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

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ClinicalTrials.gov Identifier: NCT02433158
Recruitment Status : Terminated (The B5201003 OLE study was terminated based on the failure of the 'parent" B5201002 study to meet its efficacy endpoints. Termination was not due to safety.)
First Posted : May 4, 2015
Last Update Posted : February 10, 2021
Sponsor:
Information provided by (Responsible Party):
GlycoMimetics Incorporated

Study Description
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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
Sickle Cell Anemia Sickle Cell Disease Sickle Cell Disorders Pain Crisis Vaso-occlusive Crisis Drug: Rivipansel 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
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Study Type : Interventional  (Clinical Trial)
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 : December 16, 2015
Actual Primary Completion Date : November 15, 2019
Actual Study Completion Date : November 15, 2019

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Arms and Interventions
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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 Name: 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 Name: GMI-1070


Outcome Measures
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Primary Outcome Measures :
  1. Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study. [ Time Frame: 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. [ Time Frame: 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 [ Time Frame: 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. [ Time Frame: 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 [ Time Frame: 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
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02433158


Locations
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Sponsors and Collaborators
GlycoMimetics Incorporated
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
More Information
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Responsible Party: GlycoMimetics Incorporated
ClinicalTrials.gov Identifier: NCT02433158    
Other Study ID Numbers: B5201003
First Posted: May 4, 2015    Key Record Dates
Last Update Posted: February 10, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn