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

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.

Study Overview

• Status: Completed
• Conditions: Anemia, Sickle Cell
• Intervention / Treatment: Drug: Rivipansel; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 345
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • University of Alberta Hospital
    • Edmonton, Alberta, Canada, T5H 3V9
      • Royal Alexandra Hospital
    • Edmonton, Alberta, Canada, T6G 2B7
      • Stollery Children's Hospital
    • Edmonton, Alberta, Canada, T6L 5X8
      • Grey Nuns Community Hospital
    • Edmonton, Alberta, Canada, T5R 4H5
      • Miseracordia Community Hospital
    • Edmonton, Alberta, Canada, T6G 1Z1
      • Kaye Edmonton Clinic 3 C
    • Edmonton, Alberta, Canada, T6G 1Z1
      • University of Alberta Hospital, Pharmacy Services
    • Edmonton, Alberta, Canada, T6G 2V2
      • Research transition Facility
  • British Columbia
    • Vancouver, British Columbia, Canada, V6Z 1Y6
      • St. Paul's Hospital
    • Vancouver, British Columbia, Canada, V6E 1M7
      • St. Paul's Hospital
  • Ontario
    • Ottawa, Ontario, Canada, K1H 8L1
      • Children's Hospital of Eastern Ontario
    • Toronto, Ontario, Canada, M5G1X8
      • The Hospital for Sick Children
    • Toronto, Ontario, Canada, M5G 2C4
      • University Health Network, Toronto General Hospital
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • Centre Hospitalier Universitaire Sainte-Justine
    • Montreal, Quebec, Canada, H4A 3J1
      • McGill University Health Centre, Royal Victoria Hospital
    • Montreal, Quebec, Canada, H4A 3J1
      • The Montreal Children's Hospital/ McGill University Health Centre
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • University of South Alabama Children's and Women's Hospital
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital Research Pharmacy
  • California
    • Oakland, California, United States, 94609
      • UCSF Benioff Children's Hospital Oakland
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center
    • Sacramento, California, United States, 95817
      • University of California Davis Medical Center
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center Main Hospital
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Hospital
    • Aurora, Colorado, United States, 80045
      • University of Colorado CTRC
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Medstar Health Research Institute
    • Washington, District of Columbia, United States, 20060
      • Howard University Hospital
    • Washington, District of Columbia, United States, 20001
      • Howard University Center for Sickle Cell Disease
  • Florida
    • Fort Myers, Florida, United States, 33908
      • Golisano Children's Hospital of Southwest Florida
    • Gainesville, Florida, United States, 32610
      • UF Health Shands Hospital
    • Gainesville, Florida, United States, 32608
      • UF Health Shands Cancer Hospital
    • Gainesville, Florida, United States, 32608
      • UF Health Davis Cancer Pavillion and Shands Med Plaza
    • Miami, Florida, United States, 33136
      • University of Miami
    • Miami, Florida, United States, 33136
      • Jackson Memorial Hospital
    • Tampa, Florida, United States, 33606
      • Tampa General Hospital
    • Tampa, Florida, United States, 33606
      • University of South Florida
    • Tampa, Florida, United States, 33607
      • St. Joseph's Children's Hospital
    • Tampa, Florida, United States, 33606
      • Investigational Drug Service Tampa General Hospital
    • Tampa, Florida, United States, 33606
      • Tampa General Hospital Center of Research Excellence
    • West Palm Beach, Florida, United States, 33407
      • St. Mary's Medical Center
    • West Palm Beach, Florida, United States, 33407
      • Children's Hematology and Oncology Associates
  • Georgia
    • Atlanta, Georgia, United States, 30303
      • Grady Health System
    • Atlanta, Georgia, United States, 30322
      • Emory Children's Center
    • Atlanta, Georgia, United States, 30303
      • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta: Scottish Rite Campus
    • Augusta, Georgia, United States, 30912
      • Augusta University Medical Center
    • Augusta, Georgia, United States, 30912
      • Augusta University
    • Augusta, Georgia, United States, 30912
      • Augusta University Clinical Research Pharmacy
    • Savannah, Georgia, United States, 31404
      • Memorial Health University Medical Center
    • Savannah, Georgia, United States, 31404
      • Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct
  • Illinois
    • Chicago, Illinois, United States, 60612
      • University of Illinois Hospital and Health Sciences System
    • Chicago, Illinois, United States, 60612
      • University of Illinois at Chicago Clinical Research Center
    • Chicago, Illinois, United States, 60637
      • The University of Chicago/Comer Children's Hospital
    • Chicago, Illinois, United States, 60637
      • University of Chicago, Investigational Drug Service Pharmacy
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Kosair Charities Pediatric Clinical Research Unit
    • Louisville, Kentucky, United States, 40202
      • University of Louisville Health Sciences Center
    • Louisville, Kentucky, United States, 40202
      • Norton Children´s Hospital
    • Louisville, Kentucky, United States, 40202
      • The Novak Center for Children's Health
    • Louisville, Kentucky, United States, 40202
      • University of Louisville Physicians Pediatric Hematology Oncology
  • Louisiana
    • Baton Rouge, Louisiana, United States, 70808
      • Our Lady of the Lake Regional Medical Center
    • Baton Rouge, Louisiana, United States, 70808
      • St. Jude Affiliate Clinic
    • Baton Rouge, Louisiana, United States, 70809
      • Our Lady of the Lake Physician Group-Medical Oncology
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Medical System
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Medicine
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Medicine
    • Baltimore, Maryland, United States, 21205
