Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT05972967
Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab
A Phase 2 study testing OMS906 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Omeros Corporation at 5 sites in 4 countries.
Trial summary
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of OMS906 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who have a sub-optimal response to ravulizumab. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- 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
- 12 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 27 Mar 2023to 10 Oct 2024
- Phase
- Phase 2
- 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 to 99 years
- 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% red blood cells (RBCs) and/or granulocytes.
- Male or female adults 18 years and older.
- Completed informed consent procedures.
- In relation to ravulizumab treatment prescribed in accordance with its marketing authorization and summary of product characteristics (SmPC):
- Must have a sub-optimal response to ravulizumab, defined as a hemoglobin level < 10.5 g/dL despite treatment measured at screening and confirmed at baseline (Day 1, predose). Ravulizumab treatment will have been maintained at a stable dose for at least 2 doses (4 months) prior to baseline.
- Female patients of child-bearing potential must have a negative highly sensitive urine pregnancy test at screening and prior to each dose of OMS906.
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- 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. If a female, must be sterile (either surgically or biologically)* or at least one year postmenopausal**, or have a monogamous partner who is surgically sterile, or have a same sex partner, or if in a heterosexual relationship, must agree to comply with the following contraception guidelines:
- Practice abstinence (only considered an acceptable method of contraception when it is in line with the patients' usual and preferred lifestyle and the patient agrees to refrain from heterosexual intercourse during the entire period of risk associated with the study treatments, including during the clinical trial and for 20 weeks [140 days] following their last dose of study drug), or
- Use at least 1 of the following medically acceptable methods of birth control:
- Hormonal methods as follows:
- Combined estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal). Progestogen only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
- Intrauterine devices
- Intrauterine hormone-releasing systems
- Vasectomized partner * Defined as having had a hysterectomy and/or bilateral oophorectomy, bilateral salpingectomy or bilateral tubal ligation/occlusion at least 6 weeks prior to screening; or have a congenital or acquired condition that prevents childbearing.
- Defined as at least 12 months with no menses without an alternative medical cause) [can be confirmed with follicle stimulating hormone level (FSH) in the postmenopausal range (FSH levels ≥40 milli-International unit (mIU)/mL at Screening) if the patient is not using hormonal contraception or on hormonal replacement therapy]. In the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
- 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 that include the following:
- Practice abstinence (only considered an acceptable method of contraception when it is in line with the patients' usual and preferred lifestyle and the patient agrees to refrain from heterosexual intercourse during the entire period of risk associated with the study treatments, including during the clinical trial and for 20 weeks [140 days] following their last dose of study drug), or
- Use (or have their partner use) acceptable, highly effective contraception (see Criterion #6) during heterosexual activity.
Who cannot join
- History of major organ transplant or hematopoietic stem cell/marrow transplant.
- Platelet count < 30,000/µL or absolute neutrophil count < 500 cells/µL at Screening.
- Anemia, as evidenced by hemoglobin < 10.5 g/dL, attributable to any other medical condition apart from PNH.
- Elevation of liver function tests, defined as total bilirubin > 2×ULN, direct bilirubin > 1.5× upper limit of normal (ULN), and elevated transaminases, alanine aminotransferase (ALT) or aspartate aminotransferase (AST), > 2×ULN unless due to PNH related hemolysis.
- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
- Significant active bacterial or viral infection within the 2 weeks prior to Screening, including COVID-19 infection.
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- Immunodeficiency or immunosuppression (including chronic use of immunosuppressive drugs, such as ciclosporin or tacrolimus).
- History of meningococcal disease and/or has not received vaccination for N. meningitidis.
- 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 Period.
- History of any 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 30 days (or within 5× 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 and/or registry studies is permitted.
- Unable or unwilling to comply with the requirements of the study.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
Germany2 sites
Omeros Investigational Site
Aachen
Omeros Investigational Site
Ulm
About this study
Official title: A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of OMS906 in PNH Patients With a Sub-optimal Response to the C5 Inhibitor, Ravulizumab
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of OMS906 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who have a sub-optimal response to ravulizumab.
