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

Phase 3

Completed

• Conditions: Anemia, Sickle Cell
• Interventions: Drug: Rivipansel; Other: Placebo

• Registration Number: NCT02187003
• Lead Sponsor: Biossil Inc.

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

Not available

Study Timeline

• Study First Submitted: 2014-06-12
• Study First Submitted QC: 2014-07-07
• Study First Posted: 2014-07-10
• Study Start: 2015-06-17
• Primary Completion: 2019-05-03
• Study Completion: 2019-06-27
• Disposition First Submitted: 2020-05-05
• Disposition First Submitted QC: 2020-05-07
• Disposition First Posted: 2020-05-13
• Results First Submitted: 2024-02-26
• Status Verified: 2025-02
• Results First Submitted QC: 2025-02-18
• Last Update Submitted: 2025-02-18
• Results First Posted: 2025-03-11
• Last Update Posted: 2025-03-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 345

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Rivipansel Treatment Arm	Rivipansel	-
Placebo Treatment Arm	Placebo	-

Primary Outcome Measures

Name	Time	Method
Time to Readiness for Discharge From Hospital	Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8)	Time to readiness-for-discharge from hospital was defined as the difference (in hours) between the time and date when all criteria for readiness-for-discharge were met and the start time and date of the first infusion (loading dose) of study drug. Criteria for readiness-for-discharge were met when all of the applicable 6 criteria (in relation to treatment of VOC and complications related to the VOC) were documented to have occurred. The six criteria were: 1) only oral pain medication was required, 2) acute complications related to the VOC (such as acute chest syndrome, stroke, priapism) had resolved to the extent that management could be in an outpatient setting, 3) IV opioids had been discontinued, 4) IV hydration had been discontinued, 5) IV antibiotics had been discontinued and 6) red blood cell (RBC) transfusion was no longer required for treatment of this VOC.

Secondary Outcome Measures

Name	Time	Method
Time to Discharge From Hospital	Day 1 up to the latest day when the order of hospital discharge was issued by a qualified healthcare provider (up to an average of Day 8)	Time to discharge from hospital was defined as the difference (in hours) between the time and date of the hospital discharge order from a qualified healthcare provider and the start time and date of the first infusion (loading dose) of study drug.
Cumulative Intravenous (IV) Opioids Consumption From Time of Loading Dose of Study Drug to Discharge From Hospital	Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)	Cumulative IV opioid consumption was reported as cumulative IV opioid use (standardized using morphine equivalent units \[MEU\]), from the start of the first infusion (loading dose) of study drug until hospital discharge.
Time to Discontinuation of Intravenous (IV) Opioids	Day 1 up to the latest day when IV opioid was discontinued (up to an average of Day 8)	Time to discontinuation of IV opioids was defined as the difference (in hours) between the stop time and date of the latest IV opioid dose and the start time and date of the first infusion (loading dose) of study drug.
Cumulative Intravenous (IV) Opioids Consumption Within First 24 Hours Post-Loading Dose of Study Drug	24 hours post first IV infusion of loading dose of study drug on Day 1	Cumulative IV opioid consumption (standardized using MEU) was reported as IV opioid use in the first 24 hours from the start time of the first IV infusion (loading dose) of study drug.
Percentage of Participants Re-hospitalized for Vaso-Occlusive Crisis (VOC) Within 3 Days of Discharge From Hospital	Within 3 days of discharge from hospital, where discharge from hospital was any day from Day 1 to an average of Day 8	Percentage of participants who were re-hospitalized for a vaso-occlusive crisis (VOC) within 3 days of discharge from hospital are reported.

Trial Locations

Locations (138)

University of South Alabama Children's and Women's Hospital; 🇺🇸 Mobile, Alabama, United States

Arkansas Children's Hospital Research Pharmacy; 🇺🇸 Little Rock, Arkansas, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

UCSF Benioff Children's Hospital Oakland; 🇺🇸 Oakland, California, United States

UC Davis Medical Center Main Hospital; 🇺🇸 Sacramento, California, United States

UC Davis Medical Center; 🇺🇸 Sacramento, California, United States

University of California Davis Medical Center; 🇺🇸 Sacramento, California, United States

University of Colorado CTRC; 🇺🇸 Aurora, Colorado, United States

University of Colorado Hospital; 🇺🇸 Aurora, Colorado, United States

Howard University Center for Sickle Cell Disease; 🇺🇸 Washington, District of Columbia, United States

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