CompletedPhase 1

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).

United States, Brazil, France30 participantsStarted 2022-03-26

Plain-language summary

The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.

Who can participate

Age range

12 Years – 55 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Body weight \>=40 kg. * Confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia). * Vaccination against Neisseria Meningitidis serotypes A, C, W, and Y. * Vaccinations against H. influenzae type B and S. pneumoniae. * Participants vaccinated against SARS-CoV-2 are eligible, as long as it has been 3 days or more after inoculation with the vaccine. * Diagnosis of an acute uncomplicated VOE, that requires admission to a hospital/acute medical facility and treatment with parenteral opioid analgesics. * Adequate hepatic and renal function. * Hemoglobin \>=5 grams/deciliter (g/dL) * Platelet count \>=100,000/microliter (µL) * Participants receiving SCD-directed therapies must be on a stable dose for \>=28 days. * For female participants of childbearing potential, an agreement to remain abstinent or use contraception for 322 days (approximately 10.5 months) after the dose of study treatment. Exclusion Criteria: * More than 10 VOEs within the last 12 months prior to presentation, that have required a medical facility visit. * Pain related to the current VOE ongoing for \>36 hours. * Acute pain related to avascular necrosis, hepatic or splenic sequestration, or priapism. * Pain atypical of an acute uncomplicated VOE. * Evidence of or suspicion of ACS. * Evidence or high suspicion of a severe systemic infection. * Major surgery and/or hospitalization for any reason within 30 days. * History of…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of Participants With Adverse Events (AEs)

Timeframe: Baseline up to Day 84

Percentage of Participants with Infusion-Related Reactions and Hypersensitivity

Timeframe: Baseline up to Day 84

Trial details

NCT IDNCT04912869

SponsorHoffmann-La Roche

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-23

View on ClinicalTrials.gov More Sickle Cell Disease trials