Paroxysmal Nocturnal HemoglobinuriaPhase 1NCT05889299
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
A Phase 1 study testing OMS906 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Omeros Corporation at 1 site in Ukraine.
Trial summary
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- OMS906
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUkraine
- Study dates
- 9 Dec 2022to 3 Nov 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of PNH by flow cytometry with PNH clone size of >10% RBCs and/or granulocytes.
- Male or female adults 18 years and older.
- Competent to provide consent and completed informed consent procedures.
- Patients who are not receiving complement inhibitor treatment or, alternatively, patients currently treated with eculizumab or ravulizumab with an inadequate response to treatment defined as a Hgb <10.5 g/dL. Patients receiving eculizumab or ravulizumab must be on stable doses for at least 6 months.
- Hemoglobin level <10.5 g/dL at screening and baseline.
- Lactate dehydrogenase >1.5 upper limit of normal (ULN) for patients not receiving eculizumab or ravulizumab.
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- Female patients of child-bearing potential (CBP) must have a negative serum test at screening and highly sensitive urine pregnancy test prior to each dose of OMS906.
- Females must use highly effective birth control to prevent pregnancy during the clinical trial and for 20 weeks (140 days) following their last dose of study drug.
- Males must use highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 20 weeks (140 days) following their last dose of study drug.
- Have received vaccination for Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae (if locally available). Patients who have not received these vaccinations at the time of screening may be vaccinated at any time prior to 2 weeks before the first study drug administration. Patients enrolled in the study at the time of Amendment 02 may receive S. pneumoniae and H. influenzae vaccinations, if not already vaccinated, at their next scheduled visit.
Who cannot join
- Treatment with any complement pathway inhibitor except eculizumab or ravulizumab within the 6 months prior to screening.
- For patients not receiving eculizumab or ravulizumab at the time of screening: receipt of eculizumab within 8 weeks prior to screening or receipt of ravulizumab within 24 weeks prior to screening.
- History of major organ transplant or hematopoietic stem cell/bone marrow transplant.
- Reticulocyte count <100,000 /µL, transfusion-free platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at screening.
- Anemia attributable to any other medical condition apart from PNH.
- Elevation of liver function tests, defined as total bilirubin >2×ULN, direct bilirubin >1.5xULN, and elevated transaminases, alanine aminotransaminase (ALT) or aspartate transaminase (AST), >2×ULN unless due to PNH related hemolysis.
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- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
- Significant active bacterial, fungal, or viral infection within the 2 weeks of OMS906 drug initiation, including COVID-19 infection.
- History of primary or secondary immunodeficiency or complement deficiency.
- Have human immunodeficiency virus, hepatitis B or untreated hepatitis C infection.
- History of splenectomy.
- History or prior bacterial meningitis or N. meningitidis infection.
- Patients on immunosuppressive agents such as but not limited to cyclosporine, mycophenolate mofetil (MMF), tacrolimus, cyclophosphamide, or methotrexate less than 8 weeks prior to first treatment with OMS906 unless on a stable regimen for at least 3 months prior to screening.
- Patients who require recurrent short courses of systemic corticosteroids (i.e., >4 short courses per year of >2 weeks in duration per course).
- Pregnant, planning to become pregnant, or nursing female patients.
- Recent surgery requiring general anesthesia within the 2 weeks prior to screening or expected to have surgery requiring general anesthesia during the treatment periods.
- History of any clinically significant medical, neurologic, or psychiatric disorder that in the opinion of the investigator would make the patient unsuitable for participation in the study.
- Treatment with any investigational medicinal product or investigational device within the 30 days (or within 5x its half-life in days, whichever is the longer period) prior to screening or participation in another concurrent clinical trial involving a therapeutic intervention. Participation in observational studies and/or registry studies is permitted.
