Summary

Eligibility
for people ages 12-55 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

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.

Official Title

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)

Keywords

Sickle Cell Disease, Vaso-occlusive episodes, Pain crisis, Sickle Cell Anemia, Crovalimab

Eligibility

You can join if…

Open to people ages 12-55

  • 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
    1. 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.

You CAN'T join if...

  • 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.

Locations

  • UCSF Benioff Children's Hospital Oakland accepting new patients
    Oakland California 94609 United States
  • University of Texas Southwestern Medical Center; Pediatrics accepting new patients
    Dallas Texas 75235 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Hoffmann-La Roche
ID
NCT05075824
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 90 study participants
Last Updated