Paroxysmal Nocturnal HemoglobinuriaPhase 3COMMODORE 3NCT04654468
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
A Phase 3 study testing Crovalimab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Hoffmann-La Roche at 5 sites in China.
Trial summary
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 50 participants will be treated with Crovalimab for at least 24 weeks. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Crovalimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 5 sitesChina
- Study dates
- 17 Mar 2021to 15 Jan 2027
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Body weight ≥ 40 kg at screening
- Willingness and ability to comply with all study visits and procedures
- Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
- LDH Levels ≥ 2x the ULN at screening
- Participants who have at least four transfusions during 12 months prior to screening (documented in the medical record)
- Presence of one or more of the PNH-related signs or symptoms within 3 months of screening
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- Vaccination against Neisseria meningitidis serotypes A, C, W, and Y < 3 years prior to initiation of study treatment (Day 1)
- Vaccination against Haemophilius influenzae type B and Streptococcus pneumonia according to national vaccination recommendations
- For participants receiving other therapies (e.g., immunosuppressants, corticosteroids): stable dose for ≥ 28 days prior to screening and up to the first drug administration
- Adequate hepatic and renal function
- Women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception during the treatment period and for 46 weeks (approximately 10.5 months) after the final dose of crovalimab
- Platelet count ≥30,000 per cubic millimeter (mm^3) at screening
- ANC > 500/microlitres (μl) at screening
Who cannot join
- Current or previous treatment with a complement inhibitor
- History of allogeneic bone marrow transplantation
- History of Neisseria meningitidis infection within 6 months prior to screening and up to first drug administration
- Known or suspected immune or hereditary complement deficiency
- Known HIV infection with cluster of differentiation 4 (CD4) count < 200 cells/µl within 24 weeks prior to screening
- Infection requiring hospitalization or treatment with IV antibiotics within 28 days prior to screening and up to the first drug administration, or oral antibiotics within 14 days prior to screening and up to the first drug administration
Show all 12Show fewer
- Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration
- Presence of fever (≥ 38˚C) within 7 days before the first drug administration
- Splenectomy < 6 months before screening
- History of malignancy within 5 years prior to screening and up to the first drug administration
- Pregnant or intending to become pregnant during the study or within 46 weeks (10.5 months) after the final dose of study treatment
- Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within 28 days of screening or within 5 half-lives of that investigational product, whichever is greater
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
West China Hospital, Sichuan University
Chengdu, 610041
Guangdong General Hospital
Guangzhou, 510115
Institute of Hematology and Hospital of Blood Disease
Tianjin, 300020
Tianjin Medical University General Hospital
Tianjin, 300052
Union Hospital Tongji Medical College Huazhong University of Science and Technology
Wuhan, 430022
About this study
Official title: A Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 50 participants will be treated with Crovalimab for at least 24 weeks.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Crovalimab
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrovalimabDrug
Crovalimab will be administered at a dose of 1000 milligrams (mg) IV (for participants with body weight between 40 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, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100 kg) or 1020 mg SC (for participants with body weight ≥ 100 kg). Dosing schedule will be as described above.
| Study group | Role | What they receive |
|---|---|---|
| Crovalimab | Experimental | Participants will receive a loading series of crovalimab comprised of an intravenous (IV) 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 Q4W (every 4 weeks) thereafter for a total of 24 weeks of study treatment. After 24 weeks of crovalimab treatment, participants who derive benefit from the drug may continue to receive crovalimab. Receives: Drug: Crovalimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Percentage of Participants With Hemolysis Control
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.
Measured: From Week 5 up to Week 25
Difference in Percentage of Participants With Transfusion Avoidance (TA) From Baseline Through Week 25 and Within 24 Weeks Prior to Screening
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.
Measured: 24 Weeks Prior to Screening, Baseline to Week 25
Secondary outcomes (17)
Percentage of Participants With Breakthrough Hemolysis (BTH)
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. As pre-specified in the SAP participants withdrawing before Week 25 were deemed to have experienced a BTH event. Percentages have been rounded off to the first decimal point.
Measured: Baseline, Week 25
Percentage of Participants With Stabilized Hemoglobin
Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline, in the absence of transfusion. As pre-specified in the SAP participants withdrawing before Week 25 were deemed to not have hemoglobin stabilization.
