A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT ID: NCT04861259

Last Updated: 2026-01-22

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

PHASE3

Total Enrollment

83 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-22

Study Completion Date

2029-08-20

Brief Summary

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This study aims to evaluate the efficacy and safety of crovalimab in adult and adolescent participants with aHUS.

Detailed Description

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Conditions

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Atypical Hemolytic Uremic Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Crovalimab

Participants will be enrolled in three cohorts: \[1\] Naive Cohort - participants who have not been previously treated with complement inhibitor therapy; \[2\] Switch Cohort - participants who switch to crovalimab from another Complement Component 5 (C5) inhibitor and \[3\] C5 Single Nucleotide Polymorphism (C5 inhibitor) Cohort - participants with documented C5 polymorphism.

Group Type EXPERIMENTAL

Crovalimab

Intervention Type DRUG

Crovalimab will be administered at a dose of 1000 milligrams (mg) intravenous (IV) (for participants with body weight at least 40 (\>=) and up to 100 kilograms (kg) or 1500 mg IV (for participants with body weight \>=100kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg subcutaneously (SC). On Week 5 and every 4 weeks (Q4W) thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight \>= 40kg to \<100kg) or 1020 mg SC (for participants with body weight \>=100kg).

Interventions

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Crovalimab

Crovalimab will be administered at a dose of 1000 milligrams (mg) intravenous (IV) (for participants with body weight at least 40 (\>=) and up to 100 kilograms (kg) or 1500 mg IV (for participants with body weight \>=100kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, it will be administered at a dose of 340 mg subcutaneously (SC). On Week 5 and every 4 weeks (Q4W) thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight \>= 40kg to \<100kg) or 1020 mg SC (for participants with body weight \>=100kg).

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Body weight \>= 40 kg at screening.
* Vaccination against Neisseria meningitidis serotypes A, C, W, and Y; vaccination against serotypes B, according to national vaccination recommendations.
* Vaccination against Haemophilus influenzae type B and Streptococcus pneumoniae, according to national vaccination recommendations.
* For participants continuing to receive other therapies concomitantly with crovalimab (e.g., immunosuppressants, corticosteroids, mammalian target of rapamycin inhibitor (mTORi) , or calcineurin inhibitors): stable dose for \>=28 days prior to screening and up to the first crovalimab administration.
* For female participants of childbearing potential: an agreement to remain abstinent or use contraception.
* Female participants of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of crovalimab.
* Participants with a prior kidney transplant are eligible if they have a known history of complement-mediated aHUS prior to the kidney transplant.
* Onset of initial TMA presentation within 28 days prior to the first dose of crovalimab (for Naive Cohort only).
* Documented treatment with either eculizumab or ravulizumab (for Switch Cohort only).
* Clinical evidence of response to a C5 inhibitor (for Switch Cohort only).
* Known C5 polymorphism (for C5 SNP Cohort only).
* Poorly controlled TMA following treatment with another C5 inhibitor (for C5 SNP Cohort only).

Exclusion Criteria

* TMA associated with non-aHUS related renal disease.
* Positive direct Coombs test.
* Chronic dialysis within 90 days prior to first crovalimab administration and/or end stage renal disease.
* Identified drug exposure-related TMA.
* Presence or history of a condition that could trigger TMA, such as malignancy, bone marrow or organ transplant (other than kidney transplant) or autoimmune disease.
* History of a kidney disease, other than aHUS.
* History of Neisseria meningitidis infection within 6 months of study enrollment.
* Known or suspected immune deficiency (e.g., history of frequent recurrent infections).
* Positive Human Immunodeficiency Virus (HIV) test.
* Active systemic bacterial, viral, or fungal infection within 14 days before first crovalimab administration
* Presence of fever (\>= 38°C)
* Multi-system organ dysfunction or failure.
* Recent intravenous immunoglobulin (IVIg) treatment.
* Pregnant or breastfeeding or intending to become pregnant.
* Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within 28 days of screening or within five half lives of that investigational product, whichever is greater.
* Recent use of tranexamic acid.
* Current or previous treatment with a complement inhibitor (for Naive Cohort only).
* First initiation of plasma exchange/plasma infusions (PE/PI) should not be more than 28 days prior to first crovalimab administration (for Naive Cohort only).
* Last PE/PI completed less than 2 hours prior to first crovalimab administration (for Naive Chorot only).
* Receiving PE/PI within 8 weeks of the first crovalimab administration (Switch Cohort only).
* Positive for active Hepatitis B and C infection (HBV/HCV) (for Switch Cohort and C5 SNP Cohort participants who recently received C5 inhibitor treatment).
* Cryoglobulinemia at screening (for Switch Cohort and C5 SNP Cohort participants who recently received C5 inhibitor treatment).
* Diagnosis of condition leading to non-aHUS TMA: Thrombotic Thrombocytopenic Purpura (TTP), Shiga Toxin producing Escherichia Coli (STEC)
* TMA, Pneumococcal HUS, TMA secondary to cobalamin C defect and TMA related to a known DGKE nephropathy.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chugai Pharmaceutical

