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Anemia, Sickle CellPhase 3NCT02187003

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

A Phase 3 study testing Rivipansel in people with Anemia, Sickle Cell, run by Biossil Inc. at 142 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 6 years and older
Conditions the study is for
Anemia, Sickle Cell
What is being tested?
Rivipansel
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
345 participants
Where
142 sitesin 2 countries
Study dates
17 Jun 2015to 27 Jun 2019
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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

Trial locations and contacts

142 sites in 2 countries.

142 sites in 2 countries

United States126 sites

  • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center

    Atlanta, Georgia, 30303

  • Grady Health System

    Atlanta, Georgia, 30303

  • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center

    Atlanta, Georgia, 30322

  • Emory Children's Center

    Atlanta, Georgia, 30322

  • Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center

    Atlanta, Georgia, 30342

  • Children's Healthcare of Atlanta: Scottish Rite Campus

    Atlanta, Georgia, 30342

  • Augusta University Clinical Research Pharmacy

    Augusta, Georgia, 30912

  • Augusta University Medical Center

    Augusta, Georgia, 30912

  • Augusta University

    Augusta, Georgia, 30912

  • University of Colorado CTRC

    Aurora, Colorado, 80045

  • University of Colorado Hospital

    Aurora, Colorado, 80045

  • University of Maryland Medical System Investigational Pharmacy

    Baltimore, Maryland, 21201

  • University of Maryland Medical System

    Baltimore, Maryland, 21201

  • Johns Hopkins Medicine

    Baltimore, Maryland, 21205

  • The Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21205

  • The Johns Hopkins Hospital

    Baltimore, Maryland, 21287-6180

  • Johns Hopkins Medicine

    Baltimore, Maryland, 21287

  • Our Lady of the Lake Regional Medical Center

    Baton Rouge, Louisiana, 70808

  • St. Jude Affiliate Clinic

    Baton Rouge, Louisiana, 70808

  • Our Lady of the Lake Physician Group-Medical Oncology

    Baton Rouge, Louisiana, 70809

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Brigham and Womens Hospital

    Boston, Massachusetts, 02115

  • Center for Clinical Investigation, Brigham & Women's Hospital

    Boston, Massachusetts, 02115

  • Investigational Drug Services

    Boston, Massachusetts, 02115

  • Boston Medical Center E7E

    Boston, Massachusetts, 02118

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Boston University Medical Center

    Boston, Massachusetts, 02118

  • Kings County Hospital Center - Pharmacy Department

    Brooklyn, New York, 11203

  • Kings County Hospital Center

    Brooklyn, New York, 11203

  • State University of New York (SUNY) - Downstate Medical Center

    Brooklyn, New York, 11203

  • SUNY Downstate Medical Center University Hospital of Brooklyn

    Brooklyn, New York, 11203

  • Medical University of South Carolina - Hospital

    Charleston, South Carolina, 29425

  • Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center

    Charleston, South Carolina, 29425

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • MUSC Investigational Drug Services

    Charleston, South Carolina, 29425

  • University of Illinois at Chicago Clinical Research Center

    Chicago, Illinois, 60612

  • University of Illinois Hospital and Health Sciences System

    Chicago, Illinois, 60612

  • The University of Chicago/Comer Children's Hospital

    Chicago, Illinois, 60637

  • University of Chicago, Investigational Drug Service Pharmacy

    Chicago, Illinois, 60637

  • University of Cincinnati Medical Center/ Investigational Pharmacy

    Cincinnati, Ohio, 45219

  • University of Cincinnati Medical Center/Research Office

    Cincinnati, Ohio, 45219

  • University of Cincinnati Medical Center

    Cincinnati, Ohio, 45219

  • University of Cincinnati Physicians Company LLC

    Cincinnati, Ohio, 45219

  • University of Cincinnati- Hoxworth Building

    Cincinnati, Ohio, 45219

  • UC Health Ridgeway Hospital

    Cincinnati, Ohio, 45229

  • The Ohio State University Wexner Medical Center East

    Columbus, Ohio, 43203

  • Nationwide Childrens Hospital

    Columbus, Ohio, 43205

  • The Ohio State University Investigational Drug Services

    Columbus, Ohio, 43210

  • The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute

    Columbus, Ohio, 43210

  • The Ohio State University Wexner Medical Center

    Columbus, Ohio, 43210

  • Five Rivers Medical Surgical Health Center

    Dayton, Ohio, 45402

  • Miami Valley Hospital

    Dayton, Ohio, 45409

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • Detroit Medical Center Pharmacy

