Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
- Conditions
- Sickle Cell AnemiaSickle Cell DiseaseSickle Cell DisordersPain CrisisVaso-occlusive Crisis
- Interventions
- Registration Number
- NCT02433158
- Lead Sponsor
- GlycoMimetics Incorporated
- Brief 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).
- Detailed 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 & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 154
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cohort 1 Rivipansel Includes one adult stratum (\>18 years old) and one pediatric stratum (12-17 years old). Subjects aged 12 and over who weigh \>40 kg, will receive a loading dose of 1680 mg followed by a maintenance dose of 840 mg. Cohort 2 Rivipansel Includes one pediatric stratum (6-11 years old). Subjects 6 to 11 years of age or subjects who weigh 40 kg, will receive a loading dose of 40 mg/kg (maximum of 1680 mg) followed by a maintenance dose of 20 mg/kg (maximum of 840 mg).
- Primary Outcome Measures
Name Time Method Number (%) of subjects with treatment emergent adverse events (TEAEs) over the study. 18 months 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.
Number (%) of subjects with adjudicated Acute Chest Syndrome (ACS) over the study. 18 months 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.
Number (%) of subjects with adjudicated severe and/or generalized cutaneous manifestations over the study 18 months 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.
Number (%) of subjects with serious adverse events (SAEs) over the study. 18 months 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.
- Secondary Outcome Measures
Name Time Method Subject re hospitalization 18 months 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.
Trial Locations
- Locations (96)
University of Maryland Medical System
🇺🇸Baltimore, Maryland, United States
The Johns Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
Johns Hopkins Medicine
🇺🇸Baltimore, Maryland, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
University of South Alabama Women's and Children's Hospital
🇺🇸Mobile, Alabama, United States
MedStar Health Research Institute
🇺🇸Washington, District of Columbia, United States
Arkansas Children's Hospital Research Pharmacy
🇺🇸Little Rock, Arkansas, United States
Howard University Center for Sickle Cell disease
🇺🇸Washington, District of Columbia, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
University of Maryland Medical System Investigational Pharmacy
🇺🇸Baltimore, Maryland, United States
Johns Hopkins Department of Medicine Clinical Trials Unit
🇺🇸Baltimore, Maryland, United States
Columbia University Medical Center Research Pharmacy
🇺🇸New York, New York, United States
Kings County Hospital Center
🇺🇸Brooklyn, New York, United States
Rhode Island Hospital-Pharmacy Service
🇺🇸Providence, Rhode Island, United States
The Miriam Hospital
🇺🇸Providence, Rhode Island, United States
Emory Children's Center
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center:
🇺🇸Atlanta, Georgia, United States
The University of Chicago/Comer Children's Hospital
🇺🇸Chicago, Illinois, United States
Grady Health System
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta Aflac Cancer and Blood Disorders Center/
🇺🇸Atlanta, Georgia, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
UPMC Presbyterian
🇺🇸Pittsburgh, Pennsylvania, United States
The Ohio State University Investigational Drug Services
🇺🇸Columbus, Ohio, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
University of Texas Medical School
🇺🇸Houston, Texas, United States
Jackson Memorial Hospital
🇺🇸Miami, Florida, United States
University of Miami
🇺🇸Miami, Florida, United States
Duke University Hospital, Investigational Drug Service
🇺🇸Durham, North Carolina, United States
Children's Hospital of Michigan
🇺🇸Detroit, Michigan, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
University of Cincinnati - Hoxworth Building
🇺🇸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
Primary Children's Hospital Laboratory
🇺🇸Salt Lake City, Utah, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Golisano Childrens Hospital of Southwest Florida
🇺🇸Fort Myers, Florida, United States
Howard University Hospital
🇺🇸Washington, District of Columbia, United States
Memorial Family Medicine Center
🇺🇸Savannah, Georgia, United States
Children's Healthcare of Atlanta: Scottish Rite Campus
🇺🇸Atlanta, Georgia, United States
The Johns Hopkins Hospital Department of Pharmacy Services
🇺🇸Baltimore, Maryland, United States
University of Chicago, Investigational Drug Service Pharmacy
🇺🇸Chicago, Illinois, United States
Investigational Drug Services
🇺🇸Boston, Massachusetts, United States
University of Mississippi Medical Center - Outpatient Clinical Research Unit
🇺🇸Jackson, Mississippi, United States
Center for Outpatient Health
🇺🇸Saint Louis, Missouri, United States
Center for Advanced Medicine
🇺🇸Saint Louis, Missouri, United States
Barnes-Jewish Hospital Department of Pharmacy
🇺🇸Saint Louis, Missouri, 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
State University of New York (SUNY) Downstate Medical Center
🇺🇸Brooklyn, 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
East Carolina University Brody School of Medicine
🇺🇸Greenville, North Carolina, United States
East Carolina University, Brody School of Medicine
🇺🇸Greenville, North Carolina, United States
Vidant Medical Center
🇺🇸Greenville, North Carolina, United States
University of Cincinnati Physicians Company LLC
🇺🇸Cincinnati, Ohio, United States
The Ohio State University James Comprehensive Cancer Hospital & Solove Research Institute
🇺🇸Columbus, Ohio, United States
Cook Children's Hematology and Oncology Center
🇺🇸Fort Worth, Texas, United States
Cook Children's Hematology and Oncology Center-Grapevine
🇺🇸Grapevine, Texas, United States
The Montreal Children's Hospital / McGill University Health Centre
🇨🇦Montreal, Quebec, Canada
Centre Hospitalier Universitaire Sainte-Justine
🇨🇦Montreal, Quebec, Canada
Memorial Health University Medical Center
🇺🇸Savannah, Georgia, United States
Rutgers-Robert Wood Johnson Medical School
🇺🇸New Brunswick, New Jersey, United States
St. Mary's Medical Center
🇺🇸West Palm Beach, Florida, United States
Center for Clinical Investigation, Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
University of Cincinnati Medical Center
🇺🇸Cincinnati, Ohio, United States
Barnes-Jewish Hospital
🇺🇸Saint Louis, Missouri, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Leo W. Jenkins Cancer Center
🇺🇸Greenville, North Carolina, United States
The Ohio State University Wexner Center East
🇺🇸Columbus, Ohio, United States
UPMC Hillman Cancer Center
🇺🇸Pittsburgh, Pennsylvania, United States
UC Health Ridgeway Hospital
🇺🇸Cincinnati, Ohio, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
SUNY Downstate Medical Center University Hospital of Brooklyn
🇺🇸Brooklyn, New York, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
UC Davis Medical Center Main Hospital
🇺🇸Sacramento, California, 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
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
MUSC Investigational Drug Services
🇺🇸Charleston, South Carolina, 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
Jacobi Medical Center
🇺🇸Bronx, New York, United States
Royal Alexandra Hospital
🇨🇦Edmonton, Alberta, Canada
Miseracordia Community Hospital
🇨🇦Edmonton, Alberta, Canada
Kaye Edmonton Clinic 3C
🇨🇦Edmonton, Alberta, Canada
University of Alberta Hospital, Pharmacy Services
🇨🇦Edmonton, Alberta, Canada
Stollery Children's Hospital
🇨🇦Edmonton, Alberta, Canada
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Research transition Facility
🇨🇦Edmonton, Alberta, Canada
Grey Nuns Community Hospital
🇨🇦Edmonton, Alberta, Canada
Rhode Island Hospital
🇺🇸Providence, Rhode Island, United States
Hasbro Children's Hospital
🇺🇸Providence, Rhode Island, United States