Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
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
2 other identifiers
interventional
345
2 countries
142
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Jun 2015
Typical duration for phase_3
142 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 12, 2014
CompletedFirst Posted
Study publicly available on registry
July 10, 2014
CompletedStudy Start
First participant enrolled
June 17, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 3, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
June 27, 2019
CompletedResults Posted
Study results publicly available
March 11, 2025
CompletedMarch 11, 2025
February 1, 2025
3.9 years
June 12, 2014
February 26, 2024
February 18, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Time to Readiness for Discharge From Hospital
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.
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 (5)
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)
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)
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)
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
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
Other Outcomes (9)
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) as Per Genotype
Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Number of Participants With Treatment Emergent Adverse Event (AEs) as Per Severity
Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Number of Participants With Clinical Laboratory Abnormalities
Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
- +6 more other outcomes
Study Arms (2)
Rivipansel Treatment Arm
EXPERIMENTALPlacebo Treatment Arm
PLACEBO COMPARATORInterventions
Rivipansel (GMI-1070) will be infused intravenously every 12 hours up to 15 doses maximum. Subjects aged 12 and over who weigh more than 40 kilograms will receive a dose of 1680 mg of rivipansel, followed by a dose of 840 mg of rivipansel every 12 hours. All subjects aged 6 to 11 years and any subject who weighs 40 kilograms or less, will receive weight-based dosing (mg/kg) of 40 mg/kg of rivipansel (maximum of 1680 mg) followed by a dose of 20 mg/kg of rivipansel (maximum of 840 mg) every 12 hours.
Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biossil Inc.lead
- GlycoMimetics Incorporatedcollaborator
Study Sites (142)
University of South Alabama Children's and Women's Hospital
Mobile, Alabama, 36604, United States
Arkansas Children's Hospital Research Pharmacy
Little Rock, Arkansas, 72202, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
UC Davis Medical Center Main Hospital
Sacramento, California, 95817, United States
UC Davis Medical Center
Sacramento, California, 95817, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of Colorado CTRC
Aurora, Colorado, 80045, United States
University of Colorado Hospital
Aurora, Colorado, 80045, United States
Howard University Center for Sickle Cell Disease
Washington D.C., District of Columbia, 20001, United States
Medstar Health Research Institute
Washington D.C., District of Columbia, 20010, United States
Howard University Hospital
Washington D.C., District of Columbia, 20060, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908, United States
UF Health Davis Cancer Pavillion and Shands Med Plaza
Gainesville, Florida, 32608, United States
UF Health Shands Cancer Hospital
Gainesville, Florida, 32608, United States
UF Health Shands Hospital
Gainesville, Florida, 32610, United States
Jackson Memorial Hospital
Miami, Florida, 33136, United States
University Of Miami
Miami, Florida, 33136, United States
Investigational Drug Service Tampa General Hospital
Tampa, Florida, 33606, United States
Tampa General Hospital Center of Research Excellence
Tampa, Florida, 33606, United States
Tampa General Hospital
Tampa, Florida, 33606, United States
University of South Florida
Tampa, Florida, 33606, United States
St. Joseph's Children's Hospital
Tampa, Florida, 33607, United States
Children's Hematology and Oncology Associates
West Palm Beach, Florida, 33407, United States
St. Mary's Medical Center
West Palm Beach, Florida, 33407, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
Atlanta, Georgia, 30303, United States
Grady Health System
Atlanta, Georgia, 30303, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
Atlanta, Georgia, 30322, United States
Emory Children's Center
Atlanta, Georgia, 30322, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
