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Crovalimab

Phase 3

Atypical Hemolytic Uremic Syndrome | Small molecule | Rare Disease |Roche Holding AG|Last Updated: Sep 1, 2026

Target and mechanism

Molecular targetC5
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials2
Total Enrollment124

FDA Designations

No designations recorded

Clinical trial landscape

Crovalimab · 8 trials · 4 indications

Phase 3 5Phase 2 1Phase 1 2
NCT04958265A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)Atypical Hemolytic Uremic Syndrome
ACTIVE NOT_RECRUITING41 Analytics
NCT04861259A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)Atypical Hemolytic Uremic Syndrome
ACTIVE NOT_RECRUITING83 Analytics
NCT04654468A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionParoxysmal Nocturnal Hemoglobinuria
ACTIVE NOT_RECRUITING51 Analytics
NCT04434092A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitorsParoxysmal Nocturnal Hemoglobinuria
ACTIVE NOT_RECRUITING210 Analytics
NCT04432584A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement InhibitorsParoxysmal Nocturnal Hemoglobinuria
ACTIVE NOT_RECRUITING190 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic SyndromeUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic SyndromeUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
Paroxysmal Nocturnal HemoglobinuriaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
Paroxysmal Nocturnal HemoglobinuriaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
Paroxysmal Nocturnal HemoglobinuriaUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants with complete TMA response (cTMAr) (Naive Cohort only)
Baseline up to Week 25 (after 24 weeks on treatment)
Percentage of Participants with Complete Thormbotic Microangiopathy Response (cTMAr)
Baseline up to Week 25 (after 24 weeks on treatment)
Mean Percentage of Participants With Hemolysis Control
From Week 5 up to Week 25

A Generalized Estimating Equation (GEE) was used to estimate the population-average percentage of the participants with hemolysis control (measured by lactate dehydrogenase (LDH) ≤1.5 x upper limit of normal (ULN)) from Week 5 to Week 25 taking account of the intra-patient and inter-patient correlation between LDH control statuses across visits. The dependent variable was the binary indicator for hemolysis control. Independent variables are categorical effects of visits, continuous baseline LDH.

Difference in Percentage of Participants With Transfusion Avoidance (TA) From Baseline Through Week 25 and Within 24 Weeks Prior to Screening
24 Weeks Prior to Screening, Baseline to Week 25

TA was defined as participants who were packed red blood cell (pRBC) transfusion-free and did not require transfusion per protocol-specified guidelines. TA within 24 weeks prior to screening was based on the pRBC transfusion history in the medical records. Reported in this outcome measure is the difference in the percentage of participants between "baseline through Week 25" and "within 24 weeks prior to screening". 95% Confidence Interval (CI) for the difference between the percentage of participants with transfusion avoidance between Pre-screening and Post-baseline is calculated using the Newcombe method. Screening= Day -28 to Day -1 and Baseline= Day 1.

Percentage of Participants With Transfusion Avoidance (TA)
Baseline to Week 25

TA is defined as participants who are packed red blood cell (pRBC) transfusion-free and do not require transfusion per protocol-specified guidelines. 95% Confidence Interval (CI) for percentage of participants with TA was calculated using the Wilson's method with continuity correction. Participants who withdrew before Week 25 were deemed to have had a transfusion. Percentages are rounded off to the nearest single decimal.

Percentage of Participants With Hemolysis Control Measured by LDH
Week 5 to Week 25

A Generalized Estimating Equation (GEE) was used to estimate the population-average percentage of the participants with hemolysis control (measured by LDH ≤1.5 x upper limit of normal (ULN)) from Week 5 to Week 25 taking account of the intra-patient and inter-patient correlation between LDH control statuses across visits. Percentages are rounded off to the nearest single decimal.

Percentage of Participants With Adverse Events (AEs) and by Severity
Up to approximately 6 years

Severity determined according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events, Version 5 (CTCAE v5).

