Paroxysmal Hemoglobinuria, NocturnalPhase 1/2COMPOSERNCT03157635
Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria
A Phase 1/2 study testing Crovalimab in people with Paroxysmal Hemoglobinuria, Nocturnal, run by Hoffmann-La Roche at 15 sites in 7 countries.
Trial summary
This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be evaluated in healthy volunteers (HV) during part 1. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Hemoglobinuria, Nocturnal
- What is being tested?
- Crovalimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 59 participants
- Where
- 15 sitesin 7 countries
- Study dates
- 14 Nov 2016to 13 Aug 2026
- Phase
- Phase 1/2
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 18 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Part 1 (HVs only):
- Healthy male volunteers, aged between 21 and 55 years inclusive
- Participants with a negative hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C antibody, and human immunodeficiency virus (HIV) test result
- Participants who have been vaccinated against hepatitis B
- No evidence of Neisseria meningococci in nasopharyngeal swab
- Neisseria meningitidis vaccination against serogroups B and A, C, W, and Y
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- Non-smokers, or former smokers, who have not smoked for at least 60 days prior to screening
- Parts 2, 3 and 4 (PNH participants only):
- Male or female participants with PNH between 18 and 75 years of age
- Neisseria meningitidis vaccination in accordance with most current local guidelines or standard of care (SOC) for participants at increased risk for meningococcal disease (Part 2 and 4)
- Participant has been vaccinated with Neisseria meningitidis vaccine(s) in accordance with most current local guidelines or SOC for participants at increased risk for meningococcal disease or is being revaccinated if applicable (Part 3 and 4)
- Antibiotic prophylaxis for meningococcal infection must be initiated prior to initiation of crovalimab therapy if the time period between initial Neisseria meningitidis vaccination and first dose of crovalimab is less than 2 weeks (Part 2 and 4)
- Antibiotic prophylaxis of meningococcal infection may be initiated prior to initiation of crovalimab therapy based on local guidelines or SOC for participants at increased risk for meningococcal disease e.g., splenectomized patients (Parts 2 and 4)
- Stable dose for greater than or equal to (>/=) 28 days prior to screening of other therapies (immunosuppressant therapy, corticosteroids, iron supplements)
- Part 2 and 4 (currently untreated PNH participants who are candidates for treatment with complement inhibitors only):
- PNH participants who have not been treated with any complement inhibitor or if previously treated stopped treatment due to lack of efficacy based on a single missense C5 heterozygous mutation
- Serum LDH levels at least 1.5-fold above the ULN at screening
- Hepatitis B participants can be enrolled if their liver function test values are less than 2 x ULN and there is no liver function impairment
- Part 3 and 4 (PNH participants currently treated with eculizumab only):
- PNH participants who have been treated continuously with eculizumab for at least 3 months preceding enrollment in the trial
- Participants receive regular infusions of eculizumab
- Subjects with a negative hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C antibody, and HIV test result
- OLE only - PNH participants:
- PNH participants who have completed Parts 2, 3 and 4 respectively
- PNH participants who derived, in the investigator's opinion, benefit from treatment with crovalimab
- Vaccination currency for Neisseria meningitidis serotypes A, C, W, Y and B should be maintained throughout the OLE
- All Parts:
- Female participants should use proper means of contraception
Who cannot join
- Part 1 (HVs only):
- Any clinically relevant history or the presence of moderate to severe respiratory, renal, hepatic, gastrointestinal, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, or connective tissue disease
- Any major illness within 1 month before the screening
- Prior splenectomy
- History of clinically significant hypersensitivity (example: drugs, excipients) or allergic reactions
- History or presence of clinically significant electrocardiogram (ECG) abnormalities or cardiovascular disease
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- Any contra-indication for receiving Neisseria meningitides vaccination and antibiotic prophylaxis therapy as required in the study
- Congenital or acquired complement deficiency
- Carriers of Neisseria meningitides based on cultures from nasopharyngeal swabs
- Known active viral, bacterial or fungal infection including herpes, herpes zoster or cold sores, during the last 14 days prior to first study drug administration
- Signs of parasitic infection (example: eosinophilia, diarrhea)
- History of significant recurrent infections in the opinion of the investigator
- Parts 2, 3 and 4 - PNH participants only:
- Evidence of moderate to severe concurrent renal, liver, cardiac, pulmonary or gastrointestinal disease not related to PNH as determined by the investigator
- History of an illness that, in the opinion of the study investigator, might confound the results of the study or that poses an additional risk to the participant by his or her participation in the study
- History of bone marrow transplantation
- Treatment with azathioprine or erythrocyte-stimulating agents within 14 days prior to first study drug administration
- Splenectomy <1 year before start of crovalimab.