Atlanta, Georgia, 30342, United States
Children's Healthcare of Atlanta: Scottish Rite Campus
Atlanta, Georgia, 30342, United States
Augusta University Clinical Research Pharmacy
Augusta, Georgia, 30912, United States
Augusta University Medical Center
Augusta, Georgia, 30912, United States
Augusta University
Augusta, Georgia, 30912, United States
Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct
Savannah, Georgia, 31404, United States
Memorial Health University Medical Center
Savannah, Georgia, 31404, United States
University of Illinois at Chicago Clinical Research Center
Chicago, Illinois, 60612, United States
University of Illinois Hospital and Health Sciences System
Chicago, Illinois, 60612, United States
The University of Chicago/Comer Children's Hospital
Chicago, Illinois, 60637, United States
University of Chicago, Investigational Drug Service Pharmacy
Chicago, Illinois, 60637, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
Norton Children´s Hospital
Louisville, Kentucky, 40202, United States
The Novak Center for Children's Health
Louisville, Kentucky, 40202, United States
University of Louisville Health Sciences Center
Louisville, Kentucky, 40202, United States
University of Louisville Physicians Pediatric Hematology Oncology
Louisville, Kentucky, 40202, United States
Our Lady of the Lake Regional Medical Center
Baton Rouge, Louisiana, 70808, United States
St. Jude Affiliate Clinic
Baton Rouge, Louisiana, 70808, United States
Our Lady of the Lake Physician Group-Medical Oncology
Baton Rouge, Louisiana, 70809, United States
University of Maryland Medical System Investigational Pharmacy
Baltimore, Maryland, 21201, United States
University of Maryland Medical System
Baltimore, Maryland, 21201, United States
Johns Hopkins Medicine
Baltimore, Maryland, 21205, United States
The Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205, United States
The Johns Hopkins Hospital
Baltimore, Maryland, 21287-6180, United States
Johns Hopkins Medicine
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Brigham and Womens Hospital
Boston, Massachusetts, 02115, United States
Center for Clinical Investigation, Brigham & Women's Hospital
Boston, Massachusetts, 02115, United States
Investigational Drug Services
Boston, Massachusetts, 02115, United States
Boston Medical Center E7E
Boston, Massachusetts, 02118, United States
Boston Medical Center
Boston, Massachusetts, 02118, United States
Boston University Medical Center
Boston, Massachusetts, 02118, United States
Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Detroit Medical Center Pharmacy
Detroit, Michigan, 48201, United States
Wayne State University / Detroit Receiving Hospital
Detroit, Michigan, 48201, United States
University of Mississippi Medical Center-Outpatient Clinical Research Unit
Jackson, Mississippi, 39216, United States
University Of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Center for Outpatient Health
St Louis, Missouri, 63108, United States
Barnes-Jewish Hospital Department of Pharmacy
St Louis, Missouri, 63110, United States
Barnes-Jewish Hospital
St Louis, Missouri, 63110, United States
Center for Advanced Medicine
St Louis, Missouri, 63110, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital
New Brunswick, New Jersey, 08901, United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
Rutgers Cancer Institute Of New Jersey
New Brunswick, New Jersey, 08903, United States
Kings County Hospital Center - Pharmacy Department
Brooklyn, New York, 11203, United States
Kings County Hospital Center
Brooklyn, New York, 11203, United States
State University of New York (SUNY) - Downstate Medical Center
Brooklyn, New York, 11203, United States
SUNY Downstate Medical Center University Hospital of Brooklyn
Brooklyn, New York, 11203, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Columbia University Medical Center Research Pharmacy
New York, New York, 10032, United States
MS CHONY Pediatric Emergency Department
New York, New York, 10032, United States
MS CHONY Pediatric Hematology/Oncology Unit
New York, New York, 10032, United States
Staten Island University Hospital
Staten Island, New York, 10305, United States
Jacobi Medical Center
The Bronx, New York, 10461, United States
Duke University Hospital
Durham, North Carolina, 27710, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
East Carolina University Brody School of Medicine
Greenville, North Carolina, 27834, United States