Measured: Baseline, Week 25
Change From Baseline in Fatigue in Adults Aged >=18 Years
Fatigue was assessed using functional assessment of chronic illness therapy-fatigue (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. FACIT-F assessment was unintentionally missed in the Schedule of Activities (SoA) table, which site used to guide the assessments at each timepoint. Therefore, data were not collected at Week 25.
Measured: Baseline, Week 2, Week 5, Week 9, Week 17, Week 25
Percentage of Participants With Adverse Events (AEs)
Measured: Up to 7 years
Percentage of Participants With Injection-Site Reactions, Infusion-Related Reactions, Hypersensitivity, and Infections (Including Meningococcal Meningitis)
Measured: Up to 7 years
Percentage of Participants With Adverse Events (AEs) Leading to Study Drug Discontinuation
Measured: Up to 7 years
Trough Serum Concentration of Crovalimab Over Time
Measured: Up to 7 years
Serum Concentrations of Crovalimab
Measured: Up to 7 years
Percentage of Participants With Anti-Drug Antibodies (ADAs) to Crovalimab
Measured: Up to 7 years
Terminal Complement Activity as Measured by Liposome Immunoassay (LIA)
Measured: Up to 7 years
Change Over Time in Total and Free C5 Concentration
Measured: Up to 7 years
Observed Value in Absolute Reticulocyte Count
Measured: Up to 7 years
Observed Value in Free Hemoglobin
Measured: Up to 7 years
Observed Value in Haptoglobin
Measured: Up to 7 years
Percent Change From Baseline in Absolute Reticulocyte Count
Measured: Baseline, Week 25
Percent Change From Baseline in Free Hemoglobin
Measured: Baseline, Week 25
Percent Change From Baseline in Haptoglobin
Measured: Baseline, Week 25
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2020
- Study start17 Mar 2021
- Primary completion10 Feb 2022
- Study completion15 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04654468
- Sponsor's protocol ID
- YO42311
- Acronym
- COMMODORE 3
- Status last verified
- Aug 2026
- First submitted
- 30 Nov 2020
- Last updated
- 26 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Publications
Roth A, Fu R, He G, Alzahrani H, Chou SC, Hicheri Y, Kazmierczak M, Recova VL, Uchiyama M, Vladareanu AM, Beveridge L, Buatois S, Buri M, Compagno N, Shi D, Balachandran N, Sreckovic S, Scheinberg P. Safety of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH): Pooled Results From the Phase 3 COMMODORE Studies. Eur J Haematol. 2025 Feb;114(2):373-382. doi: 10.1111/ejh.14339. Epub 2024 Nov 13.
PubMed 39535306Liu H, Xia L, Weng J, Zhang F, He C, Gao S, Jia J, Chang AC, Lundberg P, Shi J, Sima CS, Sostelly A, Sreckovic S, Xiao Z, Zhang Z, Fu R. Efficacy and safety of the C5 inhibitor crovalimab in complement inhibitor-naive patients with PNH (COMMODORE 3): A multicenter, Phase 3, single-arm study. Am J Hematol. 2023 Sep;98(9):1407-1414. doi: 10.1002/ajh.26998. Epub 2023 Jul 8.