Percentage of Participants With Injection-site Reactions, Infusion-related Reactions, Hypersensitivity and Infections (including Meningococcal Meningitis)
Up to approximately 6 years
Percentage of Participants With Adverse Events (AEs) Leading to Study Drug Discontinuation
Up to approximately 6 years
Percentage of Participants With Clinical Manifestations of Drug-target-drug Complex (DTDC) Formation Amongst Those Participants Who Switched to Crovalimab Treatment From Eculizumab Treatment or Ravulizumab Treatment
Up to approximately 6 years
Annualized rate of medical facility VOEs (AVR)
Baseline up to Week 49
Percentage of Participants With Adverse Events (AEs)
Baseline up to Day 84
Percentage of Participants with Infusion-Related Reactions and Hypersensitivity
Baseline up to Day 84
Part 1: Percentage of Participants With Dose-Limiting Events (DLEs)
Baseline up to approximately 3 months
Part 1: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline up to approximately 3 months
Part 2: Percentage of Participants With AEs and SAEs
Baseline up to approximately 8 months
Part 3: Percentage of Participants With AEs and SAEs
Baseline up to approximately 8 months
Part 4: Percentage of Participants With AEs and SAEs
Baseline up to approximately 8 months
Part 2: Terminal Complement Activity in Serum as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Baseline up to Day 224
Part 3: Terminal Complement Activity as Assessed by Ex Vivo Liposome Lysis in Serum Using the LIA
Baseline up to Day 224
Part 4: Terminal Complement Activity in Serum as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Baseline up to Day 224
OLE: Percentage of Participants With AEs and SAEs
OLE: Week 21 up to Week 567

