fakultesi Saglik Arastirma ve Uygulama Merkezi Hastanesi | Yenisehir | Mersin |
United Kingdom | Guy's and St Thomas' NHS Foundation Trust | London | London CITY OF |
United Kingdom | King's College Hospital NHS Foundation Trust | London | London CITY OF |
United States | University of Michigan Hospitals - Michigan Medicine | Ann Arbor | Michigan |
United States | Children's Healthcare of Atlanta | Atlanta | Georgia |
United States | Children's Healthcare of Atlanta - Egleston | Atlanta | Georgia |
United States | Children's Healthcare of Atlanta - Scottish Rite | Atlanta | Georgia |
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 County Medical Center | Buffalo | New York |
United States | Hospital Pharmacy Services- Investigational Drug Services | Chicago | Illinois |
United States | Rush University Medical Center | Chicago | Illinois |
United States | Rush University Medical Center Investigator Pharmacy | Chicago | Illinois |
United States | University of Illinois Clinical Research Center (CRC) | Chicago | Illinois |
United States | University of Illinois Hospital and Health Sciences System(UI Health) | Chicago | Illinois |
United States | Duke University Medical Center | Durham | North Carolina |
United States | DUMC Investigational Drug Services Pharmacy | Durham | North Carolina |
United States | Uconn Health/Uconn John Dempsey Hospital/Neag Comprehensive Cancer Center/New England Sickle Cell | Farmington | Connecticut |
United States | McGovern Medical School/Health Science Center Houston | Houston | Texas |
United States | Memorial Hermann - TMC Investigational Drugs, IDS Pharmacy | Houston | Texas |
United States | Memorial Hermann Hospital, Texas Medical Center - Clinical Research Unit (CRU) | Houston | Texas |
United States | UT Physicians Comprehensive Sickle Cell Clinic | Houston | Texas |
United States | Arkansas Children's Hospital | Little Rock | Arkansas |
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
United States | University of South Alabama Children's and Women's Hospital | Mobile | Alabama |
United States | University of South Alabama Mitchell Cancer Institute | Mobile | Alabama |
United States | University of South Alabama Strada Patient Care Center | Mobile | Alabama |
United States | UCSF Benioff Children's Hospital, Oakland | Oakland | California |
United States | UC Irvine Health | Orange | California |
United States | Phoenix Children's Hospital | Phoenix | Arizona |
United States | John S. Curran, MD.,Children's Health Center | Tampa | Florida |
United States | University of South Florida | Tampa | Florida |
United States | USF Clinical Investigational Research Pharmacy | Tampa | Florida |
United States | USF Health Carol & Frank Morsani Center for Advanced Healthcare | Tampa | Florida |
United States | USF Health South Tampa Center for Advanced Healthcare | Tampa | Florida |
Zambia | Matero Clinical Research Site, | Lusaka | |
Zambia | University Teaching Hospital- Children's Hospital | Lusaka | |
Zambia | Arthur Davison Childrens's Hospital | Ndola | Copperbelt |
Lead Sponsor | Collaborator |
---|---|
Pfizer |
United States, Zambia, Brazil, Colombia, Egypt, France, Germany, Ghana, Italy, Kenya, Lebanon, Nigeria, Oman, Saudi Arabia, Tanzania, Turkey, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | PD parameter (P-selectin inhibition) | To characterize the pharmacodynamics (PD) (P-selectin inhibition) of inclacumab at 30 mg/kg | Day 1- Week 48 | |
Other | PD parameter (Platelet Leukocyte Aggregation) | To characterize the pharmacodynamics (PD) (PLA) of inclacumab at 30 mg/kg | Day 1- Week 48 | |
Primary | Rate of VOCs during the 48-week treatment period. | A VOC is defined as an acute episode of pain that:
has no medically determined cause other than a vaso-occlusive event, and results in a visit to a medical facility (hospitalization, 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 non-steroidal anti-inflammatoroy drugs (NSAIDS), or an increase in treatment with oral narcotics. Complicated VOCs of acute chest syndrome (ACS), hepatic sequestration, splenic sequestration, and priapism that meet the requirements listed above will be included in the primary endpoint |
Day 1- Week 48 | |
Secondary | Time to first VOC during the 48-week treatment period. | Day 1- Week 48 | ||
Secondary | Time to second VOC during the 48-week treatment period Efficacy. | Day 1- Week 48 | ||
Secondary | Proportion of participants with no VOCs during the 48-week treatment period. | Day 1- Week 48 | ||
Secondary | Rate of VOCs that required admission to a healthcare facility and treatment with parenteral pain medication during the 48-week treatment period. | Admission includes: (a) A hospital admission, or (b) An admission to an emergency room, observation unit, or infusion center for = 12 hours, or (c) 2 visits to an emergency room, observation unit, or infusion center over a 72-hour period. | Day 1- Week 48 | |
Secondary | Number of days of inpatient hospitalization for a VOC during the 48-week treatment period. | Day 1- Week 48 | ||
Secondary | Incidence of treatment-emergent adverse events (TEAEs). | Day 1- Week 48 |
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