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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02433158
• Other study ID #: B5201003
• Status: Terminated
• Phase: Phase 3
• Start date: December 16, 2015
• Est. completion date: November 15, 2019

Study information

• Verified date: February 2021
• Source: GlycoMimetics Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 154
• Est. completion date: November 15, 2019
• Est. primary completion date: November 15, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Pain Crisis
• Sickle Cell Anemia
• Sickle Cell Disease
• Sickle Cell Disorders
• Vaso-occlusive Crisis

Intervention

Drug:
• Rivipansel
Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses.

Locations

Country	Name	City	State
Canada	Grey Nuns Community Hospital	Edmonton	Alberta
Canada	Kaye Edmonton Clinic 3C	Edmonton	Alberta
Canada	Miseracordia Community Hospital	Edmonton	Alberta
Canada	Research transition Facility	Edmonton	Alberta
Canada	Royal Alexandra Hospital	Edmonton	Alberta
Canada	Stollery Children's Hospital	Edmonton	Alberta
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	University of Alberta Hospital, Pharmacy Services	Edmonton	Alberta
Canada	Centre Hospitalier Universitaire Sainte-Justine	Montreal	Quebec
Canada	The Montreal Children's Hospital / McGill University Health Centre	Montreal	Quebec
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center/	Atlanta	Georgia
United States	Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:	Atlanta	Georgia
United States	Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:	Atlanta	Georgia
United States	Children's Healthcare of Atlanta: Scottish Rite Campus	Atlanta	Georgia
United States	Emory Children's Center	Atlanta	Georgia
United States	Grady Health System	Atlanta	Georgia
United States	Johns Hopkins Department of Medicine Clinical Trials Unit	Baltimore	Maryland
United States	Johns Hopkins Medicine	Baltimore	Maryland
United States	The Johns Hopkins Hospital Department of Pharmacy Services	Baltimore	Maryland
United States	The Johns Hopkins University School of Medicine	Baltimore	Maryland
United States	University of Maryland Medical System	Baltimore	Maryland
United States	University of Maryland Medical System Investigational Pharmacy	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Center for Clinical Investigation, Brigham and Women's Hospital	Boston	Massachusetts
United States	Investigational Drug Services	Boston	Massachusetts
United States	Jacobi Medical Center	Bronx	New York
United States	Kings County Hospital Center	Brooklyn	New York
United States	State University of New York (SUNY) Downstate Medical Center	Brooklyn	New York
United States	SUNY Downstate Medical Center University Hospital of Brooklyn	Brooklyn	New York
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Medical University of South Carolina Lifespan Comprehensive Sickle Cell Center	Charleston	South Carolina
United States	Medical University of South Carolina-Hospital	Charleston	South Carolina
United States	MUSC Investigational Drug Services	Charleston	South Carolina
United States	The University of Chicago/Comer Children's Hospital	Chicago	Illinois
United States	University of Chicago, Investigational Drug Service Pharmacy	Chicago	Illinois
United States	UC Health Ridgeway Hospital	Cincinnati	Ohio
United States	University of Cincinnati - Hoxworth Building	Cincinnati	Ohio
United States	University of Cincinnati Medical Center	Cincinnati	Ohio
United States	University of Cincinnati Medical Center / Investigational Pharmacy	Cincinnati	Ohio
United States	University of Cincinnati Medical Center / Research Office	Cincinnati	Ohio
United States	University of Cincinnati Physicians Company LLC	Cincinnati	Ohio
United States	The Ohio State University Investigational Drug Services	Columbus	Ohio
United States	The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute	Columbus	Ohio
United States	The Ohio State University Wexner Center East	Columbus	Ohio
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Children's Hospital of Michigan	Detroit	Michigan
United States	Duke University Hospital, Investigational Drug Service	Durham	North Carolina
United States	Duke University Medical Center	Durham	North Carolina
United States	Golisano Childrens Hospital of Southwest Florida	Fort Myers	Florida
United States	Cook Children's Hematology and Oncology Center	Fort Worth	Texas
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Cook Children's Hematology and Oncology Center-Grapevine	Grapevine	Texas
United States	East Carolina University Brody School of Medicine	Greenville	North Carolina
United States	East Carolina University, Brody School of Medicine	Greenville	North Carolina
United States	Leo W. Jenkins Cancer Center	Greenville	North Carolina
United States	Vidant Medical Center	Greenville	North Carolina
United States	University of Texas Medical School	Houston	Texas
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	University of Mississippi Medical Center - Outpatient Clinical Research Unit	Jackson	Mississippi
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Arkansas Children's Hospital Research Pharmacy	Little Rock	Arkansas
United States	Jackson Memorial Hospital	Miami	Florida
United States	University of Miami	Miami	Florida
United States	University of South Alabama Women's and Children's Hospital	Mobile	Alabama
United States	Bristol Myers Squibb Children's Hospital at Robert Wood Johnson University Hospital	New Brunswick	New Jersey
United States	Rutgers Cancer Institute of New Jersey	New Brunswick	New Jersey
United States	Rutgers-Robert Wood Johnson Medical School	New Brunswick	New Jersey
United States	Columbia University Medical Center Research Pharmacy	New York	New York
United States	MS CHONY Pediatric Emergency Department	New York	New York
United States	MS CHONY Pediatric Hematology/Oncology Unit	New York	New York
United States	UPMC Hillman Cancer Center	Pittsburgh	Pennsylvania
United States	UPMC Presbyterian	Pittsburgh	Pennsylvania
United States	Hasbro Children's Hospital	Providence	Rhode Island
United States	Rhode Island Hospital	Providence	Rhode Island
United States	Rhode Island Hospital-Pharmacy Service	Providence	Rhode Island
United States	The Miriam Hospital	Providence	Rhode Island
United States	Main Hospital-VCU	Richmond	Virginia
United States	Virginia Commonwealth University - Investigational Drug Services	Richmond	Virginia
United States	Virginia Commonwealth University- Clinical Research Services Unit	Richmond	Virginia
United States	UC Davis Medical Center Main Hospital	Sacramento	California
United States	University of California Davis Medical Center	Sacramento	California
United States	Barnes-Jewish Hospital	Saint Louis	Missouri
United States	Barnes-Jewish Hospital Department of Pharmacy	Saint Louis	Missouri
United States	Center for Advanced Medicine	Saint Louis	Missouri
United States	Center for Outpatient Health	Saint Louis	Missouri
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	Primary Children's Hospital	Salt Lake City	Utah
United States	Primary Children's Hospital Laboratory	Salt Lake City	Utah
United States	Memorial Family Medicine Center	Savannah	Georgia
United States	Memorial Health University Medical Center	Savannah	Georgia
United States	Howard University Center for Sickle Cell disease	Washington	District of Columbia
United States	Howard University Hospital	Washington	District of Columbia
United States	MedStar Health Research Institute	Washington	District of Columbia
United States	St. Mary's Medical Center	West Palm Beach	Florida

Sponsors (1)

Lead Sponsor	Collaborator
GlycoMimetics Incorporated

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study.	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.	18 months
Primary	Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study.	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.	18 months
Primary	Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study	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.	18 months
Primary	Number (%) of subjects with serious adverse events (SAEs) over the study.	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.	18 months
Secondary	Subject re hospitalization	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.	18 months