      • The Johns Hopkins University School of Medicine
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Medical System Investigational Pharmacy
    • Baltimore, Maryland, United States, 21287-6180
      • The Johns Hopkins Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02118
      • Boston Medical Center
    • Boston, Massachusetts, United States, 02115
      • Brigham and Womens Hospital
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
    • Boston, Massachusetts, United States, 02118
      • Boston University Medical Center
    • Boston, Massachusetts, United States, 02115
      • Center for Clinical Investigation, Brigham & Women's Hospital
    • Boston, Massachusetts, United States, 02115
      • Investigational Drug Services
    • Boston, Massachusetts, United States, 02118
      • Boston Medical Center E7E
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Children's Hospital of Michigan
    • Detroit, Michigan, United States, 48201
      • Detroit Medical Center Pharmacy
    • Detroit, Michigan, United States, 48201
      • Wayne State University / Detroit Receiving Hospital
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • University of Mississippi Medical Center
    • Jackson, Mississippi, United States, 39216
      • University of Mississippi Medical Center-Outpatient Clinical Research Unit
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
    • Saint Louis, Missouri, United States, 63110
      • Barnes-Jewish Hospital
    • Saint Louis, Missouri, United States, 63108
      • Center for Outpatient Health
    • Saint Louis, Missouri, United States, 63110
      • Center for Advanced Medicine
    • Saint Louis, Missouri, United States, 63110
      • Barnes-Jewish Hospital Department of Pharmacy
  • New Jersey
    • New Brunswick, New Jersey, United States, 08903
      • Rutgers Cancer Institute of New Jersey
    • New Brunswick, New Jersey, United States, 08901
      • Rutgers-Robert Wood Johnson Medical School
    • New Brunswick, New Jersey, United States, 08901
      • Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital
  • New York
    • Bronx, New York, United States, 10461
      • Jacobi Medical Center
    • Brooklyn, New York, United States, 11203
      • Kings County Hospital Center
    • Brooklyn, New York, United States, 11203
      • Kings County Hospital Center - Pharmacy Department
    • Brooklyn, New York, United States, 11203
      • State University of New York (SUNY) - Downstate Medical Center
    • Brooklyn, New York, United States, 11203
      • SUNY Downstate Medical Center University Hospital of Brooklyn
    • New York, New York, United States, 10029
      • Icahn School of Medicine at Mount Sinai
    • New York, New York, United States, 10032
      • Columbia University Medical Center Research Pharmacy
    • New York, New York, United States, 10032
      • MS CHONY Pediatric Emergency Department
    • New York, New York, United States, 10032
      • MS CHONY Pediatric Hematology/Oncology Unit
    • Staten Island, New York, United States, 10305
      • Staten Island University Hospital
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
    • Durham, North Carolina, United States, 27710
      • Duke University Hospital
    • Greenville, North Carolina, United States, 27834
      • Vidant Medical Center
    • Greenville, North Carolina, United States, 27834
      • Leo W. Jenkins Cancer Center
    • Greenville, North Carolina, United States, 27834
      • East Carolina University Brody School of Medicine
  • Ohio
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center/ Investigational Pharmacy
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center/Research Office
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Physicians Company LLC
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati- Hoxworth Building
    • Cincinnati, Ohio, United States, 45229
      • UC Health Ridgeway Hospital
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Investigational Drug Services
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
    • Columbus, Ohio, United States, 43205
      • Nationwide Childrens Hospital
    • Columbus, Ohio, United States, 43203
      • The Ohio State University Wexner Medical Center East
    • Columbus, Ohio, United States, 43210
      • The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
    • Dayton, Ohio, United States, 45409
      • Miami Valley Hospital
    • Dayton, Ohio, United States, 45402
      • Five Rivers Medical Surgical Health Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19141
      • Einstein Medical Center Philadelphia
    • Pittsburgh, Pennsylvania, United States, 15232
      • UPMC Hillman Cancer Center
    • Pittsburgh, Pennsylvania, United States, 15224
      • Children's Hospital of Pittsburgh of UPMC
    • Pittsburgh, Pennsylvania, United States, 15213
      • UPMC Presbyterian
  • Rhode Island
    • Providence, Rhode Island, United States, 02906
      • The Miriam Hospital
    • Providence, Rhode Island, United States, 02903
      • Rhode Island Hospital
    • Providence, Rhode Island, United States, 02903
      • Hasbro Children's Hospital
    • Providence, Rhode Island, United States, 02903
      • Rhode Island Hospital-Pharmacy Service
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina - Hospital
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center
    • Charleston, South Carolina, United States, 29425
      • MUSC Investigational Drug Services
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Hematology and Oncology Center
    • Grapevine, Texas, United States, 76051
      • Cook Children's Hematology and Oncology Center-Grapevine
    • Houston, Texas, United States, 77030
      • University of Texas Medical School
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital - Clinical Research Center
    • Houston, Texas, United States, 77030
      • Texas Children´s Hospital
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University
    • Richmond, Virginia, United States, 23298
      • Main Hospital - Virginia Commonwealth University
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University-Investigational Drug Services

