Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

June 16, 2020 updated by: GlycoMimetics Incorporated

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

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

345

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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:

  • 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to readiness-for-discharge.
Time Frame: Assessments will be every 4 hours (from 6am to 10pm, daily) for the duration of hospitalization, an expected average of 5 days
Time to readiness-for-discharge, defined as the difference between the readiness-for-discharge date and time and the start date and time of the first infusion of study drug.
Assessments will be every 4 hours (from 6am to 10pm, daily) for the duration of hospitalization, an expected average of 5 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to discharge.
Time Frame: Participants will be followed for the duration of hospital stay, an expected average of 5 days
Time to discharge, is the difference between the time and date of hospital discharge order and the time and date of start of the first infusion of study drug.
Participants will be followed for the duration of hospital stay, an expected average of 5 days
Cumulative IV opioid consumption from the time of the loading dose of study drug to discharge.
Time Frame: Participants will be followed for the duration of hospital stay, an expected average of 5 days
Cumulative IV opioid consumption from the time of the loading dose of study drug to discharge.
Participants will be followed for the duration of hospital stay, an expected average of 5 days
Time to discontinuation of IV opioids.
Time Frame: Participants will be followed for the duration of hospital stay, an expected average of 5 days
Time to discontinuation of IV opioids.
Participants will be followed for the duration of hospital stay, an expected average of 5 days
Cumulative IV opioid consumption within the first 24 hours post-loading dose of study drug.
Time Frame: Day 1
Cumulative IV opioid consumption within the first 24 hours post-loading dose of study drug.
Day 1
Percent of subjects re-hospitalized for VOC within 3 days of discharge.
Time Frame: Within 3 days of discharge
Percent of subjects re-hospitalized for VOC within 3 days of discharge.
Within 3 days of discharge

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

GlycoMimetics Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 17, 2015

Primary Completion (Actual)

May 3, 2019

Study Completion (Actual)

June 25, 2019

Study Registration Dates

First Submitted

June 12, 2014

First Submitted That Met QC Criteria

July 7, 2014

First Posted (Estimate)

July 10, 2014

Study Record Updates

Last Update Posted (Actual)

June 22, 2020

Last Update Submitted That Met QC Criteria

June 16, 2020

Last Verified

June 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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