A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)

• Status: Not yet recruiting
• Trial end date: 2025-01-10
• Target enrollment: 0
• Participant gender: All

Summary

This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche

Criteria

Inclusion Criteria:

• Body weight >=40 kg.

• Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia).

• Two or more (>=2) to <=10 documented VOEs in the 12 months prior to randomisation.

• If receiving concurrent SCD-directed therapy, the participant must have been on a stable dose for a minimum of 3 months prior to study enrollment. There should be no plans to modify the participants' dosing throughout the study duration, other than for safety reasons.

• If receiving erythropoietin, the participant must have been prescribed this medication for the preceding 3 months and be dose-stabilised for at least 3 months prior to study enrollment.

• Vaccination against N. meningitides and Vaccinations against H. influenza type B and S. pneumonia.

• Participants who have been vaccinated (partially or in full) against SARS-CoV-2 with a locally approved vaccine are eligible to be enrolled in the study, 3 days or longer after inoculation.

• Adequate hepatic and renal function.

• For women of childbearing potential: agreement to remain abstinent or use contraception during the treatment period and for 6 months after the final dose of study treatment.

Exclusion Criteria:

• History of hematopoietic stem cell transplant.

• Participating in a chronic transfusion program and/or planning on undergoing an exchange transfusion during the duration of the study.

• History of hypersensitivity, allergic, or anaphylactic reactions to any ingredient contained in the study treatment.

• Received active treatment on another investigational trial within 28 days (or within five half-lives of that agent, whichever is greater) prior to screening visit, or plans to participate in another investigational drug trial.

• Hemoglobin <6 g/dL.

• Known or suspected hereditary complement deficiency.

• Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration.

• Presence of fever (>=38 degrees Celsius) within 7 days before the first drug administration.

• Immunised with a live attenuated vaccine within 1 month before first drug administration.

• Pregnant or breastfeeding, or intending to become pregnant during the study or within 6 months after the final dose of study treatment.

• Known HIV infection with documented CD4 count <200 cells/microliter within 24 weeks prior to screening.

• History of N. meningitidis infection within the prior 6 months.