Trial · NCT04434092
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
NCT04434092, Phase 3, Active not recruiting, Hoffmann-La Roche; results first posted 2025-11-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. Trials, record NCT04434092. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04434092
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
- NCT number
- NCT04434092
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 210 participants, actual
- Lead sponsor
- Hoffmann-La Roche
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Crovalimab, Eculizumab
- First posted
- 2020-06-16
- Last updated
- 2026-10-26
- Other registries' IDs
- eudract:2019-004931-21
- Results first posted
- 2025-11-10
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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. |
| Primary | 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. |
| Secondary | Percentage of Participants With Breakthrough Hemolysis (BTH) | Baseline to Week 25 | BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \< 10 grams per deciliter (g/dL)\], major adverse vascular event \[MAVE, including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2xULN after a prior LDH reduction to ≤1.5xULN from the start of study treatment. 95% CI for percentage of participants with BTH was calculated using the Wilson's method with continuity correction. Participants who withdrew before Week 25 were deemed to have experienced a BTH event. Percentages are rounded off to the nearest single decimal. |
| Secondary | Percentage of Participants With Stabilization of Hemoglobin | Baseline to Week 25 | Stabilized hemoglobin is defined as avoidance of a \>= 2 g/dL decrease in hemoglobin level from baseline, in the absence of transfusion. 95% CI for percentage of participants with stabilized hemoglobin was calculated using the Wilson's method with continuity correction. Participants who withdrew before Week 25 were deemed to not have had hemoglobin stabilization. Percentages are rounded off to the nearest single decimal. |
| Secondary | Change From Baseline in Fatigue Assessed by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale | Baseline to Week 25 | Fatigue was assessed using FACIT-F scale. FACIT-F is a self-assessment questionnaire with a 7-day recall period and 13 items evaluating fatigue and its impact on daily life activities. Items are scored on a response scale that ranges from 0 ("not at all") to 4 ("very much so"). Relevant items are reverse scored, and all the items are summed to create a total score range from 0 to 52, with 0 being the worst possible score and 52 being the best possible score. A higher score indicates low fatigue severity. A positive mean change indicates improvement. FACIT-F was assessed in adult participants only. |
| Secondary | Percentage of Participants With Adverse Events (AEs) | Up to 6 years | An AE is any untoward medical occurrence in a participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. AEs are reported based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0. |
| Secondary | Percentage of Participants With Injection-Site Reactions, Infusion-Related Reactions, Hypersensitivity and Infections (Including Meningococcal Meningitis) | Up to 6 years | An AE is any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product or other protocol-imposed intervention, regardless of attribution. Injection-site reactions or infusion-related reaction are AEs related to the injection site that occur during or within 24 hours after study drug administration that are judged to be related to the study drug injection. |
| Secondary | Percentage of Participants With AEs Leading to Study Drug Discontinuation | Up to 6 years | An AE is any untoward medical occurrence in a participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. AEs are reported based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0. |
| Secondary | Percentage of Participants With Clinical Manifestations of Drug-Target-Drug Complex (DTDC) Formation Amongst Participants Who Switched to Crovalimab Treatment From Eculizumab Treatment | Up to 6 years | |
| Secondary | Serum Concentrations of Crovalimab And Eculizumab Over Time | Up to 6 years |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Transfusion Avoidance (TA) | Baseline to Week 25 | Primary Analysis Population (PAP) included all randomized participants in Arms A and B who received at least one dose of the originally assigned treatment and having at least one valid lactate dehydrogenase (LDH) level assessment by the central laboratory after the first IV infusion by planned treatment. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Hemolysis Control Measured by LDH | Week 5 to Week 25 | PAP included all randomized participants in Arms A and B who received at least one dose of the originally assigned treatment and having at least one valid LDH level assessment by the central laboratory after the first IV infusion by planned treatment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Breakthrough Hemolysis (BTH) | Baseline to Week 25 | PAP included all randomized participants in Arms A and B who received at least one dose of the originally assigned treatment and having at least one valid LDH level assessment by the central laboratory after the first IV infusion by planned treatment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Stabilization of Hemoglobin | Baseline to Week 25 | PAP included all randomized participants in Arms A and B who received at least one dose of the originally assigned treatment and having at least one valid LDH level assessment by the central laboratory after the first IV infusion by planned treatment. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Fatigue Assessed by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale | Baseline to Week 25 | PAP included all randomized participants in Arms A and B who received at least one dose of the originally assigned treatment and having at least one valid LDH level assessment by the central laboratory after the first IV infusion by planned treatment. Overall number of participants analyzed is the number of participants ≥18 years with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoints. | score on scale | MEAN |
