Pain Crisis, Sickle Cell Anemia, Sickle Cell Disease, Sickle Cell Disorders, Vaso-occlusive Crisis
Conditions
Keywords
Rivipansel, GMI-1070, Selectin Inhibitor, SCD, VOC
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.
Interventions
DRUGRivipansel
Rivipansel will be infused intravenously every 12 hours up to a maximum of 15 doses.
Sponsors
GlycoMimetics Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Canada, United States
Outcome results
None listed