A Randomized Double-Blind Phase IIA 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)

About the study

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.

Study point of contact

Reference Study ID Number: BO42451 https://forpatients.roche.com/
888-662-6728 (U.S. and Canada)
[email protected]

Locations

3 Italy sites

3 Spain sites

2 United States sites

Age

12 Years - 55 Years

Genotypes

HbSS

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

participation requirements

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.

participation restrictions

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.

Locations

  • New York, New York, United States, Icahn School of Medicine at Mount Sinai
  • Greenville, North Carolina, United States, East Carolina University; Brody School of Medicine
  • Napoli, Campania, Italy, Università degli Studi della Campania Luigi Vanvitelli; UOC Ematologia ed oncologia pediatrica
  • Genova, Liguria, Italy, Ospedale Galliera; S.S.D. Ematologia
  • Verona, Veneto, Italy, Azienda Ospedaliera di Verona-Policlinico G.B. Rossi; Medicina Interna
  • Barcelona, Spain, Hospital Universitari Vall d'Hebron; Servicio de Hematologia
  • Madrid, Spain, Hospital General Univ. Gregorio Maranon
  • Sevilla, Spain, Hospital Universitario Virgen del Rocio; Servicio de Hematologia
Last updated 2022-09-19