- Part 3 and 4 - PNH patients only:
- Any evidence of sero-positive auto-immune connective tissue diseases (such as systemic lupus erythematosus, or rheumatoid arthritis)
- Any evidence of active inflammatory conditions (including inflammatory bowel disease, or cryoglobulinemia)
- All Parts:
- Under active therapy with intravenous immunoglobulin (IVIG)
- Mentally incapacitated or history of a clinically significant psychiatric disorder over the previous 5 years
- Known or suspected hereditary complement deficiency
- History of meningococcal meningitis
- History of allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies or known hypersensitivity to any constituent of the product
- Any major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within 28 days prior to screening or oral antibiotics within 2 weeks prior to screening and up to first study drug administration
- History of or currently active primary or secondary immunodeficiency, including known history of human immunodeficiency virus (HIV) infection
- Evidence of chronic active hepatitis C infection
- Evidence of malignant disease including myelodysplastic syndrome, or malignancies diagnosed within the previous 5 years
- Pregnant or breastfeeding, or intending to become pregnant during the study, including the OLE period, within 46 weeks (approximately 10.5 months) after the final dose of crovalimab
Trial locations and contacts
15 sites in 7 countries.
15 sites in 7 countries
Japan5 sites
Tohoku University Hospital
Miyagi, 980-8574
Osaka University Hospital
Osaka, 565-0871
NTT Medical Center Tokyo
Tokyo, 141-8625
Tokyo Medical University Hospital
Tokyo, 160-0023
University of Tsukuba Hospital
Tsukuba, 305-8576
Germany3 sites
Uniklinik RWTH Aachen
Aachen, 52074
Universitätsklinikum Essen
Essen, 45122
Universitätsklinikum Ulm
Ulm, 89081
Hungary2 sites
Semmelweis Egyetem, 1. Szamu Belgyogyaszati Klinika, Diabetologia
Budapest, 1083
Kaposi Mor Teaching Hospital, Dept of Internal Medicine/Hematology
Kaposvár, 7400
South Korea2 sites
Severance Hospital, Yonsei University Health System
Seoul, 03722
Seoul National University Hosp
Seoul, 110-744
France1 site
Institut hematologie Centre Hayem CHU paris Saint-Louis Lariboisiere F Widal Hopital St Louis
Paris, 75475
About this study
Official title: An Adaptive Phase I/II Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a Phase I/II, first-in-human study consisting of four sequential parts and an open-label extension (OLE). The safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single doses of crovalimab will be evaluated in healthy volunteers (HV) during part 1. The safety, tolerability, PK and PD of multiple doses of crovalimab will be evaluated in participants with paroxysmal nocturnal hemoglobinuria (PNH) in parts 2, 3, 4, and OLE of the study. Efficacy of crovalimab will be evaluated in Parts 2, 3, and 4.
- Conditions
- Paroxysmal Hemoglobinuria, Nocturnal
- Being tested
- Crovalimab, Placebo
- 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 as per schedule described in individual arm.