PubMed 37421604
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Multicenter, Single Arm Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
- ClinicalTrials.gov ID
- NCT04654468
- Acronym / sponsor ID
- COMMODORE 3 / YO42311
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 51 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 17 Mar 2021 (actual)
- Primary completion
- 10 Feb 2022 (actual)
- Study completion
- 15 Jan 2027 (estimated)
- First posted
- 4 Dec 2020 (actual)
- Results
- Posted 5 Jun 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 50 participants will be treated with Crovalimab for at least 24 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Crovalimab | Experimental | Drug: CrovalimabParticipants will receive a loading series of crovalimab comprised of an intravenous (IV) 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 Q4W (every 4 weeks) thereafter for a total of 24 weeks of study treatment. After 24 weeks of crovalimab treatment, participants who derive benefit from the drug may continue to receive crovalimab. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Body weight ≥ 40 kg at screening
- Willingness and ability to comply with all study visits and procedures
- Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
- LDH Levels ≥ 2x the ULN at screening
- Participants who have at least four transfusions during 12 months prior to screening (documented in the medical record)
Key exclusion criteria (12 listed)
- Current or previous treatment with a complement inhibitor
- History of allogeneic bone marrow transplantation
- History of Neisseria meningitidis infection within 6 months prior to screening and up to first drug administration
- Known or suspected immune or hereditary complement deficiency
- Known HIV infection with cluster of differentiation 4 (CD4) count < 200 cells/µl within 24 weeks prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CrovalimabCrovalimab will be administered at a dose of 1000 milligrams (mg) IV (for participants with body weight between 40 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, it will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, it will be administered at a dose of 680 mg SC (for participants with body weight between 40 and 100 kg) or 1020 mg SC (for participants with body weight ≥ 100 kg). Dosing schedule will be as described above. | Drug | Crovalimab |
Primary outcome
Mean Percentage of Participants With Hemolysis Control
Time frame: 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
Time frame: 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.
17 secondary outcomes
Percentage of Participants With Breakthrough Hemolysis (BTH)
Time frame: Baseline, Week 25
Percentage of Participants With Stabilized Hemoglobin
Time frame: Baseline, Week 25
Change From Baseline in Fatigue in Adults Aged >=18 Years
Time frame: Baseline, Week 2, Week 5, Week 9, Week 17, Week 25
Percentage of Participants With Adverse Events (AEs)
Time frame: Up to 7 years
Percentage of Participants With Injection-Site Reactions, Infusion-Related Reactions, Hypersensitivity, and Infections (Including Meningococcal Meningitis)
Time frame: Up to 7 years
Percentage of Participants With Adverse Events (AEs) Leading to Study Drug Discontinuation
Time frame: Up to 7 years
Trough Serum Concentration of Crovalimab Over Time
Time frame: Up to 7 years
Serum Concentrations of Crovalimab
Time frame: Up to 7 years
Percentage of Participants With Anti-Drug Antibodies (ADAs) to Crovalimab
Time frame: Up to 7 years
Terminal Complement Activity as Measured by Liposome Immunoassay (LIA)
Time frame: Up to 7 years
Change Over Time in Total and Free C5 Concentration
Time frame: Up to 7 years
Observed Value in Absolute Reticulocyte Count
Time frame: Up to 7 years
Observed Value in Free Hemoglobin
Time frame: Up to 7 years
Observed Value in Haptoglobin
Time frame: Up to 7 years
Percent Change From Baseline in Absolute Reticulocyte Count
Time frame: Baseline, Week 25
Percent Change From Baseline in Free Hemoglobin
Time frame: Baseline, Week 25
Percent Change From Baseline in Haptoglobin
Time frame: Baseline, Week 25
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39535306Roth A, Fu R, He G, Alzahrani H, Chou SC, Hicheri Y, Kazmierczak M, Recova VL, Uchiyama M, Vladareanu AM, Beveridge L, Buatois S, Buri M, Compagno N, Shi D, Balachandran N, Sreckovic S, Scheinberg P. Safety of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH): Pooled Results From the Phase 3 COMMODORE Studies. Eur J Haematol. 2025 Feb;114(2):373-382. doi: 10.1111/ejh.14339. Epub 2024 Nov 13.
- Derived from this study: PubMed 37421604Liu H, Xia L, Weng J, Zhang F, He C, Gao S, Jia J, Chang AC, Lundberg P, Shi J, Sima CS, Sostelly A, Sreckovic S, Xiao Z, Zhang Z, Fu R. Efficacy and safety of the C5 inhibitor crovalimab in complement inhibitor-naive patients with PNH (COMMODORE 3): A multicenter, Phase 3, single-arm study. Am J Hematol. 2023 Sep;98(9):1407-1414. doi: 10.1002/ajh.26998. Epub 2023 Jul 8.
- Individual participant dataSharing planned: Yes. For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Crovalimab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Hoffmann-La Roche at 5 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Jun 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Percentage of Participants With Hemolysis Control (time frame: From Week 5 up to Week 25). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04654468, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.