Leo W. Jenkins Cancer Center
Greenville, North Carolina, 27834, United States
Vidant Medical Center
Greenville, North Carolina, 27834, United States
University of Cincinnati Medical Center/ Investigational Pharmacy
Cincinnati, Ohio, 45219, United States
University of Cincinnati Medical Center/Research Office
Cincinnati, Ohio, 45219, United States
University of Cincinnati Medical Center
Cincinnati, Ohio, 45219, United States
University of Cincinnati Physicians Company LLC
Cincinnati, Ohio, 45219, United States
University of Cincinnati- Hoxworth Building
Cincinnati, Ohio, 45219, United States
UC Health Ridgeway Hospital
Cincinnati, Ohio, 45229, United States
The Ohio State University Wexner Medical Center East
Columbus, Ohio, 43203, United States
Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
The Ohio State University Investigational Drug Services
Columbus, Ohio, 43210, United States
The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
Columbus, Ohio, 43210, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210, United States
Five Rivers Medical Surgical Health Center
Dayton, Ohio, 45402, United States
Miami Valley Hospital
Dayton, Ohio, 45409, United States
Einstein Medical Center Philadelphia
Philadelphia, Pennsylvania, 19141, United States
UPMC Presbyterian
Pittsburgh, Pennsylvania, 15213, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232, United States
Hasbro Children's Hospital
Providence, Rhode Island, 02903, United States
Rhode Island Hospital-Pharmacy Service
Providence, Rhode Island, 02903, United States
Rhode Island Hospital
Providence, Rhode Island, 02903, United States
The Miriam Hospital
Providence, Rhode Island, 02906, United States
Medical University of South Carolina - Hospital
Charleston, South Carolina, 29425, United States
Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center
Charleston, South Carolina, 29425, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
MUSC Investigational Drug Services
Charleston, South Carolina, 29425, United States
Cook Children's Hematology and Oncology Center
Fort Worth, Texas, 76104, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Cook Children's Hematology and Oncology Center-Grapevine
Grapevine, Texas, 76051, United States
Texas Children's Hospital - Clinical Research Center
Houston, Texas, 77030, United States
Texas Children´s Hospital
Houston, Texas, 77030, United States
University of Texas Medical School
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Main Hospital - Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Virginia Commonwealth University-Investigational Drug Services
Richmond, Virginia, 23298, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Royal Alexandra Hospital
Edmonton, Alberta, T5H 3V9, Canada
Miseracordia Community Hospital
Edmonton, Alberta, T5R 4H5, Canada
Kaye Edmonton Clinic 3 C
Edmonton, Alberta, T6G 1Z1, Canada
University of Alberta Hospital, Pharmacy Services
Edmonton, Alberta, T6G 1Z1, Canada
Stollery Children's Hospital
Edmonton, Alberta, T6G 2B7, Canada
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7, Canada
Research transition Facility
Edmonton, Alberta, T6G 2V2, Canada
Grey Nuns Community Hospital
Edmonton, Alberta, T6L 5X8, Canada
St. Paul's Hospital
Vancouver, British Columbia, V6E 1M7, Canada
St. Paul's Hospital
Vancouver, British Columbia, V6Z 1Y6, Canada
Children's Hospital Of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
University Health Network, Toronto General Hospital
Toronto, Ontario, M5G 2C4, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G1X8, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada
McGill University Health Centre, Royal Victoria Hospital
Montreal, Quebec, H4A 3J1, Canada
The Montreal Children's Hospital/ McGill University Health Centre
Montreal, Quebec, H4A 3J1, Canada
Related Publications (2)
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.
PMID: 35981565DERIVEDRebelo 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.
PMID: 35120836DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Head of Regulatory
- Organization
- Biossil Inc.
Study Officials
- STUDY DIRECTOR
Pfizer CT.gov Call Center
Pfizer
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 12, 2014
First Posted
July 10, 2014
Study Start
June 17, 2015
Primary Completion
May 3, 2019
Study Completion
June 27, 2019
Last Updated
March 11, 2025
Results First Posted
March 11, 2025
Record last verified: 2025-02
Data Sharing
- IPD Sharing
- Will not share