PlaceboDrug
Placebo will be administered as per schedule described in Part 1 placebo arm.
| Study group | Role | What they receive |
|---|---|---|
| Part 1 (Healthy Volunteers): Crovalimab | Experimental | Healthy 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. Receives: Drug: Crovalimab |
| Part 1 (Healthy Volunteers): Placebo | Placebo comparator | Healthy participants will receive a single dose of crovalimab matching placebo in each dose-escalation cohort of Part 1. Receives: Drug: Placebo |
| Part 2 (PNH Participants): Crovalimab | Experimental | PNH 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. Receives: Drug: Crovalimab |
| Part 3 (PNH Participants): Crovalimab QW | Experimental | Participants 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. Receives: Drug: Crovalimab |
| Part 3 (PNH Participants): Crovalimab Q2W | Experimental | Participants 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. Receives: Drug: Crovalimab |
| Part 3 (PNH Participants): Crovalimab Q4W | Experimental | Participants 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. Receives: Drug: Crovalimab |
| Part 4 (eculizumab pretreated PNH Participants): Crovalimab | Experimental | PNH 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. Receives: Drug: Crovalimab |
| Part 4 (treatment naïve PNH Participants): Crovalimab | Experimental | Treatment 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. Receives: Drug: Crovalimab |
| OLE (PNH Participants): Crovalimab | Experimental | PNH 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. Receives: Drug: Crovalimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Percentage of Participants With Dose-Limiting Events (DLEs)
Measured: Baseline up to approximately 3 months
Part 1: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Baseline up to approximately 3 months
Part 2: Percentage of Participants With AEs and SAEs
Measured: Baseline up to approximately 8 months
Part 3: Percentage of Participants With AEs and SAEs
Measured: Baseline up to approximately 8 months
Part 4: Percentage of Participants With AEs and SAEs
Measured: Baseline up to approximately 8 months
Part 2: Terminal Complement Activity in Serum as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Measured: Baseline up to Day 224
Part 3: Terminal Complement Activity as Assessed by Ex Vivo Liposome Lysis in Serum Using the LIA
Measured: Baseline up to Day 224
Part 4: Terminal Complement Activity in Serum as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Measured: Baseline up to Day 224
OLE: Percentage of Participants With AEs and SAEs
Measured: OLE: Week 21 up to Week 567
Secondary outcomes (52)
Part 1: Terminal Complement Activity as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Measured: Part 1: Baseline up to Day 91 (assessed at predose [Hour 0], end of infusion [EOI] [1 Hour], Hours 2, 6, 12 on Day 1; Days 2, 3, 4, 5, 7, 14, 21, 28, 35, 42, 56, 91)
Part 2: Serum Lactate Dehydrogenase (LDH) Levels
Measured: Predose (Hour 0), Hours 10-12 on Days 1, 8; Days 2, 5, 9, 15, 22, 29, 36, 43, 50, 64, 78, 92, 106, 120, 134, 224
Part 3: Serum LDH Levels
Measured: Part 3: Predose (Hour 0), Hours 10-12 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 36, 64, 78, 92, 106, 134; Day 224
Part 4: Serum LDH Levels
Measured: Part 4: Predose (Hour 0), Hour 6 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 43, 57, 85, 113, 134; Day 224
Part 1: Total Complement Component 5 (C5) Concentration
Measured: Part 1: Predose (Hour 0), EOI (1 Hour), Hours 2, 6, 12 on Day 1; Days 2, 3, 4, 5, 7, 14, 21, 28, 35, 42, 56, 91