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Rivipansel Treatment Arm	Drug: Rivipansel; 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.; Other Names: GMI-1070
Placebo Comparator: Placebo Treatment Arm	Other: Placebo; Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.; Other Names: phosphate buffered saline (PBS)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Discharge From Hospital	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.	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	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.	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	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.	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	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.	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	Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported.	Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) as Per Genotype	AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. SAE: an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. An adverse event was considered a treatment-emergent adverse event (TEAE) if the event started during the effective duration of treatment (all events that started on or after the first dosing). AEs included both serious and non-serious adverse events. Participants with AEs and SAEs were categorized by genotype categories. Category 1= participants with hemoglobin SS, hemoglobin S beta0 thalassemia and hemoglobin SD; category 2= participants with hemoglobin SC, hemoglobin S beta+ thalassemia and hemoglobin S-Variant (other than HbSD).	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	AE: any untoward medical occurrence in a participant who received investigational product without regard to possibility of a causal relationship. AEs were classified according to the severity in 3 categories a) mild - AEs did not interfere with participant's usual function, b) moderate - AEs interfered to some extent with participant's usual function, c) severe - AEs interfered significantly with participant's usual function.	Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Number of Participants With Clinical Laboratory Abnormalities	Hematology: hemoglobin, hematocrit, erythrocytes <0.8*lower limit of normal (LLN), reticulocytes <0.5*LLN >1.5*ULN, platelets<0.5*LLN>1.75*upper limit of normal (ULN), reticulocytes/erythrocytes<0.5*LLN>1.5*ULN, leukocytes <0.6*LLN >1.5*ULN, lymphocytes, lymphocytes/leukocytes, neutrophils, neutrophils/leukocytes <0.8*LLN >1.2*ULN, basophils, basophils/leukocytes, eosinophils, eosinophils/leukocytes, monocytes monocytes/leukocytes >1.2*ULN. Clinical chemistry: bilirubin, direct, bilirubin, indirect bilirubin>1.5*ULN, aspartate aminotransferase (AT), alanine AT, lactate dehydrogenase, alkaline phosphatase>3.0*ULN, urea nitrogen, urea, creatinine >1.3*ULN, sodium<0.95*LLN>1.05*ULN, potassium, chloride, bicarbonate<0.9*LLN>1.1*ULN, glucose<0.6*LLN>1.5*ULN, estimated glomerular filtration rate <=60. Urinalysis: urine glucose, ketones, urine protein, urine hemoglobin, nitrite, leukocyte esterase >=1.	Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Number of Participants With Increase in Grades From Baseline in Clinical Laboratory Parameters Over the Study	Hemoglobin(grade [G] 0:>11g/dl,G1:>9-11g/dl,G2:>7-9g/dl,G3:5-7g/dl,G4:<5g/dl),reticulocytes count(G0:<1%,G1:1-5%,G2:>5-10%,G3:>10-20%,G4:>20%),leukocytes(G0:<=upper limit of normal [ULN],G1:>ULN-15,000/mm^3,G2:>15,000- 20,000/mm^3,G3:>20,000- 50,000/mm^, G4:>50,000/mm^3),neutrophils(G0:>=LLN, G1:<LLN-1,500/mm^3, G2:<1,500-1,000/mm^3, G3:<1,000-500/mm^3, G4:<500/mm^3),blood urea