Clincosm LogoSign in Get a demo

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

Sponsor
GlycoMimetics Incorporated (Industry)
Overall Status
Terminated
CT.gov ID
NCT02433158
Collaborator
(none)
154
Enrollment
97
Locations
2
Arms
47
Actual Duration (Months)
1.6
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 3

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 Design

Study Type:
Interventional
Actual Enrollment :
154 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
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
Actual Study Start Date :
Dec 16, 2015
Actual Primary Completion Date :
Nov 15, 2019
Actual Study Completion Date :
Nov 15, 2019

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study. [18 months]

      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.

    2. Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study. [18 months]

      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.

    3. Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study [18 months]

      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.

    4. Number (%) of subjects with serious adverse events (SAEs) over the study. [18 months]

      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.

    Secondary Outcome Measures

    1. Subject re hospitalization [18 months]

      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.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    6 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of South Alabama Women's and Children's Hospital Mobile Alabama United States 36604
    2 Arkansas Children's Hospital Research Pharmacy Little Rock Arkansas United States 72202
    3 Arkansas Children's Hospital Little Rock Arkansas United States 72202
    4 UC Davis Medical Center Main Hospital Sacramento California United States 95817
    5 University of California Davis Medical Center Sacramento California United States 95817
    6 Howard University Center for Sickle Cell disease Washington District of Columbia United States 20001
    7 MedStar Health Research Institute Washington District of Columbia United States 20010
    8 Howard University Hospital Washington District of Columbia United States 20060
    9 Golisano Childrens Hospital of Southwest Florida Fort Myers Florida United States 33908
    10 Jackson Memorial Hospital Miami Florida United States 33136
    11 University of Miami Miami Florida United States 33136
    12 St. Mary's Medical Center West Palm Beach Florida United States 33407
    13 Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center: Atlanta Georgia United States 30303
    14 Grady Health System Atlanta Georgia United States 30303
    15 Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center: Atlanta Georgia United States 30322
    16 Emory Children's Center Atlanta Georgia United States 30322
    17 Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center/ Atlanta Georgia United States 30342
    18 Children's Healthcare of Atlanta: Scottish Rite Campus Atlanta Georgia United States 30342
    19 Memorial Family Medicine Center Savannah Georgia United States 31404
    20 Memorial Health University Medical Center Savannah Georgia United States 31404
    21 The University of Chicago/Comer Children's Hospital Chicago Illinois United States 60637
    22 University of Chicago, Investigational Drug Service Pharmacy Chicago Illinois United States 60637
    23 University of Maryland Medical System Investigational Pharmacy Baltimore Maryland United States 21201
    24 University of Maryland Medical System Baltimore Maryland United States 21201
    25 Johns Hopkins Department of Medicine Clinical Trials Unit Baltimore Maryland United States 21205
    26 The Johns Hopkins University School of Medicine Baltimore Maryland United States 21205
    27 The Johns Hopkins Hospital Department of Pharmacy Services Baltimore Maryland United States 21287-6180
    28 Johns Hopkins Medicine Baltimore Maryland United States 21287
    29 Boston Children's Hospital Boston Massachusetts United States 02115
    30 Brigham and Women's Hospital Boston Massachusetts United States 02115
    31 Center for Clinical Investigation, Brigham and Women's Hospital Boston Massachusetts United States 02115
    32 Investigational Drug Services Boston Massachusetts United States 02115
    33 Children's Hospital of Michigan Detroit Michigan United States 48201
    34 University of Mississippi Medical Center - Outpatient Clinical Research Unit Jackson Mississippi United States 39216
    35 University of Mississippi Medical Center Jackson Mississippi United States 39216
    36 Center for Outpatient Health Saint Louis Missouri United States 63108
    37 Barnes-Jewish Hospital Department of Pharmacy Saint Louis Missouri United States 63110
    38 Barnes-Jewish Hospital Saint Louis Missouri United States 63110
    39 Center for Advanced Medicine Saint Louis Missouri United States 63110
    40 Washington University School of Medicine Saint Louis Missouri United States 63110
    41 Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital New Brunswick New Jersey United States 08901
    42 Rutgers-Robert Wood Johnson Medical School New Brunswick New Jersey United States 08901
    43 Rutgers Cancer Institute of New Jersey New Brunswick New Jersey United States 08903
