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)

NCT ID: NCT05075824

Last Updated: 2025-12-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

90 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-09

Study Completion Date

2026-04-08

Brief Summary

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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.

Detailed Description

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Conditions

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Sickle Cell Disease

Keywords

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Vaso-occlusive episodes Pain crisis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Crovalimab

Participants will receive a loading series of Crovalimab comprised of an intravenous (IV) loading dose on Day 1, followed by weekly Crovalimab subcutaneous (SC) doses for 4 weeks on Week 1 Day 2, then on Weeks 2, 3 and 4. Maintenance SC dosing will begin at Week 5 and will continue every 4 weeks (Q4W) thereafter for a total of 48 weeks of treatment.

Group Type EXPERIMENTAL

Crovalimab

Intervention Type DRUG

Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 kg and 100 kg) or 1500 mg IV (for participants with body weight \>= 100 kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, crovalimab will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, crovalimab will be administered at a dose of 680 mg SC (for participants with body weight between 40 kg and 100 kg) or 1020 mg SC (for participants with body weight \>= 100 kg). Dosing schedule will be as per Arm Description.

Placebo

Participants will receive matching Placebo administered by IV infusion and SC injection over the same duration as Crovalimab, for a total of 48 weeks of treatment.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching Placebo will be administered with the same dosing schedule and equivalent IV and SC volume as weight-based Crovalimab.

Interventions

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Crovalimab

Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 kg and 100 kg) or 1500 mg IV (for participants with body weight \>= 100 kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, crovalimab will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, crovalimab will be administered at a dose of 680 mg SC (for participants with body weight between 40 kg and 100 kg) or 1020 mg SC (for participants with body weight \>= 100 kg). Dosing schedule will be as per Arm Description.

Intervention Type DRUG

Placebo

Matching Placebo will be administered with the same dosing schedule and equivalent IV and SC volume as weight-based Crovalimab.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Mississippi Center for Advanced Medicine

Madison, Mississippi, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

East Carolina University

Greenville, North Carolina, United States

Site Status

Hospital Sao Rafael - HSR

Salvador, Estado de Bahia, Brazil

Site Status

Hospital das Clinicas - UFRGS

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

UNESP - Faculdade de Medicina da Universidade Estadual Paulista - Campus Botucatu

Botucatu, São Paulo, Brazil

Site Status

Hospital das Clínicas Faculdades Médicas de Ribeirão Preto

Ribeirão Preto, São Paulo, Brazil

Site Status

Hospital de Base de Sao Jose do Rio Preto

São José do Rio Preto, São Paulo, Brazil

Site Status

Beneficencia Portuguesa de Sao Paulo

São Paulo, São Paulo, Brazil

Site Status

HEMORIO

Rio de Janeiro, , Brazil

Site Status

Hospital Samaritano

São Paulo, , Brazil

Site Status

CHU Henri Mondor

Créteil, , France

Site Status

Università degli Studi della Campania Luigi Vanvitelli

Napoli, Campania, Italy

Site Status

Azienda Ospedaliera di Verona-Policlinico G.B. Rossi

Verona, Veneto, Italy

Site Status

International Cancer Institute (ICI)

Eldoret, , Kenya

Site Status

Gertrude's Children Hospital

Nairobi, , Kenya

Site Status

Hopital Nini

Tripoli, , Lebanon

Site Status

Amsterdam UMC Location VUMC

Amsterdam, , Netherlands

Site Status

Charlotte Maxeke Johannesburg Hospital

Johannesburg, , South Africa

Site Status

Hospital General Univ. Gregorio Maranon

Madrid, , Spain

Site Status

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Site Status

Adana Acibadem Hospital; Pediatric Hematology

Adana, , Turkey (Türkiye)

Site Status

Cukurova University Medical Faculty Balcali Hospital

Adana, , Turkey (Türkiye)

Site Status

Mersin Universitesi Tip Fakultesi Hastanesi

Mersin, , Turkey (Türkiye)

Site Status

Central Middlesex Hospital

London, , United Kingdom

Site Status

Hammersmith Hospital

London, , United Kingdom

Site Status

Countries

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United States Brazil France Italy Kenya Lebanon Netherlands South Africa Spain Turkey (Türkiye) United Kingdom

Other Identifiers

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2020-004839-25

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2022-502542-28-00

Identifier Type: CTIS

Identifier Source: secondary_id

BO42451

Identifier Type: -

Identifier Source: org_study_id