nitrogen, creatinine(G0:<=ULN, G1:>ULN-1.5*UL, G2:>1.5-3.0*ULN, G3:>3.0*ULN, G4:>6.0*ULN), lactate dehydrogenase, alanine transaminase, aspartate aminotransferase(G0:<=ULN, G1:>ULN-3.0*ULN, G2:>3.0-5.0*ULN, G3:>5.0-20.0*ULN, G4:>20.0*ULN), bilirubin(G0:<=ULN, G1:>ULN-1.5*ULN, G2:>1.5-3.0*ULN, G3:>3.0-10.0*ULN, G4:>10.0*ULN), urine protein(G0:0-15mg/dL, G1:>15-30mg/dL, G2:>30-100mg/dL, G3:>100-300mg/dL, G4:>300mg/dL), platelet(G0:>=150K, G1:>=100K-<150K, G2:>=50K <100K, G3:<50K), eGFR (G0:>=90 mL/min/1.73m^2, G1:>=60-<90mL/min/1.73m^2, G2:>=30-<60mL/min/1.73m^2, G3:>=15-<30mL/min/1.73m^2, G4:<15 mL/min/1.73m^2)	Baseline up to the 35-day post discharge visit (up to an average of Day 43)
Number of Participants With Clinically Significant Changes in Physical Examination	Physical examination included assessment of the general appearance and the skin, head, ears, eyes, nose, mouth, throat, spine, neck, thyroid, chest, extremities, lymph nodes and abdomen (including liver and kidneys) plus the respiratory, cardiovascular, musculoskeletal, neurological and genitourinary systems. Clinical significance was assessed by the Investigator.	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)
Number of Participants With Treatment Related Changes From Baseline in Vital Signs Over the Study	Vital signs included temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure. Relatedness to treatment was assessed by the Investigator.	Baseline (Day 1) up to the 35-day post discharge visit (up to an average of Day 43)
Percentage of Participants With Adjudicated Acute Chest Syndrome (ACS)	Investigator reported events of Acute Chest Syndrome (ACS) and other reported respiratory events were sent for adjudication by the Acute Chest Syndrome Safety Endpoint Adjudication Committee. The committee, which consisted of physicians with relevant SCD expertise, evaluated these events and determined whether they were consistent with cases of ACS.	Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Percentage of Participants With Severe Adjudicated and/or Generalized Cutaneous Manifestations	Investigator reported cutaneous events were sent for adjudication by the Cutaneous Manifestations Safety Endpoint Adjudication Committee. The committee, which consisted of dermatologists, evaluated these events and determined whether they were cases of severe and/or generalized cutaneous manifestations and specifically whether any event was consistent with Acute Generalized Exanthematous Pustulosis.	Day 1 up to the 35-day post discharge visit (up to an average of Day 43)
Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 7, 14 and 30 Days of Discharge From Hospital	Percentage of participants re-hospitalized for VOC within 7, 14 and 30 days of hospital discharge.	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

Collaborators and Investigators

• Sponsor: Biossil Inc.
• Collaborators: GlycoMimetics Incorporated
• Investigators: Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

General Publications

• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (Actual): June 17, 2015
• Primary Completion (Actual): May 3, 2019
• Study Completion (Actual): June 27, 2019

Study Registration Dates

• First Submitted: June 12, 2014
• First Submitted That Met QC Criteria: July 7, 2014
• First Posted (Estimated): July 10, 2014

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 18, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: vaso-occlusive crisis; Sickle Cell Disease; SCD; VOC; Sickle Cell Anemia; pain crisis; Sickle Cell Disorders; rivipansel; GMI-1070; selectin inhibitor
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: B5201002; RESET (Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No