Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease

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

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

Study Overview

• Status: Terminated
• Conditions: Sickle Cell Disease; Sickle Cell Anemia; Vaso-occlusive Crisis; Pain Crisis; Sickle Cell Disorders
• Intervention / Treatment: Drug: Rivipansel

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.

• Study Type: Interventional
• Enrollment (Actual): 154
• 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
      • University of Alberta Hospital, Pharmacy Services
    • Edmonton, Alberta, Canada, T6G 2V2
      • Research transition Facility
    • Edmonton, Alberta, Canada, T6G 1Z1
      • Kaye Edmonton Clinic 3C
  • Ontario
    • Ottawa, Ontario, Canada, K1H 8L1
      • Children's Hospital of Eastern Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • Centre Hospitalier Universitaire Sainte-Justine
    • Montreal, Quebec, Canada, H4A3J1
      • The Montreal Children's Hospital / McGill University Health Centre
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • University of South Alabama Women's and Children'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
    • Sacramento, California, United States, 95817
      • University of California Davis Medical Center
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center Main Hospital
  • 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 Childrens Hospital of Southwest Florida
    • Miami, Florida, United States, 33136
      • University of Miami
    • Miami, Florida, United States, 33136
      • Jackson Memorial Hospital
    • West Palm Beach, Florida, United States, 33407
      • St. Mary's Medical Center
  • Georgia
    • Atlanta, Georgia, United States, 30303
      • Grady Health System
    • Atlanta, Georgia, United States, 30322
      • Emory Children's Center
    • Atlanta, Georgia, United States, 30342
      • Children's Healthcare of Atlanta: Scottish Rite Campus
    • 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/
    • Savannah, Georgia, United States, 31404
      • Memorial Health University Medical Center
    • Savannah, Georgia, United States, 31404
      • Memorial Family Medicine Center
  • Illinois
    • 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
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Medical System
    • Baltimore, Maryland, United States, 21287
      • 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, 21205
      • Johns Hopkins Department of Medicine Clinical Trials Unit
    • Baltimore, Maryland, United States, 21287-6180
      • The Johns Hopkins Hospital Department of Pharmacy Services
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Brigham and Women's Hospital
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
    • Boston, Massachusetts, United States, 02115
      • Investigational Drug Services
    • Boston, Massachusetts, United States, 02115
      • Center for Clinical Investigation, Brigham and Women's Hospital
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Children's Hospital of Michigan
  • 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
    • 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
      • 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, 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
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
    • Durham, North Carolina, United States, 27710
      • Duke University Hospital, Investigational Drug Service
    • 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
    • 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 Physicians Company LLC
    • Cincinnati, Ohio, United States, 45229
      • UC Health Ridgeway Hospital
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati - Hoxworth Building
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center / Investigational Pharmacy
    • Cincinnati, Ohio, United States, 45219
      • University of Cincinnati Medical Center / Research Office
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Wexner Medical Center
    • Columbus, Ohio, United States, 43210
      • The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
    • Columbus, Ohio, United States, 43203
      • The Ohio State University Investigational Drug Services
    • Columbus, Ohio, United States, 43203
      • The Ohio State University Wexner Center East
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15232
      • UPMC Hillman Cancer Center
    • 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 Lifespan Comprehensive Sickle Cell Center
    • Charleston, South Carolina, United States, 29425
      • MUSC Investigational Drug Services
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina-Hospital
  • 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
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital
    • Salt Lake City, Utah, United States, 84113
      • Primary Children's Hospital Laboratory
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Main Hospital-VCU
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University - Investigational Drug Services
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University- Clinical Research Services Unit

Participation Criteria

Eligibility Criteria

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

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Includes one adult stratum (>18 years old) and one pediatric stratum (12-17 years old). Subjects aged 12 and over who weigh >40 kg, will receive a loading dose of 1680 mg followed by a maintenance dose of 840 mg.	Drug: Rivipansel; Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses.; Other Names: GMI-1070
Experimental: Cohort 2; Includes one pediatric stratum (6-11 years old). Subjects 6 to 11 years of age or subjects who weigh 40 kg, will receive a loading dose of 40 mg/kg (maximum of 1680 mg) followed by a maintenance dose of 20 mg/kg (maximum of 840 mg).	Drug: Rivipansel; Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses.; Other Names: GMI-1070

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study.	Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study, number of TEAEs over the study and rate of TEAEs per subject per Vaso-Occlusive Crisis (VOC) will be summarized overall, by system organ class and by preferred term.	18 months
Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study.	Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study, number of events of adjudicated ACS and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.	18 months
Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study	Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study, number of events of adjudicated severe and/or generalized cutaneous manifestations and the rates of these events per subject per Vaso-Occlusive Crisis (VOC) will be summarized.	18 months
Number (%) of subjects with serious adverse events (SAEs) over the study.	Number (%) of subjects with serious adverse events (SAEs) over the study, number of SAEs over the study and rate of SAEs per subject per VOC will be summarized overall, by system organ class and by preferred term.	18 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Subject re hospitalization	Number (%) of subjects re hospitalized for VOC within 7, 14, and 30 days of most recent discharge will be provided overall. Number of re hospitalizations for VOC within 7, 14, and 30 days of most recent discharge and the rate of re hospitalization for VOC within 7, 14, and 30 days of most recent discharge per subject per VOC will be provided as well.	18 months

Collaborators and Investigators

• Sponsor: GlycoMimetics Incorporated

Publications and helpful links

General Publications

• 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): December 16, 2015
• Primary Completion (Actual): November 15, 2019
• Study Completion (Actual): November 15, 2019

Study Registration Dates

• First Submitted: April 29, 2015
• First Submitted That Met QC Criteria: April 29, 2015
• First Posted (Estimate): May 4, 2015

Study Record Updates

• Last Update Posted (Actual): February 10, 2021
• Last Update Submitted That Met QC Criteria: February 8, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Keywords: SCD; VOC; GMI-1070; Rivipansel; Selectin Inhibitor
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: B5201003

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