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)

ID#: NCT05075824

Age: 12 - 55 years

Gender: All

Healthy Subjects: No

Study Phase: Phase 2

Recruitment Status: Recruiting

Start Date: March 09, 2022

End Date: January 10, 2025

Contact Information:
Reference Study ID Number
888-662-6728 (U.S. and Canada)
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.
Eligibility:

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 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.

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 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. meningitidis infection within the prior 6 months.