Clinical trial NCT02433158: Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
Official title: An Open-Label Extension Study to Evaluate the Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Sickle Cell Anemia
Sickle Cell Disease
Sickle Cell Disorders
Pain Crisis
Vaso-occlusive Crisis
Keywords: Rivipansel
GMI-1070
Selectin Inhibitor
SCD
VOC
Lead sponsor: GlycoMimetics Incorporated
Sponsor class: INDUSTRY
First posted: 2015-05-04
Enrollment: 154
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).

Detailed description: This is an open label extension study in subjects who are 6 years of age or older with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002). This study is designed to evaluate the safety and describe the efficacy of rivipansel as treatment for one or more vaso-occlusive crisis (VOC) events in hospitalized subjects with SCD.

Eligibility criteria: Inclusion Criteria:

* Completion of Study B5201002.
* Documented diagnosis of SCD.
* At least 6 years of age.
* Male and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception throughout the study.
* Diagnosis of VOC necessitating IV opioids and admission to the hospital.
* Able to receive the first dose of rivipansel within 24 hours from administration of the first dose of IV opioids for this hospitalization.

Exclusion Criteria:

* Non-compliance with study procedures in the double blind study (B5201002).
* Occurrence of any severe and/or generalized cutaneous manifestation or any other adverse event during participation in Study B5201002 that was related to study drug and which would therefore make it inappropriate for the subject to receive rivipansel in the current study.
* Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results.
* Clinically significant deterioration in renal function in Study B5201002.
* Pregnant female subjects, breastfeeding female subjects and male and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception.
* Active use of illicit drugs and/or alcohol dependence.