    Detroit, Michigan, 48201

  • Wayne State University / Detroit Receiving Hospital

    Detroit, Michigan, 48201

  • Duke University Hospital

    Durham, North Carolina, 27710

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Golisano Children's Hospital of Southwest Florida

    Fort Myers, Florida, 33908

  • Cook Children's Hematology and Oncology Center

    Fort Worth, Texas, 76104

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

  • UF Health Davis Cancer Pavillion and Shands Med Plaza

    Gainesville, Florida, 32608

  • UF Health Shands Cancer Hospital

    Gainesville, Florida, 32608

  • UF Health Shands Hospital

    Gainesville, Florida, 32610

  • Cook Children's Hematology and Oncology Center-Grapevine

    Grapevine, Texas, 76051

  • East Carolina University Brody School of Medicine

    Greenville, North Carolina, 27834

  • Leo W. Jenkins Cancer Center

    Greenville, North Carolina, 27834

  • Vidant Medical Center

    Greenville, North Carolina, 27834

  • Texas Children's Hospital - Clinical Research Center

    Houston, Texas, 77030

  • Texas Children´s Hospital

    Houston, Texas, 77030

  • University of Texas Medical School

    Houston, Texas, 77030

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • University of Mississippi Medical Center-Outpatient Clinical Research Unit

    Jackson, Mississippi, 39216

  • University Of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • Children's Mercy Hospital

    Kansas City, Missouri, 64108

  • Arkansas Children's Hospital Research Pharmacy

    Little Rock, Arkansas, 72202

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • Kosair Charities Pediatric Clinical Research Unit

    Louisville, Kentucky, 40202

  • Norton Children´s Hospital

    Louisville, Kentucky, 40202

  • The Novak Center for Children's Health

    Louisville, Kentucky, 40202

  • University of Louisville Health Sciences Center

    Louisville, Kentucky, 40202

  • University of Louisville Physicians Pediatric Hematology Oncology

    Louisville, Kentucky, 40202

  • Jackson Memorial Hospital

    Miami, Florida, 33136

  • University Of Miami

    Miami, Florida, 33136

  • University of South Alabama Children's and Women's Hospital

    Mobile, Alabama, 36604

  • Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital

    New Brunswick, New Jersey, 08901

  • Rutgers-Robert Wood Johnson Medical School

    New Brunswick, New Jersey, 08901

  • Rutgers Cancer Institute Of New Jersey

    New Brunswick, New Jersey, 08903

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Columbia University Medical Center Research Pharmacy

    New York, New York, 10032

  • MS CHONY Pediatric Emergency Department

    New York, New York, 10032

  • MS CHONY Pediatric Hematology/Oncology Unit

    New York, New York, 10032

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609

  • Einstein Medical Center Philadelphia

    Philadelphia, Pennsylvania, 19141

  • UPMC Presbyterian

    Pittsburgh, Pennsylvania, 15213

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Hasbro Children's Hospital

    Providence, Rhode Island, 02903

  • Rhode Island Hospital-Pharmacy Service

    Providence, Rhode Island, 02903

  • Rhode Island Hospital

    Providence, Rhode Island, 02903

  • The Miriam Hospital

    Providence, Rhode Island, 02906

  • Main Hospital - Virginia Commonwealth University

    Richmond, Virginia, 23298

  • Virginia Commonwealth University-Investigational Drug Services

    Richmond, Virginia, 23298

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

  • UC Davis Medical Center Main Hospital

    Sacramento, California, 95817

  • UC Davis Medical Center

    Sacramento, California, 95817

  • University of California Davis Medical Center

    Sacramento, California, 95817

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

  • Memorial Family Medicine Ctr. @Memorial Health Univ Med Ct

    Savannah, Georgia, 31404

  • Memorial Health University Medical Center

    Savannah, Georgia, 31404

  • Center for Outpatient Health

    St Louis, Missouri, 63108

  • Barnes-Jewish Hospital Department of Pharmacy

    St Louis, Missouri, 63110

  • Barnes-Jewish Hospital

    St Louis, Missouri, 63110

  • Center for Advanced Medicine

    St Louis, Missouri, 63110

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Staten Island University Hospital