Secondary Endpoints

Change from Baseline in Dialysis Status
Baseline up to Week 25 (after 24 weeks on treatment)
Change in Estimated Glomerular Filtration Rate (eGFR) (Naive and Switch Cohorts)
Baseline up to Week 25 (after 24 weeks on treatment)
Percentage of Participants with Change from Baseline in Chronic Kidney Disease (CKD) stage (Naive and Switch Cohorts)
Baseline up to Week 25 (after 24 weeks on treatment)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
CrovalimabEXPERIMENTALParticipants 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 C5 inhibitor and \[3\] Pretreated Cohort (includes C5 SNP (Single Nucleotide Polymorphism) participants) - participants who received treatment with another C5 inhibitor and subsequently discontinued it.
Arm A (Crovalimab)EXPERIMENTALCrovalimab will be administered at an initial loading dose of 1000 milligrams (mg) (for participants with body weight between 40 and 100 kilograms \[kg\]) or 1500 mg (for participants with body weight ≥ 100 kg), as intravenous (IV) injection on Day 1 of Week 1 followed by four weekly subcutaneous (SC) injections of 340 mg starting on Day 2 of Week 1 and then once weekly (QW) at Weeks 2,3 and 4. Thereafter crovalimab will be administered, as SC injection, at a maintenance dose of 680 mg (for participants with body weight between 40 and 100 kg) or 1020 mg (for participants with body weight ≥ 100 kg) once every 4 weeks (Q4W) from Week 5 for a total of 24 weeks of study treatment. Participants may continue to receive crovalimab after 24 weeks of treatment up to maximum of 5 years.
Arm B (Eculizumab)ACTIVE_COMPARATORParticipants will receive loading dose of eculizumab 600 mg on Days 1, 8, 15, and 22, followed by maintenance dose of 900 mg on Day 29 and every 2 weeks (Q2W) thereafter until 24 weeks. Participants may switch to receive crovalimab after 24 weeks of eculizumab treatment.
Arm C (Crovalimab) (Exploratory)EXPERIMENTALParticipants with a body weight ≥ 5 to \<12 kg will receive a loading series of crovalimab doses comprised of an IV dose on Day 1 of Week 1, followed by crovalimab SC dose on Day 2 Week 1. Maintenance doses will begin at Week 3 and will be administered Q2W, thereafter. Participants with a body weight ≥ 12 to \< 20 kg and ≥ 20 kg will receive a loading series of crovalimab doses comprised of an IV dose on Day 1 of Week 1, followed by weekly crovalimab SC doses for 4 weeks at Week 1 (Day 2) and then at Weeks 2, 3, and 4. Maintenance doses will begin at Week 5 and will be administered Q2W thereafter, for participants with a body weight ≥ 12 to \< 20 kg and Q4W thereafter, for participants with a body weight \> 20 kg. After 24 weeks of crovalimab treatment, participants who derive benefit from the drug may continue to receive crovalimab.
PlaceboPLACEBO_COMPARATORParticipants 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.
Part 1 (Healthy Volunteers): CrovalimabEXPERIMENTALHealthy participants will receive a single dose of crovalimab in each dose-escalation cohort of Part 1. Crovalimab will be administered at a starting dose of 75 milligrams (mg) intravenous (IV) infusion. Doses are planned to be escalated up to Cohort 5.
Part 1 (Healthy Volunteers): PlaceboPLACEBO_COMPARATORHealthy participants will receive a single dose of crovalimab matching placebo in each dose-escalation cohort of Part 1.
Part 2 (PNH Participants): CrovalimabEXPERIMENTALPNH participants will receive 3 single ascending doses (375 mg IV, 500 mg IV, 1000 mg IV of crovalimab) on Days 1, 8, and 22 followed by weekly crovalimab administrations up to a maximum of 5 months. Weekly crovalimab administrations will start no earlier than Day 36. The starting dose of Part 2 is based on data from Part 1 of the study.
Part 3 (PNH Participants): Crovalimab QWEXPERIMENTALParticipants will receive crovalimab at a dose of 1000 mg on Day 1 and 170 mg weekly (QW) starting on Day 8 for a maximum treatment duration of 5 months.
Part 3 (PNH Participants): Crovalimab Q2WEXPERIMENTALParticipants will receive crovalimab at a dose of 1000 mg on Day 1 and 340 mg every 2 weeks (Q2W) for a maximum treatment duration of 5 months.
Part 3 (PNH Participants): Crovalimab Q4WEXPERIMENTALParticipants will receive crovalimab at a dose of 1000 mg on Day 1 and 680 mg every 4 weeks (Q4W) starting on Day 8 for a maximum treatment duration of 5 months.
Part 4 (eculizumab pretreated PNH Participants): CrovalimabEXPERIMENTALPNH Participants pretreated with eculizumab will receive crovalimab: Participants \>/= 100 kilograms (kg): loading dose of 1500 mg IV on Day 1; Participants \< 100 kg: loading dose of 1000 mg IV on Day 1. In all Participants, the remainder of the loading series schedule will be 340 mg subcutaneous (SC) on Days 2, 8, 15, and 22. For Participants \>/= 100 kg, maintenance dosing will be 1020 mg SC on week 5 and then Q4W thereafter. Patients \< 100 kg will receive a maintenance dose of 680 mg SC on the same schedule.
Part 4 (treatment naïve PNH Participants): CrovalimabEXPERIMENTALTreatment naïve PNH Participants will receive: Participants \>/= 100 kg: loading dose of 1500 mg IV on day 1; Participants \< 100 kg: loading dose of 1000 mg IV on day 1. In all Participants, the remainder of the loading series schedule will be 340 mg SC on Days 2, 8, 15, and 22. For Participants \>/= 100 kg, maintenance dosing will be 1020 mg SC on week 5 and then Q4W thereafter. Patients \< 100 kg will receive a maintenance dose of 680 mg SC on the same schedule.
OLE (PNH Participants): CrovalimabEXPERIMENTALPNH Participants who participated in Parts 2, 3 and 4 and who derive clinical benefit from crovalimab may enroll into OLE. Participants will either receive 680 mg SC Q4W (body weight \>/= 40 kg to \< 100 kg) or 1020 mg SC Q4W (body weight \>/= 100 kg) for up to a maximum treatment duration of ten years from entry into OLE.