Part 2: Total C5 Concentration
Measured: Part 2: Predose (Hour 0), EOI (1 Hour), Hours 2, 6, 10-12 on Day 1; Days 2, 5, 9, 15, 29, 224; predose [Hour 0], EOI [1 Hour], Hours 10-12 on Days 8, 22; predose [Hour 0] on Days 36, 43, 50, 64, 78, 92, 106, 120, 134
Part 3: Total C5 Concentration
Measured: Part 3: Predose (Hour 0), EOI (1 Hour), Hours 2 and 6 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 36, 43, 50, 57, 64, 78, 92, 106; Day 224
Part 4: Total C5 Concentration
Measured: Part 4: Predose (Hour 0), EOI (1 Hour), Hours 2, 6 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 57, 85, 113; Days 43, 134, 224
Part 1: Free C5 Concentration
Measured: Part 1: Predose (Hour 0), EOI (1 Hour), Hours 2, 6, 12 on Day 1; Days 2, 3, 4, 5, 7, 14, 21, 28, 35, 42, 56, 91
Part 2: Free C5 Concentration
Measured: Part 2: Predose (Hour 0), EOI (1 Hour), Hours 2, 6, 10-12 on Day 1; Days 2, 5, 9, 15, 29, 224; predose [Hour 0], EOI [1 Hour], Hours 10-12 on Days 8, 22; predose [Hour 0] on Day 36, 43, 50, 64, 78, 92, 106, 120, 134
Part 3: Free C5 Concentration
Measured: Part 3: Predose (Hour 0), EOI (1 Hour), Hours 2, 6 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 36, 43, 50, 57, 64, 78, 92, 106; Day 224
Part 4: Free C5 Concentration
Measured: Part 4: Predose (Hour 0), EOI (1 Hour), Hours 2, 6 on Day 1; predose (Hour 0), on Days 2, 8, 15, 22, 29, 57, 85, 113; Days 43, 134, 224
Part 2: Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Day 64
Measured: Baseline, Day 64
Part 3: Change From Baseline in Fatigue as Measured by FACIT-Fatigue Scale Score at Day 8, 22, 50, 78, 106, and 134
Measured: Baseline, Day 8, 22, 50, 78, 106, 134
Part 4: Change From Baseline in Fatigue as Measured by FACIT-Fatigue Scale Score at Day 8, 22, 57, 85, 113 and 134
Measured: Baseline, Day 8, 22, 57, 85, 113, 134
Part 2: Change From Baseline in Health-Related Quality of Life (HRQoL) as Measured by European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) Score at Day 64
Measured: Baseline, Day 64
Part 3: Change From Baseline in HRQoL as Measured by EORTC QLQ-C30 Score at Day 78 and 134
Measured: Baseline, Day 78, 134
Part 4: Change From Baseline in HRQoL as Measured by EORTC QLQC30 Score at Day 85 and 134
Measured: Baseline, Day 85, 134
Part 2: Participant Treatment Satisfaction as Measured by Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Day 8, 22, 36, 50 and 64
Measured: Baseline, Day 8, 22, 36, 50, 64
Part 3: Participant Treatment Satisfaction as Measured by TSQM Score at Day 8 and 50
Measured: Baseline, Day 8, 50
Part 4: Participant Treatment Satisfaction as Measured by TSQM Score at Day 8 and 57
Measured: Baseline, Day 8, 57
Part 2: Number of Packed Red Blood Cell (RBC) Units Transfused per Participant
Measured: Baseline up to Day 224
Part 3: Number of Packed RBCs Units Transfused per Participant
Measured: Baseline up to Day 224
Part 4: Number of Packed RBCs Units Transfused per Participant
Measured: Baseline up to Day 224
Part 2: Percentage of Participants With Packed RBC Units Transfused
Measured: Baseline up to Day 224
Part 3: Percentage of Participants With Packed RBC Units Transfused
Measured: Baseline up to Day 224
Part 4: Percentage of Participants With Packed RBC Units Transfused
Measured: Baseline up to Day 224
Part 1: Percentage of Participants With Anti-Drug Antibodies (ADAs) to Crovalimab
Measured: Part 1: Day 1 up to Day 91 (assessed at predose [Hour 0] on Day 1; on Days 14, 28, 56, 84, and 91)
Part 2: Percentage of Participants With ADAs to Crovalimab
Measured: Part 2: Day 1 up to Day 224 (assessed at predose [Hour 0] on Days 1, 8, 50, 106, 134); Days 29, 224