    Staten Island, New York, 10305

  • Investigational Drug Service Tampa General Hospital

    Tampa, Florida, 33606

  • Tampa General Hospital Center of Research Excellence

    Tampa, Florida, 33606

  • Tampa General Hospital

    Tampa, Florida, 33606

  • University of South Florida

    Tampa, Florida, 33606

  • St. Joseph's Children's Hospital

    Tampa, Florida, 33607

  • Jacobi Medical Center

    The Bronx, New York, 10461

  • Howard University Center for Sickle Cell Disease

    Washington D.C., District of Columbia, 20001

  • Medstar Health Research Institute

    Washington D.C., District of Columbia, 20010

  • Howard University Hospital

    Washington D.C., District of Columbia, 20060

  • Children's Hematology and Oncology Associates

    West Palm Beach, Florida, 33407

  • St. Mary's Medical Center

    West Palm Beach, Florida, 33407

Other trials in United States

Canada16 sites

  • Royal Alexandra Hospital

    Edmonton, Alberta, T5H 3V9

  • Miseracordia Community Hospital

    Edmonton, Alberta, T5R 4H5

  • Kaye Edmonton Clinic 3 C

    Edmonton, Alberta, T6G 1Z1

  • University of Alberta Hospital, Pharmacy Services

    Edmonton, Alberta, T6G 1Z1

  • Stollery Children's Hospital

    Edmonton, Alberta, T6G 2B7

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2B7

  • Research transition Facility

    Edmonton, Alberta, T6G 2V2

  • Grey Nuns Community Hospital

    Edmonton, Alberta, T6L 5X8

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3T 1C5

  • McGill University Health Centre, Royal Victoria Hospital

    Montreal, Quebec, H4A 3J1

  • The Montreal Children's Hospital/ McGill University Health Centre

    Montreal, Quebec, H4A 3J1

  • Children's Hospital Of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

  • University Health Network, Toronto General Hospital

    Toronto, Ontario, M5G 2C4

  • The Hospital for Sick Children

    Toronto, Ontario, M5G1X8

  • St. Paul's Hospital

    Vancouver, British Columbia, V6E 1M7

  • St. Paul's Hospital

    Vancouver, British Columbia, V6Z 1Y6

Other trials in Canada

About this study

Official title: 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.

Being tested
Rivipansel, Placebo
Keywords
Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders, pain crisis, vaso-occlusive crisis, rivipansel, GMI-1070, selectin inhibitor, SCD, VOC
Medical subject headings
Anemia, Sickle Cell, Vaso-Occlusive Crises

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RivipanselDrug

    Also called GMI-1070

    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.

  • PlaceboOther

    Also called phosphate buffered saline (PBS)

    Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.

Study groups in NCT02187003
Study groupRoleWhat they receive
Rivipansel Treatment ArmExperimentalNot described
Receives: Drug: Rivipansel
Placebo Treatment ArmPlacebo comparatorNot described
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 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)

  1. 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.

    Measured: 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)

  2. 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.

    Measured: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)

  3. 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.

    Measured: Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)

  4. 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.

    Measured: 24 hours post first IV infusion of loading dose of study drug on Day 1

  5. 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.

    Measured: Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jul 2014 (estimated)
  2. Study start17 Jun 2015
  3. Primary completion3 May 2019
  4. Study completion27 Jun 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
345 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02187003
Sponsor's protocol ID
B5201002
Other IDs
RESET
Status last verified
Feb 2025
First submitted
12 Jun 2014
Last updated
11 Mar 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

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.

    PubMed 35981565

  • 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.

    PubMed 35120836

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Rivipansel in people with Anemia, Sickle Cell, run by Biossil Inc. at 142 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 142 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to Readiness for Discharge From Hospital (time frame: Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02187003, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2025.

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