- Unable or unwilling to comply with the requirements of the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1b Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Read the detailed description
This is a Phase 1b, proof of concept, open-label, uncontrolled study. The primary objective is to assess the safety and tolerability of OMS906 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). The study evaluated 3 dosing regimens: (1) 5 mg/kg SC administered every 4 weeks (Q4W), (2) 5 mg/kg IV administered once followed by administration of additional doses of 5 mg/kg IV at the occurrence of protocol-defined subclinical breakthrough hemolysis, and (3) 8 mg/kg IV every 8 weeks (Q8W) on a fixed-dosing (FD) schedule
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- OMS906
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OMS906Biological
Also called zaltenibart
Biological: OMS906
| Study group | Role | What they receive |
|---|---|---|
| OMS906 study drug administration in three phases | Experimental | 1. 5 mg/kg SC administered every 4 weeks (Q4W),
2. 5 mg/kg IV administered once followed by administration of additional doses of 5 mg/kg IV at the occurrence of protocol-defined subclinical breakthrough hemolysis, and 3.) 8 mg/kg IV every 8 weeks (Q8W) on a fixed-dosing (FD) schedule Receives: Biological: OMS906 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To Assess the Overall Safety and Tolerability of Zaltenibart (OMS906) Administration in PNH patients
Number and % of participants with Treatment-emergent Adverse Events (TEAEs) as assessed by CTCAE v5.0, including abnormalities in laboratory measures, ECGs and physical examinations.
Measured: 48 weeks
Secondary outcomes (16)
Mean Lactate Dehydrogenase (LDH) change from baseline
Mean Lactate Dehydrogenase (LDH) change from baseline (Only patients not receiving complement inhibitor treatment)
Measured: 48 weeks
Mean change of hemoglobin (Hgb)
To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).
Measured: 48 weeks
Time to subclinical breakthrough hemolysis post-treatment
Time (in days) to subclinical breakthrough hemolysis during the 5 mg/kg IV on demand period.
Measured: 48 weeks
Mean change from baseline in absolute reticulocyte count
Individual patient changes from baseline in absolute reticulocyte counts
Measured: 48 weeks
Transfusion requirements
Mean change from baseline in transfusion frequency from the 6-month period prior to the first OMS906 dose to the end of the study
Measured: -24 weeks to 48 weeks
Transfusion free
Proportion of patients who are transfusion free from Week 4 through the end of the study
Measured: Week 4 to Week 48
Proportion of breakthrough hemolysis
Proportion of patients experiencing breakthrough hemolysis during the 5 mg/kg Q4W SC treatment period.
Measured: 48 weeks
Proportion of breakthrough hemolysis
Proportion of patients experiencing breakthrough hemolysis during the 8 mg/kg Q4W IV treatment period
Measured: 48 weeks
Incidence of patients with hemoglobin increase ≥ 2.0 g/dL from baseline
Number and % of participants with hemoglobin increase ≥ 2.0 g/dL from baseline
Measured: 48 weeks
Incidence of patients with hemoglobin increase ≥ 12.0 g/dL from baseline
Number and % of participants with hemoglobin increase ≥ 12.0 g/dL from baseline
Measured: 48 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906: Cmax
Maximum concentration (Cmax) of observed OMS906 plasma concentration by OMS906 dosing regimen.
Measured: 48 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906: AUC
Area under the plasma concentration versus time curve (AUC)
Measured: 48 weeks
Free Mannan-binding lectin-associated Serine protease 3 concentration
Free MASP-3 serum concentrations by dosing regimen following the first dose and repeated doses
Measured: 48 weeks
Mature Complement Factor Concentration
Mature Complement Factor D (CFD) concentrations by dosing regimen following the first dose and repeated doses
Measured: 48 weeks
Total Mannan-Binding Lectin-Associated Serine Protease 3 (MASP-3) Concentration
Total MASP-3 serum concentrations by OMS906 dosing regimen following first dose and repeated doses
Measured: 48 weeks
OMS906 anti-drug antibodies (ADA)
Number of patients with measurable ADA
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2023
- Study start9 Dec 2022
- Primary completion3 Nov 2025
- Study completion3 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Patients will receive zaltenibart, administered as subcutaneous (SC) injections or intravenous (IV) infusions
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Omeros Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Steve Whitaker, MD, Study director, Omeros Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05889299
- Sponsor's protocol ID
- OMS906-PNH-002
- Status last verified
- May 2026
- First submitted
- 4 May 2023
- Last updated
- 11 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
- ClinicalTrials.gov ID
- NCT05889299
- Acronym / sponsor ID
- OMS906-PNH-002
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Omeros CorporationIndustry (pharma, biotech, devices)
- Study start
- 9 Dec 2022 (actual)
- Primary completion
- 3 Nov 2025 (actual)
- Study completion
- 3 Nov 2025 (actual)
- First posted
- 5 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Detailed description
This is a Phase 1b, proof of concept, open-label, uncontrolled study. The primary objective is to assess the safety and tolerability of OMS906 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). The study evaluated 3 dosing regimens: (1) 5 mg/kg SC administered every 4 weeks (Q4W), (2) 5 mg/kg IV administered once followed by administration of additional doses of 5 mg/kg IV at the occurrence of protocol-defined subclinical breakthrough hemolysis, and (3) 8 mg/kg IV every 8 weeks (Q8W) on a fixed-dosing (FD) schedule
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Patients will receive zaltenibart, administered as subcutaneous (SC) injections or intravenous (IV) infusions
| Arm | Type | Receives |
|---|---|---|
| OMS906 study drug administration in three phases | Experimental | Biological: OMS9061. 5 mg/kg SC administered every 4 weeks (Q4W), 2. 5 mg/kg IV administered once followed by administration of additional doses of 5 mg/kg IV at the occurrence of protocol-defined subclinical breakthrough hemolysis, and 3.) 8 mg/kg IV every 8 weeks (Q8W) on a fixed-dosing (FD) schedule |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Confirmed diagnosis of PNH by flow cytometry with PNH clone size of >10% RBCs and/or granulocytes.
