A randomized double-blind phase 2A 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)

Roche0 个研究点目标入组 90 人开始时间: 2023年7月24日最近更新: 2024年3月4日

适应症

试验速览

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•Body weight >=40 kg.
•- Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSß0 (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 serotypes A, C, W, and Y 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 10.5 months after the final dose of study treatment.

•- 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 10.5 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. meningitis infection within the prior 6 months.

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