    44 Jacobi Medical Center Bronx New York United States 10461
    45 Kings County Hospital Center Brooklyn New York United States 11203
    46 State University of New York (SUNY) Downstate Medical Center Brooklyn New York United States 11203
    47 SUNY Downstate Medical Center University Hospital of Brooklyn Brooklyn New York United States 11203
    48 Columbia University Medical Center Research Pharmacy New York New York United States 10032
    49 MS CHONY Pediatric Emergency Department New York New York United States 10032
    50 MS CHONY Pediatric Hematology/Oncology Unit New York New York United States 10032
    51 Duke University Hospital, Investigational Drug Service Durham North Carolina United States 27710
    52 Duke University Medical Center Durham North Carolina United States 27710
    53 East Carolina University Brody School of Medicine Greenville North Carolina United States 27834
    54 East Carolina University, Brody School of Medicine Greenville North Carolina United States 27834
    55 Leo W. Jenkins Cancer Center Greenville North Carolina United States 27834
    56 Vidant Medical Center Greenville North Carolina United States 27834
    57 University of Cincinnati - Hoxworth Building Cincinnati Ohio United States 45219
    58 University of Cincinnati Medical Center / Investigational Pharmacy Cincinnati Ohio United States 45219
    59 University of Cincinnati Medical Center / Research Office Cincinnati Ohio United States 45219
    60 University of Cincinnati Medical Center Cincinnati Ohio United States 45219
    61 University of Cincinnati Physicians Company LLC Cincinnati Ohio United States 45219
    62 UC Health Ridgeway Hospital Cincinnati Ohio United States 45229
    63 The Ohio State University Investigational Drug Services Columbus Ohio United States 43203
    64 The Ohio State University Wexner Center East Columbus Ohio United States 43203
    65 The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute Columbus Ohio United States 43210
    66 The Ohio State University Wexner Medical Center Columbus Ohio United States 43210
    67 UPMC Presbyterian Pittsburgh Pennsylvania United States 15213
    68 UPMC Hillman Cancer Center Pittsburgh Pennsylvania United States 15232
    69 Hasbro Children's Hospital Providence Rhode Island United States 02903
    70 Rhode Island Hospital-Pharmacy Service Providence Rhode Island United States 02903
    71 Rhode Island Hospital Providence Rhode Island United States 02903
    72 The Miriam Hospital Providence Rhode Island United States 02906
    73 Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center Charleston South Carolina United States 29425
    74 Medical University of South Carolina-Hospital Charleston South Carolina United States 29425
    75 Medical University of South Carolina Charleston South Carolina United States 29425
    76 MUSC Investigational Drug Services Charleston South Carolina United States 29425
    77 Cook Children's Hematology and Oncology Center Fort Worth Texas United States 76104
    78 Cook Children's Medical Center Fort Worth Texas United States 76104
    79 Cook Children's Hematology and Oncology Center-Grapevine Grapevine Texas United States 76051
    80 University of Texas Medical School Houston Texas United States 77030
    81 Primary Children's Hospital Laboratory Salt Lake City Utah United States 84113
    82 Primary Children's Hospital Salt Lake City Utah United States 84113
    83 Main Hospital-VCU Richmond Virginia United States 23298
    84 Virginia Commonwealth University - Investigational Drug Services Richmond Virginia United States 23298
    85 Virginia Commonwealth University- Clinical Research Services Unit Richmond Virginia United States 23298
    86 Royal Alexandra Hospital Edmonton Alberta Canada T5H 3V9
    87 Miseracordia Community Hospital Edmonton Alberta Canada T5R 4H5
    88 Kaye Edmonton Clinic 3C Edmonton Alberta Canada T6G 1Z1
    89 University of Alberta Hospital, Pharmacy Services Edmonton Alberta Canada T6G 1Z1
    90 Stollery Children's Hospital Edmonton Alberta Canada T6G 2B7
    91 University of Alberta Hospital Edmonton Alberta Canada T6G 2B7
    92 Research transition Facility Edmonton Alberta Canada T6G 2V2
    93 Grey Nuns Community Hospital Edmonton Alberta Canada T6L 5X8
    94 Children's Hospital of Eastern Ontario Ottawa Ontario Canada K1H 8L1
    95 The Hospital for Sick Children Toronto Ontario Canada M5G 1X8
    96 Centre Hospitalier Universitaire Sainte-Justine Montreal Quebec Canada H3T 1C5
    97 The Montreal Children's Hospital / McGill University Health Centre Montreal Quebec Canada H4A3J1

    Sponsors and Collaborators

    • GlycoMimetics Incorporated

    Investigators

    • Study Director: Pfizer CT.gov Call Center, Pfizer

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    GlycoMimetics Incorporated
    ClinicalTrials.gov Identifier:
    NCT02433158
    Other Study ID Numbers:
    • B5201003
    First Posted:
    May 4, 2015
    Last Update Posted:
    Feb 10, 2021
    Last Verified:
    Feb 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by GlycoMimetics Incorporated
    Rivipansel
    GMI-1070
    Selectin Inhibitor
    SCD
    VOC
    Additional relevant MeSH terms:
    Anemia, Sickle Cell

    Study Results

    No Results Posted as of Feb 10, 2021