| SECONDARY | Percentage of Participants With Adverse Events (AEs) | Up to 6 years | |||
| SECONDARY | Percentage of Participants With Injection-Site Reactions, Infusion-Related Reactions, Hypersensitivity and Infections (Including Meningococcal Meningitis) | Up to 6 years | |||
| SECONDARY | Percentage of Participants With AEs Leading to Study Drug Discontinuation | Up to 6 years | |||
| SECONDARY | Percentage of Participants With Clinical Manifestations of Drug-Target-Drug Complex (DTDC) Formation Amongst Participants Who Switched to Crovalimab Treatment From Eculizumab Treatment | Up to 6 years | |||
| SECONDARY | Serum Concentrations of Crovalimab And Eculizumab Over Time | Up to 6 years |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group83
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Transfusion Avoidance (TA) | Arm A: Crovalimab | 65.7 | 95% Confidence Interval 56.91 to 73.52 | percentage of participants | |
| Percentage of Participants With Transfusion Avoidance (TA) | Arm B: Eculizumab | 68.1 | 95% Confidence Interval 55.67 to 78.53 | percentage of participants | |
| Percentage of Participants With Hemolysis Control Measured by LDH | Arm A: Crovalimab | 79.3 | 95% Confidence Interval 72.86 to 84.48 | percentage of participants | |
| Percentage of Participants With Hemolysis Control Measured by LDH | Arm B: Eculizumab | 79.0 | 95% Confidence Interval 69.66 to 85.99 | percentage of participants | |
| Percentage of Participants With Breakthrough Hemolysis (BTH) | Arm A: Crovalimab | 10.4 | 95% Confidence Interval 6.04 to 17.21 | percentage of participants | |
| Percentage of Participants With Breakthrough Hemolysis (BTH) | Arm B: Eculizumab | 14.5 | 95% Confidence Interval 7.54 to 25.50 | percentage of participants | |
| Percentage of Participants With Stabilization of Hemoglobin | Arm A: Crovalimab | 63.4 | 95% Confidence Interval 54.63 to 71.45 | percentage of participants | |
| Percentage of Participants With Stabilization of Hemoglobin | Arm B: Eculizumab | 60.9 | 95% Confidence Interval 48.35 to 72.17 | percentage of participants | |
| Change From Baseline in Fatigue Assessed by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale | Arm A: Crovalimab | Baseline | 36.02 | Standard Error 0.877 | score on scale |
| Change From Baseline in Fatigue Assessed by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) Scale | Arm B: Eculizumab | Baseline | 35.09 | Standard Error 1.408 | score on scale |
1 to 10 of 83Source ClinicalTrials.gov
See all 83 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Treatment Period:Baseline-Week24 | STARTED | Arm A: Crovalimab | 135 | |
| Primary Treatment Period:Baseline-Week24 | STARTED | Arm B: Eculizumab | 69 | |
| Primary Treatment Period:Baseline-Week24 | STARTED | Arm C: Crovalimab | 6 | |
| Primary Treatment Period:Baseline-Week24 | COMPLETED | Arm A: Crovalimab | 129 | |
| Primary Treatment Period:Baseline-Week24 | COMPLETED | Arm B: Eculizumab | 68 | |
| Primary Treatment Period:Baseline-Week24 | COMPLETED | Arm C: Crovalimab | 6 | |
| Primary Treatment Period:Baseline-Week24 | NOT COMPLETED | Arm A: Crovalimab | 6 | |
| Primary Treatment Period:Baseline-Week24 | NOT COMPLETED | Arm B: Eculizumab | 1 | |
| Primary Treatment Period:Baseline-Week24 | NOT COMPLETED | Arm C: Crovalimab | 0 | |
| Extension Treatment Period:Week 25-5 Yrs | STARTED | Arm A: Crovalimab | 129 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events183
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Aphthous ulcer | Gastrointestinal disorders | Arm A: Crovalimab | 0 | 135 | 0 |
| other | Aphthous ulcer | Gastrointestinal disorders | Arm B: Eculizumab | 0 | 69 | 0 |
| other | Aphthous ulcer | Gastrointestinal disorders | Arm C: Crovalimab | 1 | 6 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Arm A: Crovalimab | 11 | 135 | 12 |
| other | Diarrhoea | Gastrointestinal disorders | Arm B: Eculizumab | 2 | 69 | 2 |
| other | Diarrhoea | Gastrointestinal disorders | Arm C: Crovalimab | 0 | 6 | 0 |
| other | Fatigue | General disorders | Arm A: Crovalimab | 4 | 135 | 4 |
| other | Fatigue | General disorders | Arm B: Eculizumab | 3 | 69 | 3 |
| other | Fatigue | General disorders | Arm C: Crovalimab | 1 | 6 | 1 |
| other | Pyrexia | General disorders | Arm A: Crovalimab | 15 | 135 | 18 |
1 to 10 of 183Source ClinicalTrials.gov
See all 183 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm A: Crovalimab | 2 | 135 |
| Total, all-cause mortality | Arm B: Eculizumab | 1 | 69 |
| Total, all-cause mortality | Arm C: Crovalimab | 0 | 6 |
| Total, other adverse events | Arm A: Crovalimab | 102 | 135 |
| Total, other adverse events | Arm B: Eculizumab | 55 | 69 |
| Total, other adverse events | Arm C: Crovalimab | 5 | 6 |
| Total, serious adverse events | Arm A: Crovalimab | 22 | 135 |
| Total, serious adverse events | Arm B: Eculizumab | 14 | 69 |
| Total, serious adverse events | Arm C: Crovalimab | 0 | 6 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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