INDUSTRY

Sponsor Role collaborator

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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Univ of CA San Francisco

San Francisco, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Site Status

UT Health Science Center

San Antonio, Texas, United States

Site Status

UZ Leuven Gasthuisberg

Leuven, , Belgium

Site Status

Santa Casa de Misericordia

Belo Horizonte, Minas Gerais, Brazil

Site Status

UPECLIN Hospital das Clinicas da Faculdade de Medicina de Botucatu

Botucatu, São Paulo, Brazil

Site Status

Hospital das Clinicas - FMUSP

São Paulo, São Paulo, Brazil

Site Status

Vancouver General Hospital

Vancouver, British Columbia, Canada

Site Status

Peking University First Hospital

Beijing, , China

Site Status

Hopital Lapeyronie

Montpellier, , France

Site Status

Hôpital Arnaud de Villeneuve

Montpellier, , France

Site Status

Hôpital Robert Debré

Paris, , France

Site Status

Hopital Tenon

Paris, , France

Site Status

Klinik II für Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin

Cologne, , Germany

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Klinik für Nephrologie des Universitätsklinikum Essen

Essen, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Del- Pesti Centrumkorhaz- Szent Laszlo Korhaz Telephely

Budapest, , Hungary

Site Status

Medanta-The Medicity

Gurgaon, Haryana, India

Site Status

All India Institute Of Medical Sciences (AIIMS)

New Delhi, National Capital Territory of Delhi, India

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

Rabin Medical Center

Petah Tikva, , Israel

Site Status

Sheba MC

Ramat Gan, , Israel

Site Status

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Lazio, Italy

Site Status

A.O. Universitaria S. Martino Di Genova

Genoa, Liguria, Italy

Site Status

Nagoya University Hospital

Aichi, , Japan

Site Status

Saitama Medical University Hospital

Saitama, , Japan

Site Status

The University of Tokyo Hospital

Tokyo, , Japan

Site Status

Hospital General de México

Distrito Federal, Mexico CITY (federal District), Mexico

Site Status

Instituto Nacional de Ciencias

Mexico City, Mexico CITY (federal District), Mexico

Site Status

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, Mexico

Site Status

Hospital de Especialidades Puerta de Hierro S.A de C.V.

Zapopan, , Mexico

Site Status

Instytut ?Centrum Zdrowia Matki Polki

Lodz, , Poland

Site Status

Hospital Clinic i Provincial

Barcelona, , Spain

Site Status

Hospital Universitario Virgen del Rocío

Seville, , Spain

Site Status

Istanbul University Istanbul Medical Faculty

Istanbul, , Turkey (Türkiye)

Site Status

Kocaeli University Medical Faculty

Kocaeli, , Turkey (Türkiye)

Site Status

Necmettin Erbakan University Meram Medical Faculty

Konya, , Turkey (Türkiye)

Site Status

Malatya Park Hospital

Malatya, , Turkey (Türkiye)

Site Status

Countries

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United States Belgium Brazil Canada China France Germany Hungary India Israel Italy Japan Mexico Poland Spain Turkey (Türkiye)

Other Identifiers

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2020-002475-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-505089-27-00

Identifier Type: CTIS

Identifier Source: secondary_id

BO42353

Identifier Type: -

Identifier Source: org_study_id

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