Interventions

NameTypeDescription
CrovalimabDRUGCrovalimab will be administered at a dose of 1000 mg intravenously (IV) (for participants weighing =\> 40 to \<100 kg) or 1500 mg IV (for participants weighing \>=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 subcutaneously (SC). On Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants weighing =\> 40 to \<100 kg) or 1020 mg SC (for participants weighing \>=100 kg). Enrollment of participants weighing \<40 kg will be staggered using two weight-based dose confirmation groups (Group 1 participants weighing \>=20 kg to \<40 kg, followed by Group 2 participants weighing \>=5 kg to \<20 kg). All participants will receive an initial IV loading dose, which will be followed by SC dosing at either Q2W or Q4W intervals (depending on body weight), until study completion.
EculizumabDRUGEculizumab will be administered as specified in the respective arm.
PlaceboDRUGMatching Placebo will be administered with the same dosing schedule and equivalent IV and SC volume as weight-based Crovalimab.
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Eligibility Criteria

Age Range28 Days to 17 Years
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Body weight \>= 5 kg at screening. * Vaccination against Neisseria meningitis serotypes A, C, W, and Y; vaccination against serotype B, according to national vaccination recommendations. * Vaccination against Haemophilus influenzae type B and Streptococcus pneumoniae, accordin...

Countries:United StatesBelgiumBrazilCanadaChinaFranceIndiaJapanMexicoPolandGermanyHungaryIsraelItalySpainTurkey (Türkiye)ArgentinaGreeceLithuaniaMalaysiaNetherlandsPhilippinesPortugalRomaniaSingaporeSouth KoreaTaiwanThailandUkraineUnited KingdomCzechiaEstoniaHong KongIrelandSaudi ArabiaSwedenKenyaLebanonSouth Africa
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Recent Changes (Last 90 Days)

HIGHSep 1, 2026NCT03157635Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHSep 1, 2026NCT03157635Status: ACTIVE_NOT_RECRUITING → COMPLETED
LOWAug 26, 2026NCT04654468Completion: 2026-12-31 → 2027-01-15
LOWAug 26, 2026NCT04654468Completion: 2026-12-31 → 2027-01-15
LOWAug 18, 2026NCT04434092Completion: 2027-09-30 → 2027-04-18
LOWAug 18, 2026NCT04434092Completion: 2027-09-30 → 2027-04-18
LOWAug 5, 2026NCT03157635primaryCompletionDate: changed

Frequently asked questions about Crovalimab

What is Crovalimab used for?

Crovalimab is an investigational drug being studied for paroxysmal nocturnal hemoglobinuria, sickle cell disease, and atypical hemolytic uremic syndrome. It is in Phase 3 clinical development for atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria, with additional studies in sickle cell disease.

What does Crovalimab target?

Crovalimab is a monoclonal antibody, indicated by its -mab suffix. It is designed to target components of the complement system, which is involved in the diseases it treats. The specific molecular target is not disclosed in available information.

Who is developing Crovalimab?

Crovalimab is being developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is conducting multiple clinical trials for the drug across various countries.

What phase is Crovalimab in?

Crovalimab is in Phase 3 clinical development. It has active Phase 3 trials for atypical hemolytic uremic syndrome in adults, adolescents, and pediatric patients. Earlier phase studies for sickle cell disease have been completed.

What clinical trials is Crovalimab in?

Crovalimab is being studied in several trials. NCT04861259 is a Phase 3 trial in atypical hemolytic uremic syndrome with 83 participants. NCT04958265 is a Phase 3 pediatric trial for the same condition with 41 participants. NCT05075824, a Phase 2 trial for sickle cell disease, has been completed.

Is Crovalimab the same as other drugs?

Crovalimab is a distinct investigational monoclonal antibody. No alternative names for Crovalimab have been reported in the available clinical trial information.