Primary outcomes:
measure: Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study.
timeFrame: 18 months
measure: Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study.
timeFrame: 18 months
measure: Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study
timeFrame: 18 months
measure: Number (%) of subjects with serious adverse events (SAEs) over the study.
timeFrame: 18 months
Secondary outcomes:
measure: Subject re hospitalization
timeFrame: 18 months
Other outcomes:
Locations:
city: Mobile
country: United States
facility: University of South Alabama Women's and Children's Hospital
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital Research Pharmacy
state: Arkansas
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Sacramento
country: United States
facility: UC Davis Medical Center Main Hospital
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Washington
country: United States
facility: Howard University Center for Sickle Cell disease
state: District of Columbia
city: Washington
country: United States
facility: MedStar Health Research Institute
state: District of Columbia
city: Washington
country: United States
facility: Howard University Hospital
state: District of Columbia
city: Fort Myers
country: United States
facility: Golisano Childrens Hospital of Southwest Florida
state: Florida
city: Miami
country: United States
facility: Jackson Memorial Hospital
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: West Palm Beach
country: United States
facility: St. Mary's Medical Center
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:
state: Georgia
city: Atlanta
country: United States
facility: Grady Health System
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:
state: Georgia
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center/
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta: Scottish Rite Campus
state: Georgia
city: Savannah
country: United States
facility: Memorial Family Medicine Center
state: Georgia
city: Savannah
country: United States
facility: Memorial Health University Medical Center
state: Georgia
city: Chicago
country: United States
facility: The University of Chicago/Comer Children's Hospital
state: Illinois
city: Chicago
country: United States
facility: University of Chicago, Investigational Drug Service Pharmacy
state: Illinois
city: Baltimore
country: United States
facility: University of Maryland Medical System Investigational Pharmacy
state: Maryland
city: Baltimore
country: United States
facility: University of Maryland Medical System
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins Department of Medicine Clinical Trials Unit
state: Maryland
city: Baltimore
country: United States
facility: The Johns Hopkins University School of Medicine
state: Maryland
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital Department of Pharmacy Services
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins Medicine
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Center for Clinical Investigation, Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Investigational Drug Services
state: Massachusetts
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Jackson
country: United States
facility: University of Mississippi Medical Center - Outpatient Clinical Research Unit
state: Mississippi
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Saint Louis
country: United States
facility: Center for Outpatient Health
state: Missouri
city: Saint Louis
country: United States
facility: Barnes-Jewish Hospital Department of Pharmacy
state: Missouri
city: Saint Louis
country: United States
facility: Barnes-Jewish Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Center for Advanced Medicine
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New Brunswick
country: United States
facility: Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers-Robert Wood Johnson Medical School
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute of New Jersey
state: New Jersey
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Brooklyn
country: United States
facility: Kings County Hospital Center
state: New York
city: Brooklyn
country: United States
facility: State University of New York (SUNY) Downstate Medical Center
state: New York
city: Brooklyn
country: United States
facility: SUNY Downstate Medical Center University Hospital of Brooklyn
state: New York
city: New York
country: United States
facility: Columbia University Medical Center Research Pharmacy
state: New York
city: New York
country: United States
facility: MS CHONY Pediatric Emergency Department
state: New York
city: New York
country: United States
facility: MS CHONY Pediatric Hematology/Oncology Unit
state: New York
city: Durham
country: United States
facility: Duke University Hospital, Investigational Drug Service
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University Brody School of Medicine
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University, Brody School of Medicine
state: North Carolina
city: Greenville
country: United States
facility: Leo W. Jenkins Cancer Center
state: North Carolina
city: Greenville
country: United States
facility: Vidant Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati - Hoxworth Building
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati Medical Center / Investigational Pharmacy
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati Medical Center / Research Office
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati Medical Center
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati Physicians Company LLC
state: Ohio
city: Cincinnati
country: United States
facility: UC Health Ridgeway Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Investigational Drug Services
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Center East
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: UPMC Presbyterian
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Hillman Cancer Center
state: Pennsylvania
city: Providence
country: United States
facility: Hasbro Children's Hospital
state: Rhode Island
city: Providence
country: United States
facility: Rhode Island Hospital-Pharmacy Service
state: Rhode Island
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Providence
country: United States
facility: The Miriam Hospital
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina-Hospital
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Charleston
country: United States
facility: MUSC Investigational Drug Services
state: South Carolina
city: Fort Worth
country: United States
facility: Cook Children's Hematology and Oncology Center
state: Texas
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Grapevine
country: United States
facility: Cook Children's Hematology and Oncology Center-Grapevine
state: Texas
city: Houston
country: United States
facility: University of Texas Medical School
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital Laboratory
state: Utah
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Richmond
country: United States
facility: Main Hospital-VCU
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University - Investigational Drug Services
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University- Clinical Research Services Unit
state: Virginia
city: Edmonton
country: Canada
facility: Royal Alexandra Hospital
state: Alberta
city: Edmonton
country: Canada
facility: Miseracordia Community Hospital
state: Alberta
city: Edmonton
country: Canada
facility: Kaye Edmonton Clinic 3C
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta Hospital, Pharmacy Services
state: Alberta
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta Hospital
state: Alberta
city: Edmonton
country: Canada
facility: Research transition Facility
state: Alberta
city: Edmonton
country: Canada
facility: Grey Nuns Community Hospital
state: Alberta
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Montreal
country: Canada
facility: Centre Hospitalier Universitaire Sainte-Justine
state: Quebec
city: Montreal
country: Canada
facility: The Montreal Children's Hospital / McGill University Health Centre
state: Quebec
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Dampier CD, Telen MJ, Wun T, Brown RC, Desai P, El Rassi F, Fuh B, Kanter J, Pastore Y, Rothman J, Taylor JG, Readett D, Sivamurthy KM, Tammara B, Tseng LJ, Lozier JN, Thackray H, Magnani JL, Hassell KL; RESET Investigators. A randomized clinical trial of the efficacy and safety of rivipansel for sickle cell vaso-occlusive crisis. Blood. 2023 Jan 12;141(2):168-179. doi: 10.1182/blood.2022015797.