Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
- Registration Number
- NCT02187003
- Lead Sponsor
- Biossil Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 345
- 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.
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Rivipansel Treatment Arm Rivipansel - Placebo Treatment Arm Placebo -
- Primary Outcome Measures
Name Time Method Time to Readiness for Discharge From Hospital Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8) Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC.
- Secondary Outcome Measures
Name Time Method Time to Discharge From Hospital 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) Time to discharge from hospital was defined as the difference (in hours) between the time and date of the hospital discharge order from a qualified healthcare provider and the start time and date of the first infusion (loading dose) of study drug.
Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) Cumulative IV opioid consumption was reported as cumulative IV opioid use (standardized using morphine equivalent units \[MEU\]), from the start of the first infusion (loading dose) of study drug until hospital discharge.
Time to Discontinuation of Intravenous (IV) Opioids Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8) Time to discontinuation of IV opioids was defined as the difference (in hours) between the stop time and date of the latest IV opioid dose and the start time and date of the first infusion (loading dose) of study drug.
Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug 24 hours post first IV infusion of loading dose of study drug on Day 1 Cumulative IV opioid consumption (standardized using MEU) was reported as IV opioid use in the first 24 hours from the start time of the first IV infusion (loading dose) of study drug.
Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8 Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported.
Trial Locations
- Locations (138)
Golisano Children's Hospital of Southwest Florida
🇺🇸Fort Myers, Florida, United States
The Johns Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
🇺🇸Atlanta, Georgia, United States
University of Illinois Hospital and Health Sciences System
🇺🇸Chicago, Illinois, United States
Nationwide Childrens Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
UPMC Hillman Cancer Center
🇺🇸Pittsburgh, Pennsylvania, United States
Center for Clinical Investigation, Brigham & Women's Hospital
🇺🇸Boston, Massachusetts, United States
The Miriam Hospital
🇺🇸Providence, Rhode Island, United States
Augusta University
🇺🇸Augusta, Georgia, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
Arkansas Children's Hospital Research Pharmacy
🇺🇸Little Rock, Arkansas, United States
Howard University Hospital
🇺🇸Washington, District of Columbia, United States
Jackson Memorial Hospital
🇺🇸Miami, Florida, United States
University Of Miami
🇺🇸Miami, Florida, United States
Grady Health System
🇺🇸Atlanta, Georgia, United States
Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct
🇺🇸Savannah, Georgia, United States
University of Maryland Medical System Investigational Pharmacy
🇺🇸Baltimore, Maryland, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
University of Maryland Medical System
🇺🇸Baltimore, Maryland, United States
Center for Advanced Medicine
🇺🇸Saint Louis, Missouri, United States
Center for Outpatient Health
🇺🇸Saint Louis, Missouri, United States
Barnes-Jewish Hospital
🇺🇸Saint Louis, Missouri, United States
Barnes-Jewish Hospital Department of Pharmacy
🇺🇸Saint Louis, Missouri, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Kings County Hospital Center
🇺🇸Brooklyn, New York, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
State University of New York (SUNY) - Downstate Medical Center
🇺🇸Brooklyn, New York, United States
East Carolina University Brody School of Medicine
🇺🇸Greenville, North Carolina, United States
Five Rivers Medical Surgical Health Center
🇺🇸Dayton, Ohio, United States
Columbia University Medical Center Research Pharmacy
🇺🇸New York, New York, United States
Staten Island University Hospital
🇺🇸Staten Island, New York, United States
The Ohio State University Wexner Medical Center East
🇺🇸Columbus, Ohio, United States
University of Cincinnati- Hoxworth Building
🇺🇸Cincinnati, Ohio, United States
MS CHONY Pediatric Emergency Department
🇺🇸New York, New York, United States
Miami Valley Hospital
🇺🇸Dayton, Ohio, United States
The Ohio State University Investigational Drug Services
🇺🇸Columbus, Ohio, United States
UPMC Presbyterian
🇺🇸Pittsburgh, Pennsylvania, United States
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States
Cook Children's Hematology and Oncology Center-Grapevine
🇺🇸Grapevine, Texas, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
St. Paul's Hospital
🇨🇦Vancouver, British Columbia, Canada
McGill University Health Centre, Royal Victoria Hospital
🇨🇦Montreal, Quebec, Canada
Centre Hospitalier Universitaire Sainte-Justine
🇨🇦Montreal, Quebec, Canada
Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Detroit Medical Center Pharmacy
🇺🇸Detroit, Michigan, United States
Duke University Hospital
🇺🇸Durham, North Carolina, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
University of Cincinnati Medical Center/ Investigational Pharmacy
🇺🇸Cincinnati, Ohio, United States
University of Cincinnati Medical Center
🇺🇸Cincinnati, Ohio, United States
University of Cincinnati Physicians Company LLC
🇺🇸Cincinnati, Ohio, United States
UC Health Ridgeway Hospital
🇺🇸Cincinnati, Ohio, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Investigational Drug Services
🇺🇸Boston, Massachusetts, United States
University of South Alabama Children's and Women's Hospital
