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)

Phase 1

Recruiting

Conditions: Sickle Cell Disease
MedDRA version: 21.0Level: PTClassification code: 10040644Term: Sickle cell disease Class: 100000004850
MedDRA version: 20.0Level: LLTClassification code: 10072397Term: Vaso-occlusive crisis Class: 10005329
Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

Registration Number: CTIS2022-502546-26-00

Lead Sponsor: F. Hoffmann-La Roche AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 53

Inclusion Criteria

Body weight >=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,, 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

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, 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: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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): 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, Incidence and severity of infusion-related reactions and hypersensitivity

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):Serum concentrations of crovalimab over time;Secondary end point(s):Relationships between drug exposure and pharmacodynamics, efficacy, or safety endpoints of crovalimab (patients randomized to crovalimab);Secondary end point(s):Change over time in PD biomarkers (CH50, free C5, sC5b-9);Secondary end point(s):Time to improvement of the primary acute uncomplicated VOE from baseline;Secondary end point(s):Prevalence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study