B5201002: 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
Study ID: STU 072014-085
This Phase 3 randomized, double blind, placebo controlled, parallel group, multicenter study is designed to demonstrate the efficacy of rivipansel in treating subjects with SCD who are at least 6 years in age experiencing an acute vaso occlusive crisis (VoC) event necessitating hospitalization. This study has two cohorts: cohort 1 includes one adult stratum ([GreaterThanorequalTo]18 years old) and one pediatric stratum (12-17 years old), cohort 2 includes one pediatric stratum (6-11 years old). These two cohorts will be analyzed separately. Subjects with acute VoC who complete the screening assessments and meet all eligibility criteria are enrolled into the study and will be randomized in a 1:1 ratio to receive multiple iV doses of rivipansel or placebo for the treatment of VoC. Randomization will be stratified by age (6-11, 12-17, and [Greater Than]/[?]18 years of age) and genotype (category 1: HbSS, HbSB0 thalassemia and HbSD; category 2: HbSC, HbSB+ thalassemia and HbS-Variant [other than HbSD]). Dosing of eligible subjects with acute VoC will occur as early as possible, but no later than 24 hours from the administration of the first dose of iV opioids. For subjects aged 12 and over who weigh [Greater Than]40 kg, study drug will be administered as a loading dose of 1680 mg followed by a maintenance dose of 840 mg every 12+/- 2 hours. Subjects aged 6 to 11 years, or any subject who weighs less than or equal to 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) every 12+/- 2 hours. it is recommended that study drug be administered until criterion for readiness for discharge criteria has been met, or up to 15 doses of study drug (1 loading dose and a maximum of 14 maintenance doses), whichever comes first. Subjects will have daily physical exams and pain assessments. Standard care labs as well as some additional chemistries will be obtained at various time points. Pharmacokinetics and biomarker samples (blood) will be obtained at scheduled time points according to randomization assignment. Subjects will receive phone calls from the study coordinator at day 7 from discharge and day 21 from discharge. Subjects will return to clinic 35 days after discharge for a follow up visit with a Pe, labs and ae assessment. Subjects will be asked to complete additional pain assessments during their inpatient stay on electronic Patient Reported outcome devices. They will be trained on how to use the device by the study coordinator. after discharge from the hospital, a 3rd party vendor will obtain copies of the hospital bill for the services associated with the subject's stay. The inormation will be used to help describe how the study drug might change hospital stays and costs. Please see attached sheet under supporting documents for the basic logistics for how this will happen.
1. Greater than or equal to 6 years of age. 2. Documented diagnosis of sickle cell disease (HbSS, HbS-B thalassemia [HbSB0 and HbSB+thalassemia], HbSC or HbS Variant, including HbSD and HbSE). 3. Diagnosis of VOC at the time of enrollment; necessitating admission to the hospital with treatment including IV opioids. 4.Able to receive the first dose of study drug within 24 hours from the administration of the first dose of IV opioids (Subjects treated as an outpatient within the past 48 hours for the same VOC episode may be enrolled if dosing with study drug is expected within 24 hours from the administration of the first dose of IV opioids for this admitting presentation). 5.Evidence of a personally signed and dated informed consent (and assent, where applicable) document indicating that the subject (or a legally acceptable representative/parent(s)/legal guardian) has been informed of all pertinent aspects of the study. 6. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. 7. Male and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception throughout the duration of the active treatment period and for at least 28 days after the last dose of assigned treatment. Female subjects who are not of childbearing potential (ie, meet at least one of the following criteria): * Have undergone a documented hysterectomy and/or bilateral oophorectomy; * Have medically confirmed ovarian failure; or * Achieved post menopausal status, defined as cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause and have a serum follicle stimulating hormone (FSH) level within the laboratory[Single Quote]s reference range for postmenopausal women. If FSH result is not available by the end of the screening period the decision will be based on the investigator[Single Quote]s judgment and subject[Single Quote]s medical history. All other female subjects (including females with tubal ligations and females that do NOT have a documented hysterectomy, bilateral oophorectomy and/or ovarian failure) will be considered to be of childbearing potential.
- Cancer Related
- Healthy Volunteers
- UT Southwestern Principal Investigator
- Zora R Rogers