🇺🇸Mobile, Alabama, United States
St. Mary's Medical Center
🇺🇸West Palm Beach, Florida, United States
Augusta University Medical Center
🇺🇸Augusta, Georgia, United States
University of Chicago, Investigational Drug Service Pharmacy
🇺🇸Chicago, Illinois, United States
The University of Chicago/Comer Children's Hospital
🇺🇸Chicago, Illinois, United States
University of Illinois at Chicago Clinical Research Center
🇺🇸Chicago, Illinois, United States
Our Lady of the Lake Physician Group-Medical Oncology
🇺🇸Baton Rouge, Louisiana, United States
Johns Hopkins Medicine
🇺🇸Baltimore, Maryland, United States
The Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Brigham and Womens Hospital
🇺🇸Boston, Massachusetts, United States
Boston Medical Center E7E
🇺🇸Boston, Massachusetts, United States
Boston University Medical Center
🇺🇸Boston, Massachusetts, United States
Wayne State University / Detroit Receiving Hospital
🇺🇸Detroit, Michigan, United States
University of Mississippi Medical Center-Outpatient Clinical Research Unit
🇺🇸Jackson, Mississippi, United States
Rutgers Cancer Institute Of New Jersey
🇺🇸New Brunswick, New Jersey, United States
Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital
🇺🇸New Brunswick, New Jersey, United States
Kings County Hospital Center - Pharmacy Department
🇺🇸Brooklyn, New York, United States
SUNY Downstate Medical Center University Hospital of Brooklyn
🇺🇸Brooklyn, New York, United States
MS CHONY Pediatric Hematology/Oncology Unit
🇺🇸New York, New York, United States
Leo W. Jenkins Cancer Center
🇺🇸Greenville, North Carolina, United States
Vidant Medical Center
🇺🇸Greenville, North Carolina, United States
University of Cincinnati Medical Center/Research Office
🇺🇸Cincinnati, Ohio, United States
Einstein Medical Center Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
🇺🇸Columbus, Ohio, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
University of Texas Medical School
🇺🇸Houston, Texas, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
Children's Hospital Of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
University Health Network, Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
The Montreal Children's Hospital/ McGill University Health Centre
🇨🇦Montreal, Quebec, Canada
Texas Children´s Hospital
🇺🇸Houston, Texas, United States
Our Lady of the Lake Regional Medical Center
🇺🇸Baton Rouge, Louisiana, United States
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
Howard University Center for Sickle Cell Disease
🇺🇸Washington, District of Columbia, United States
Medstar Health Research Institute
🇺🇸Washington, District of Columbia, United States
Augusta University Clinical Research Pharmacy
🇺🇸Augusta, Georgia, United States
Children's Hematology and Oncology Associates
🇺🇸West Palm Beach, Florida, United States
Children's Healthcare of Atlanta: Scottish Rite Campus
🇺🇸Atlanta, Georgia, United States
Memorial Health University Medical Center
🇺🇸Savannah, Georgia, United States
St. Jude Affiliate Clinic
🇺🇸Baton Rouge, Louisiana, United States
Rhode Island Hospital-Pharmacy Service
🇺🇸Providence, Rhode Island, United States
Rutgers-Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
Cook Children's Hematology and Oncology Center
🇺🇸Fort Worth, Texas, United States
Texas Children's Hospital - Clinical Research Center
🇺🇸Houston, Texas, United States
University Of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
UC Davis Medical Center Main Hospital
🇺🇸Sacramento, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
University of Colorado CTRC
🇺🇸Aurora, Colorado, United States
University of Colorado Hospital
🇺🇸Aurora, Colorado, United States
UF Health Shands Cancer Hospital
🇺🇸Gainesville, Florida, United States
UF Health Shands Hospital
🇺🇸Gainesville, Florida, United States
Investigational Drug Service Tampa General Hospital
🇺🇸Tampa, Florida, United States
UF Health Davis Cancer Pavillion and Shands Med Plaza
🇺🇸Gainesville, Florida, United States
Tampa General Hospital Center of Research Excellence
🇺🇸Tampa, Florida, United States
Tampa General Hospital
🇺🇸Tampa, Florida, United States
University of South Florida
🇺🇸Tampa, Florida, United States
St. Joseph's Children's Hospital
🇺🇸Tampa, Florida, United States
Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States
Norton Children´s Hospital
🇺🇸Louisville, Kentucky, United States
The Novak Center for Children's Health
🇺🇸Louisville, Kentucky, United States
University of Louisville Health Sciences Center
🇺🇸Louisville, Kentucky, United States
University of Louisville Physicians Pediatric Hematology Oncology
🇺🇸Louisville, Kentucky, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Jacobi Medical Center
🇺🇸Bronx, New York, United States
Medical University of South Carolina - Hospital
🇺🇸Charleston, South Carolina, United States
Virginia Commonwealth University-Investigational Drug Services
🇺🇸Richmond, Virginia, United States
Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center
🇺🇸Charleston, South Carolina, United States
Kaye Edmonton Clinic 3 C
🇨🇦Edmonton, Alberta, Canada
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
MUSC Investigational Drug Services
🇺🇸Charleston, South Carolina, United States
Royal Alexandra Hospital
🇨🇦Edmonton, Alberta, Canada
University of Alberta Hospital, Pharmacy Services
🇨🇦Edmonton, Alberta, Canada
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Main Hospital - Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
Miseracordia Community Hospital
🇨🇦Edmonton, Alberta, Canada
Stollery Children's Hospital
🇨🇦Edmonton, Alberta, Canada
Research transition Facility
🇨🇦Edmonton, Alberta, Canada
Grey Nuns Community Hospital
🇨🇦Edmonton, Alberta, Canada
Hasbro Children's Hospital
🇺🇸Providence, Rhode Island, United States