- Male or female adults 18 years and older.
- Competent to provide consent and completed informed consent procedures.
- Patients who are not receiving complement inhibitor treatment or, alternatively, patients currently treated with eculizumab or ravulizumab with an inadequate response to treatment defined as a Hgb <10.5 g/dL. Patients receiving eculizumab or ravulizumab must be on stable doses for at least 6 months.
- Hemoglobin level <10.5 g/dL at screening and baseline.
Key exclusion criteria (19 listed)
- Treatment with any complement pathway inhibitor except eculizumab or ravulizumab within the 6 months prior to screening.
- For patients not receiving eculizumab or ravulizumab at the time of screening: receipt of eculizumab within 8 weeks prior to screening or receipt of ravulizumab within 24 weeks prior to screening.
- History of major organ transplant or hematopoietic stem cell/bone marrow transplant.
- Reticulocyte count <100,000 /µL, transfusion-free platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at screening.
- Anemia attributable to any other medical condition apart from PNH.
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 |
|---|---|---|
| OMS906Also: zaltenibartBiological: OMS906 | Biological | OMS906 study drug administration in three phases |
Primary outcome
To Assess the Overall Safety and Tolerability of Zaltenibart (OMS906) Administration in PNH patients
Time frame: 48 weeks
Number and % of participants with Treatment-emergent Adverse Events (TEAEs) as assessed by CTCAE v5.0, including abnormalities in laboratory measures, ECGs and physical examinations.
16 secondary outcomes
Mean Lactate Dehydrogenase (LDH) change from baseline
Time frame: 48 weeks
Mean change of hemoglobin (Hgb)
Time frame: 48 weeks
Time to subclinical breakthrough hemolysis post-treatment
Time frame: 48 weeks
Mean change from baseline in absolute reticulocyte count
Time frame: 48 weeks
Transfusion requirements
Time frame: -24 weeks to 48 weeks
Transfusion free
Time frame: Week 4 to Week 48
Proportion of breakthrough hemolysis
Time frame: 48 weeks
Proportion of breakthrough hemolysis
Time frame: 48 weeks
Incidence of patients with hemoglobin increase ≥ 2.0 g/dL from baseline
Time frame: 48 weeks
Incidence of patients with hemoglobin increase ≥ 12.0 g/dL from baseline
Time frame: 48 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906: Cmax
Time frame: 48 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906: AUC
Time frame: 48 weeks
Free Mannan-binding lectin-associated Serine protease 3 concentration
Time frame: 48 weeks
Mature Complement Factor Concentration
Time frame: 48 weeks
Total Mannan-Binding Lectin-Associated Serine Protease 3 (MASP-3) Concentration
Time frame: 48 weeks
OMS906 anti-drug antibodies (ADA)
Time frame: 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2026.
- Individual participant dataSharing planned: No
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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.
Steve Whitaker, MD study director
Omeros Corporation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OMS906 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Omeros Corporation at 1 site in Ukraine.
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 10 inclusion and 19 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: finished, as of the sponsor's last update on 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Ukraine. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To Assess the Overall Safety and Tolerability of Zaltenibart (OMS906) Administration in PNH patients (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 May 2026.