Part 3: Percentage of Participants With ADAs to Crovalimab
Measured: Part 3: Day 1 up to Day 106 assessed at predose [Hour 0] on Days 1, 8, 29, 64, and 106; Day 224
Part 4: Percentage of Participants With ADAs to Crovalimab
Measured: Day 1 up to Day 224 (assessed at predose [Hour 0] on Days 1, 8, 29, 113); Days 134, 224
OLE: Total C5 Concentration
Measured: OLE: Predose (Hour 0) on Week 36 up to Week 521
OLE: Serum LDH Levels
Measured: OLE: Predose (Hour 0) on Week 28 up to Week 521
OLE: Terminal Complement Activity in Serum as Assessed by Ex Vivo Liposome Immunoassay (LIA)
Measured: OLE: Week 36 up to Week 521
Part 2: Percentage of Participants With LDH Below Upper Limit of Normal (ULN)
Measured: Baseline up to Day 224
Part 3: Percentage of Participants With LDH Below ULN
Measured: Baseline up to Day 224
Part 4: Percentage of Participants With LDH Below ULN
Measured: Baseline up to Day 224
Part 2: Percentage of Participants With Complement Suppression
Measured: Baseline up to Day 134
Part 3: Percentage of Participants With Complement Suppression
Measured: Baseline up to Day 134
Part 4: Percentage of Participants With Complement Suppression
Measured: Baseline up to Day 134
Part 2: Monthly Rate of pRBC Transfusions per Participant
Measured: Baseline up to 10 years
Part 3: Monthly Rate of pRBC Transfusions per Participant
Measured: Baseline up to 10 years
Part 4: Monthly Rate of pRBC Transfusions per Participant
Measured: Baseline up to 10 years
Part 2: Proportion of Transfusion-Free Participants
Measured: Baseline up to 10 years
Part 3: Proportion of Transfusion-Free Participants
Measured: Baseline up to 10 years
Part 4: Proportion of Transfusion-Free Participants
Measured: Baseline up to 10 years
Part 2: Annual Rate of Transfusion Avoidance per Participant
Measured: Baseline up to 10 years
Part 3: Annual Rate of Transfusion Avoidance per Participant
Measured: Baseline up to 10 years
Part 4: Annual Rate of Transfusion Avoidance per Participant
Measured: Baseline up to 10 years
Part 2: Annual Rate of Breakthrough Hemolysis (BTH)
Measured: Baseline up to 10 years
Part 3: Annual Rate of Breakthrough Hemolysis (BTH)
Measured: Baseline up to 10 years
Part 4: Annual Rate of Breakthrough Hemolysis (BTH)
Measured: Baseline up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 May 2017
- Study start14 Nov 2016
- Primary completion13 Aug 2026
- Study completion13 Aug 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Hemoglobinuria, NocturnalStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Chugai Pharmaceutical
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03157635
- Sponsor's protocol ID
- BP39144
- Other IDs
- 2016-002128-10, 2023-506287-14-00, 2023, 2016
- Acronym
- COMPOSER
- Status last verified
- Aug 2026
- First submitted
- 1 Nov 2016
- Last updated
- 1 Sep 2026
- Results posted
- No
- Individual data sharing
- Yes: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/members/ourmembers/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/innovation/process/clinical -trials/data-sharing/)
Publications
Roth A, Nishimura JI, Nagy Z, Gaal-Weisinger J, Panse J, Yoon SS, Egyed M, Ichikawa S, Ito Y, Kim JS, Ninomiya H, Schrezenmeier H, Sica S, Usuki K, Sicre de Fontbrune F, Soret J, Sostelly A, Higginson J, Dieckmann A, Gentile B, Anzures-Cabrera J, Shinomiya K, Jordan G, Biedzka-Sarek M, Klughammer B, Jahreis A, Bucher C, Peffault de Latour R. The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. Blood. 2020 Mar 19;135(12):912-920. doi: 10.1182/blood.2019003399.
PubMed 31978221