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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Anemia, Sickle Cell

Intervention / Treatment
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* Drug: Rivipansel
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Rivipansel Treatment Arm<br> | Drug: Rivipansel<br>* 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.<br>* Other names: GMI-1070;|
| Placebo Comparator: Placebo Treatment Arm<br> | Other: Placebo<br>* Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.<br>* Other names: phosphate buffered saline;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to readiness-for-discharge. | 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 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. | 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 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. | 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. | Percent of subjects re-hospitalized for VOC within 3 days of discharge. | Within 3 days of discharge | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SCD, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders, pain crisis, vaso-occlusive crisis, rivipansel, GMI-1070, selectin inhibitor, VOC