Sickle cell disease (SCD)
Conditions
Interventions
Product Name: Crovalimab
Product Code: RO7112689/F03-10
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: CROVALIMAB
Current Sponsor code: RO7112689
Other descriptive name: C5
Sponsors
F.Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Signed ICF or Assent Form (as determined by patient’s age and individual site and country standards) • Age >=12 to ==40 kg • Confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSß0 (SCD genotype of sickle cell beta zero thalassemia) • Vaccination against Neisseria meningitidis • Vaccinations against H. influenzae type B and S. pneumoniae • Diagnosis of an acute uncomplicated VOE, that requires admission to a hospital/acute medical facility and treatment with parenteral opioid analgesics • Adequate hepatic and renal function • Hemoglobin >=5 g/dL • Platelet count >=100,000/µL • Patients receiving sickle cell therapies must be on a stable dose for >=28 days • For female patients of childbearing potential, an agreement to remain abstinent or use contraception for 6 months after the dose of study treatment Are the trial subjects under 18? yes Number of subjects for this age range: 8 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 22 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • More than 10 VOEs within the last 12 months prior to presentation, that have required a medical facility visit • Pain related to the current VOE ongoing for >48 hours • Acute pain related to avascular necrosis, hepatic or splenic sequestration, or priapism • Pain atypical of an acute uncomplicated VOE • Evidence of or suspicion of ACS • Evidence or high suspicion of a severe systemic infection • Major surgery and/or hospitalization for any reason within 30 days prior to VOE presentation • History of Neisseria meningitidis infection within 6 months prior • Known HIV infection with a documented CD4 count <200 cells/µL • Transfusion or receipt of blood products within 3 months or current participation in a chronic transfusion protocol • Immunized with a live attenuated vaccine within 30 days • History of hematopoietic stem cell transplant • Known or suspected hereditary complement deficiency • Pregnant or breastfeeding, or intending to become pregnant during the study or within 6 months after the study drug administration • Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within the prior 28 days or within five half-lives of that investigational product, whichever was greater
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To evaluate the safety of crovalimab compared with placebo;Secondary Objective: • To evaluate the pharmacokinetics (PK) of crovalimab • To evaluate the pharmacodynamics (PD) of crovalimab • To evaluate the efficacy of crovalimab compared with placebo • To evaluate the immune response to crovalimab ;Primary end point(s): 1. Incidence and severity of adverse events, with severity determined according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events, Version 5.0 (CTCAE v5.0) 2. Change from baseline in targeted vital signs and clinical laboratory test results 3. Incidence and severity of infusion-related reactions and hypersensitivity ;Timepoint(s) of evaluation of this end point: 1-3. Up to 84 days | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Serum concentrations of crovalimab over time 2. Relationships between drug exposure and pharmacodynamics, efficacy or safety endpoints of crovalimab 3. Change over time in PD markers (CH50, free C5, sC5b-9) 4. Time to improvement of the primary acute uncomplicated VOE from baseline 5. Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study ;Timepoint(s) of evaluation of this end point: 1-3. Baseline to Day 84 4. Baseline to hospital discharge (up to 84 days) 5. Baseline to Day 84 | — |
Countries
Brazil, France, Italy, Netherlands, Spain, United States
Contacts
Public ContactTrial Information Support Line-TISL
F.Hoffmann-La Roche Ltd.
Outcome results
None listed