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

ID: NCT02187003
Status: Recruiting
Phase: Phase 3
Start Date: June 01, 2015
First Submitted: June 12, 2014
Last Updated: February 22, 2018
Results: N/A
Organization: Pfizer
Sponsors & Collaborators: Pfizer
Location: Canada, United States
Conditions: Anemia, Sickle Cell
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Study Description

Brief 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.

Detailed Description

Condition or disease Intervention/treatment Phase

Anemia, Sickle Cell

Drug: Rivipansel
Other Names
GMI-1070
Other: Placebo
Other Names
phosphate buffered saline
Phase 3

Tracking Information

First Submitted DateJune 12, 2014
Last Update Posted DateFebruary 22, 2018
Actual Start DateJune 01, 2015
Anticipated Completion DateNovember 01, 2018
Actual Primary Completion DateNovember 01, 2018
Results First Submitted DateN/A
Received Results Disposit DateN/A

Current Primary Outcome Measures

  • 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.

Original Primary Outcome Measures

Not Available

Current Secondary Outcome Measures

  • 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.

  • 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.

  • 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.

  • 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.

  • 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.

Original Secondary Outcome Measures

Not Available

Study Design

Brief TitleEfficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease
Official TitleA 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
Brief 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.

Detailed Description

Study TypeInterventional
Study PhasePhase 3
Estimated Enrollment
350
Allocation
Randomized
Interventional Model
Parallel Assignment
Masking
Quadruple
Primary Purpose
Treatment
Conditions
Anemia, Sickle Cell
Target Follow-Up Duration N/A
Biospecimen:
N/A
Sampling MethodN/A
Study PopulationN/A
Intervention
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
Other: Placebo

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

Other Names
phosphate buffered saline
Study Groups/Cohorts
Rivipansel Treatment Arm

Placebo Treatment Arm

Study Arms
Placebo Comparator Placebo Treatment Arm
Other : Placebo
Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.

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.

Arm Intervention/Treatment
Placebo Comparator Placebo Treatment Arm
Other : Placebo
Experimental Rivipansel Treatment Arm
Drug : Rivipansel

Recruitment Information

Recruitment Status:Recruiting
Enrollment350
Completion DateNovember 01, 2018
Eligibility Criteria: 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
GenderAll
Age6 Years to N/A
Accepts Healthy VolunteersNo
Contacts
Listed Location Countries
Canada
United States

Administrative Information

NCT Number:NCT02187003
Other Study ID Numbers
B5201002
Has Data Monitoring CommitteeYes
U.S. FDA-regulated Product Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Device Product Not Approved or Cleared by U.S. FDA: No
IPD Sharing Statement
Not Available
Responsible Party,
Study Sponsor
Pfizer
Collaborators
Not Available
Investigators
Study Director
Pfizer CT.gov Call Center
Pfizer