A Study Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso‑Occlusive Episodes (VOE) in Patients With Sickle Cell Disease (SCD)
- Conditions
- Sickle Cell Disease
- Registration Number
- 2022-502546-26-00
- Lead Sponsor
- F. Hoffmann-La Roche AG
- Brief Summary
To evaluate the safety of crovalimab compared with placebo
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing, recruitment ended
- Sex
- Not specified
- Target Recruitment
- 16
Body weight >=40 kg
Confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia)
Vaccination against Neisseria Meningitidis,, and 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
Participants receiving sickle cell therapies must be on a stable dose for >=28 days
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, and pain atypical of an acute uncomplicated VOE
Transfusion or receipt of blood products within 3 months or current participation in a chronic transfusion protocol
Known or suspected hereditary complement deficiency
Pregnant or breastfeeding, or intending to become pregnant during the study or within 322 days (approximately 10.5 months) after the study drug administration
Study & Design
- Study Type
- Not specified
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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) 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)
Change from baseline in targeted vital signs and clinical laboratory test results Change from baseline in targeted vital signs and clinical laboratory test results
Incidence and severity of infusion-related reactions and hypersensitivity Incidence and severity of infusion-related reactions and hypersensitivity
- Secondary Outcome Measures
Name Time Method Time to improvement of the primary acute uncomplicated VOE from baseline Time to improvement of the primary acute uncomplicated VOE from baseline
Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study
Serum concentrations of crovalimab over time Serum concentrations of crovalimab over time
Relationships between drug exposure and pharmacodynamics, efficacy, or safety endpoints of crovalimab (patients randomized to crovalimab) Relationships between drug exposure and pharmacodynamics, efficacy, or safety endpoints of crovalimab (patients randomized to crovalimab)
Change over time in PD biomarkers (CH50, free C5, sC5b-9) Change over time in PD biomarkers (CH50, free C5, sC5b-9)
Trial Locations
- Locations (7)
Assistance Publique Hopitaux De Paris
🇫🇷Creteil, France
Centre Hospitalier Universitaire De Montpellier
🇫🇷Montpellier Cedex 5, France
Centro Ricerche Cliniche Di Verona S.r.l.
🇮🇹Verona, Italy
Amsterdam UMC
🇳🇱Amsterdam, Netherlands
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
University Hospital Virgen Del Rocio S.L.
🇪🇸Sevilla, Spain
Hospital Unviersitario Miguel Servet
🇪🇸Zaragoza, Spain
Assistance Publique Hopitaux De Paris🇫🇷Creteil, FrancePablo BARTOLUCCISite contact+33149812440pablo.bartolucci@aphp.fr