Sickle cell disease
Conditions
Interventions
Study product:
CSL889 is a human 60 kilodalton glycoprotein that is purified from human plasma
using a Kistler Cohn Fraction IV-4 as the starting material for the
manufacturing process. CSL889 will
Sponsors
CSL Behring LLC
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: • Diagnosis of SCD as documented in the subject's medical record • Aged 18 to 60 years, inclusive • Stable SCD for at least 30 days before Day 1. Stable SCD is defined as the subject being at his or her medical baseline, with no evidence of worsening of disease over the last 30 days (including VOC, recent major surgery, hospitalization, serious infection, significant bleeding, cerebrovascular accident, seizures, or IV opioids)(Part A) • Uncomplicated VOC requiring parenteral opioid treatment and admission to hospital for management Uncomplicated VOC is defined as sickle cell pain without the following associated clinical features (Part B): o Fever (> 38.5 °C) o Hypotension (
Exclusion criteria
Exclusion criteria: • History of primary hemorrhagic stroke • History or evidence of inherited bleeding diathesis or significant coagulopathy at risk for bleeding • Hospitalization for vaso-occlusive crisis (VOC) or treated with parenteral pain medications in other medical settings such as the emergency department or day hospital for VOC during the past 30 days before Day 1 • Blood transfusion within the 90 days before Day 1, or expecting blood transfusion during the study • Weight >110 kg (242 lbs) • Surgery within 30 days before Day 1 or any preplanned surgeries during the study (minor surgeries may be permitted under local anesthesia before screening, with permission of the medical monitor) • Female subjects who are pregnant or breastfeeding • Female subject of childbearing potential or fertile male subject either not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of CSL889. • Treatment with any other drug / biologic that is newly approved for SCD during the conduct of this study within 90 days before Day 1. Exceptions: crizanlizumab [Adakveo®] and voxelotor [Oxbryta®] are permitted (where prescribed). • Treatment with another investigational product within 30 days or within 5 half-lives of the product (whichever is greater) before Day 1 • Vaccination within 30 days before Day 1, or planned vaccination during the study • Body-mass index < 16 kg/m2 or weight < 50 kg (110 lbs) • History of anaphylactic-type reactions, transfusion related reaction, asthma, or autoimmune disease
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: 1. Serum PK parameters of CSL889, with and without adjustment for baseline hemopexin: Cmax, AUC from time 0 to the last measurable concentration (AUC0-last), AUC0-inf, time of maximum concentration (tmax), terminal half-life (t1/2), clearance (CL), volume of distribution during the elimination phase (Vz) 2. Anti-CSL889 antibodies | — |
Primary
| Measure | Time frame |
|---|---|
| Primary Endpoints: Frequency, nature and severity of TEAEs from start of infusion until 32 days after infusion in subjects with stable SCD and in subjects with SCD in VOC | — |
Countries
Netherlands
Outcome results
None listed