Clinical trial NCT02187003: Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
Official title: A Phase 3, Multicenter ,Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Anemia, Sickle Cell
Keywords: Sickle Cell Anemia
Sickle Cell Disease
Sickle Cell Disorders
pain crisis
vaso-occlusive crisis
rivipansel
GMI-1070
selectin inhibitor
SCD
VOC
Lead sponsor: Biossil Inc.
Sponsor class: INDUSTRY
Collaborators: name: GlycoMimetics Incorporated
First posted: 2014-07-10
Enrollment: 345
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.


Eligibility criteria: Inclusion Criteria:

* At least 6 years of age.
* Documented diagnosis of sickle cell disease.
* Diagnosis of vaso-occlusive crisis necessitating admission to the hospital with treatment including IV opioids.
* Able to receive the first dose of study drug within 24 hours from the administration of IV opioids.

Exclusion Criteria:

* Serious systemic infection
* Acute Chest Syndrome
* Serious concomitant medical problems (for example, stroke)
* SCD pain atypical of VOC
* Severe renal or hepatic impairment
* Chronic pain rather than a presentation of acute VOC

Primary outcomes:
measure: Time to Readiness for Discharge From Hospital
timeFrame: Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8)
Secondary outcomes:
measure: Time to Discharge From Hospital
timeFrame: Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8)
measure: Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital
timeFrame: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)
measure: Time to Discontinuation of Intravenous (IV) Opioids
timeFrame: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)
measure: Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug
timeFrame: 24 hours post first IV infusion of loading dose of study drug on Day 1
measure: Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital
timeFrame: Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8
Other outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) as Per Genotype
timeFrame: Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
measure: Number of Participants With Treatment Emergent Adverse Event (AEs) as Per Severity
timeFrame: Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
measure: Number of Participants With Clinical Laboratory Abnormalities
timeFrame: Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
measure: Number of Participants With Increase in Grades From Baseline in Clinical Laboratory Parameters Over the Study
timeFrame: Baseline up to the 35-day post discharge visit (up to an average of Day 43)
measure: Number of Participants With Clinically Significant Changes in Physical Examination
timeFrame: From Post-screening up to end of treatment (up to an average of Day 8), From Post-discharge up to 35 days post-discharge (up to an average of Day 43)
measure: Number of Participants With Treatment Related Changes From Baseline in Vital Signs Over the Study
timeFrame: Baseline (Day 1) up to the 35-day post discharge visit (up to an average of Day 43)
measure: Percentage of Participants With Adjudicated Acute Chest Syndrome (ACS)
timeFrame: Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
measure: Percentage of Participants With Severe Adjudicated and/or Generalized Cutaneous Manifestations
timeFrame: Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
measure: Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 7, 14 and 30 Days of Discharge From Hospital
timeFrame: Within 7 days of discharge; Within 14 days of discharge; Within 30 days of discharge, where discharge from hospital was any day from Day 1 to an average of Day 8
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 Research Pharmacy
state: Arkansas
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center Main Hospital
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado CTRC
state: Colorado
city: Aurora
country: United States
facility: University of Colorado Hospital
state: Colorado
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 Children's Hospital of Southwest Florida
state: Florida
city: Gainesville
country: United States
facility: UF Health Davis Cancer Pavillion and Shands Med Plaza
state: Florida
city: Gainesville
country: United States
facility: UF Health Shands Cancer Hospital
state: Florida
city: Gainesville
country: United States
facility: UF Health Shands Hospital
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: Tampa
country: United States
facility: Investigational Drug Service Tampa General Hospital
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital Center of Research Excellence
state: Florida
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Tampa
country: United States
facility: St. Joseph's Children's Hospital
state: Florida
city: West Palm Beach
country: United States
facility: Children's Hematology and Oncology Associates
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: Augusta
country: United States
facility: Augusta University Clinical Research Pharmacy
state: Georgia
city: Augusta
country: United States
facility: Augusta University Medical Center
state: Georgia
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Savannah
country: United States
facility: Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct
state: Georgia
city: Savannah
country: United States
facility: Memorial Health University Medical Center
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago Clinical Research Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois Hospital and Health Sciences System
state: Illinois
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: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Louisville
country: United States
facility: Kosair Charities Pediatric Clinical Research Unit
state: Kentucky
city: Louisville
country: United States
facility: Norton Children´s Hospital
state: Kentucky
city: Louisville
country: United States
facility: The Novak Center for Children's Health
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Health Sciences Center
state: Kentucky
city: Louisville
country: United States
facility: University of Louisville Physicians Pediatric Hematology Oncology
state: Kentucky
city: Baton Rouge
country: United States
facility: Our Lady of the Lake Regional Medical Center
state: Louisiana
city: Baton Rouge
country: United States
facility: St. Jude Affiliate Clinic
state: Louisiana
city: Baton Rouge
country: United States
facility: Our Lady of the Lake Physician Group-Medical Oncology
state: Louisiana
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 Medicine
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
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 Womens Hospital
state: Massachusetts
city: Boston
country: United States
facility: Center for Clinical Investigation, Brigham & Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Investigational Drug Services
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center E7E
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Boston University Medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Children's Hospital of Michigan
state: Michigan
city: Detroit
country: United States
facility: Detroit Medical Center Pharmacy
state: Michigan
city: Detroit
country: United States
facility: Wayne State University / Detroit Receiving Hospital
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: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
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 - Pharmacy Department
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: Icahn School of Medicine at Mount Sinai
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: Staten Island
country: United States
facility: Staten Island University Hospital
state: New York
city: Durham
country: United States
facility: Duke University Hospital
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: 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 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: University of Cincinnati- Hoxworth Building
state: Ohio
city: Cincinnati
country: United States
facility: UC Health Ridgeway Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center East
state: Ohio
city: Columbus
country: United States
facility: Nationwide Childrens 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 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: Dayton
country: United States
facility: Five Rivers Medical Surgical Health Center
state: Ohio
city: Dayton
country: United States
facility: Miami Valley Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Einstein Medical Center Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Presbyterian
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
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 - Hospital
state: South Carolina
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
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: Texas Children's Hospital - Clinical Research Center
state: Texas
city: Houston
country: United States
facility: Texas Children´s Hospital
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
state: Utah
city: Richmond
country: United States
facility: Main Hospital - Virginia Commonwealth University
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
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 3 C
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: Vancouver
country: Canada
facility: St. Paul's Hospital
state: British Columbia
city: Vancouver
country: Canada
facility: St. Paul's Hospital
state: British Columbia
city: Ottawa
country: Canada
facility: Children's Hospital Of Eastern Ontario
state: Ontario
city: Toronto
country: Canada
facility: University Health Network, Toronto General Hospital
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: McGill University Health Centre, Royal Victoria Hospital
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.
citation: Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1.