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).

NCT ID: NCT04912869

Last Updated: 2025-08-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-03-26
• Study Completion Date: 2025-08-15

Brief 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.

Conditions

Sickle Cell Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Crovalimab

Participants will receive a single intravenous (IV) infusion of Crovalimab based on body weight.

Group Type: EXPERIMENTAL

Crovalimab

Intervention Type: DRUG

Crovalimab will be administered as a single dose of 1000 milligrams (mg) IV (for participants with a body weight between 40 kilograms (kg) and 100 kg) or 1500 mg IV (for participants with a body weight \>=100 kg).

Placebo

Participants will receive a single IV infusion of matching Placebo.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo will be administered as a single IV infusion, with an equal volume and over the same duration as weight- based crovalimab

Interventions

Crovalimab

Crovalimab will be administered as a single dose of 1000 milligrams (mg) IV (for participants with a body weight between 40 kilograms (kg) and 100 kg) or 1500 mg IV (for participants with a body weight \>=100 kg).

Intervention Type: DRUG

Placebo

Placebo will be administered as a single IV infusion, with an equal volume and over the same duration as weight- based crovalimab

Intervention Type: DRUG

Eligibility Criteria

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 Neisseria meningitidis infection within 6 months prior.
• Known HIV infection with a documented CD4 count <200 cells/µL.
• Transfusion or receipt of blood products within 3 months or current participation in a chronic transfusion protocol.
• Immunized with a live attenuated vaccine within 30 days.
• History of hematopoietic stem cell transplant.
• Known or suspected hereditary complement deficiency.
• Pregnant or breastfeeding, or intending to become pregnant during the study or within 322 days (approximately 10.5 months) after the study drug administration.
• Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within the prior 28 days or within five half-lives of that investigational product, whichever was greater.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

Children'S Healthcare of Atlanta

Atlanta, Georgia, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Hospital Sao Rafael - HSR

Salvador, Estado de Bahia, Brazil

Hospital das Clinicas - UFRGS

Porto Alegre, Rio Grande do Sul, Brazil

CHU Henri Mondor

Créteil, , France

Azienda Ospedaliera di Verona-Policlinico G.B. Rossi

Verona, Veneto, Italy

International Cancer Institute (ICI)

Eldoret, , Kenya

Gertrude's Children Hospital

Nairobi, , Kenya

American University of Beirut - Medical Center

Beirut, , Lebanon

Hopital Nini

Tripoli, , Lebanon

Amsterdam UMC Location VUMC

Amsterdam, , Netherlands

Charlotte Maxeke Johannesburg Hospital

Johannesburg, , South Africa

Hospital General Univ. Gregorio Maranon

Madrid, , Spain

Hospital Universitario Virgen del Rocio

Seville, , Spain

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Hammersmith Hospital

London, , United Kingdom

Countries

United States; Brazil; France; Italy; Kenya; Lebanon; Netherlands; South Africa; Spain; United Kingdom

Other Identifiers

2020-004840-27

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BO42452

Identifier Type: -

Identifier Source: org_study_id