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

• Sponsor: Pfizer
• Information provided by (Responsible Party): Pfizer

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.

Condition or disease	Intervention/treatment	Phase
Anemia, Sickle Cell	Drug: Rivipansel; Other: Placebo	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	341 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
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
Actual Study Start Date :	June 17, 2015
Estimated Primary Completion Date :	June 13, 2019
Estimated Study Completion Date :	June 13, 2019

Arms and Interventions

Arm	Intervention/treatment
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.; Other Name: GMI-1070
Placebo Comparator: Placebo Treatment Arm	Other: Placebo; Placebo (phosphate buffered saline) will be infused intravenously every 12 hours up to 15 doses maximum.; Other Name: phosphate buffered saline

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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.
2. 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.
3. 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.
4. 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.
5. 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.

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:

• 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

Contacts and Locations

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Sponsors and Collaborators

Pfizer

Investigators

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Study Director:	Pfizer CT.gov Call Center	Pfizer

More Information

Additional Information:

To obtain contact information for a study center near you, click here. 

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Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT02187003 History of Changes
Other Study ID Numbers:	B5201002; RESET ( Other Identifier: Alias Study Number )
First Posted:	July 10, 2014
Last Update Posted:	June 17, 2019
Last Verified:	June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Yes
Plan Description:	Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
URL:	https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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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 Pfizer:

Sickle Cell Anemia; Sickle Cell Disease; Sickle Cell Disorders; pain crisis; vaso-occlusive crisis	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