Read the detailed description
This is a Phase 2, proof of concept, open label study examining two doses of OMS906, 3 mg/kg and 5 mg/kg IV given to PNH patients at 8-week intervals, first in combination with the C5 inhibitor ravulizumab then as monotherapy. The primary objective is to assess overall safety and tolerability of repeat-dose IV OMS906 administration at 8-week intervals in patients with PNH.
- 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 |
|---|---|---|
| 3 mg/kg IV OMS906 with Ravulizumab IV | Experimental | Up to 6 doses of 3 mg/kg at 8-week intervals. All patients will receive 3 doses of OMS906 of 3 mg/kg Intravenous (IV) at 8-week intervals. Clinical responders at Week 24 will receive an additional 3 doses of OMS906 only at 8-week intervals at 5 mg/kg (monotherapy). Incomplete responders may receive an additional 3 doses of OMS906 with ravulizumab at 8-week intervals. Non responders will not receive additional OMS906. Receives: Biological: OMS906 |
| 5 mg/kg IV OMS906 with Ravulizumab IV | Experimental | Up to 6 doses of 5 mg/kg at 8-week intervals. All patients will receive 3 doses of OMS906 of 5 mg/kg Intravenous (IV) at 8-week intervals. Clinical responders at Week 24 will receive an additional 3 doses of OMS906 only at 8-week intervals (monotherapy). Incomplete responders may receive an additional 3 doses of OMS906 with ravulizumab at 8-week intervals. Non responders will not receive additional OMS906. Receives: Biological: OMS906 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the overall OMS906 administration at 8-week intervals 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: 56 weeks
Secondary outcomes (7)
Incidence of patients with hemoglobin increase ≥ 2.0 g/dL from baseline (Response criterion) baseline on adjunctive treatment and sustained during monotherapy.
Number and % of participants with hemoglobin increase ≥ 2.0 g/dL from baseline on adjunctive treatment and sustained during monotherapy
Measured: 56 weeks
Reticulocyte count
Individual patient changes from baseline in absolute reticulocyte counts
Measured: 56 weeks
Lactate dehydrogenase (LDH)
Individual patient changes from baseline in LDH
Measured: 56 weeks
Transfusion requirements
Number of transfusions required during each treatment phase will be compared with the transfusion history prior to the study
Measured: 56 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906
PK parameters including OMS906 maximum concentration (Cmax) and Area under the plasma concentration versus time curve (AUC)
Measured: 56 weeks
Pharmacodynamics (PD) of multiple-dose administration of OMS906
Mature Complement Factor D (CFD) concentration
Measured: 56 weeks
OMS906 anti-drug antibodies (ADA)
Number of patients with measurable ADA
Measured: 56 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2023
- Study start27 Mar 2023
- Primary completion10 Oct 2024
- Study completion10 Oct 2024
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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Omeros Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Edward Philpot, MD, Study director, Omeros Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05972967
- Sponsor's protocol ID
- OMS906-PNH-001
- Status last verified
- Sep 2025
- First submitted
- 11 Jul 2023
- Last updated
- 1 Oct 2025
- 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 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of OMS906 in PNH Patients With a Sub-optimal Response to the C5 Inhibitor, Ravulizumab
- ClinicalTrials.gov ID
- NCT05972967
- Acronym / sponsor ID
- OMS906-PNH-001
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Omeros CorporationIndustry (pharma, biotech, devices)
- Study start
- 27 Mar 2023 (actual)
- Primary completion
- 10 Oct 2024 (actual)
- Study completion
- 10 Oct 2024 (actual)
- First posted
- 2 Aug 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 of OMS906 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who have a sub-optimal response to ravulizumab.
Detailed description
This is a Phase 2, proof of concept, open label study examining two doses of OMS906, 3 mg/kg and 5 mg/kg IV given to PNH patients at 8-week intervals, first in combination with the C5 inhibitor ravulizumab then as monotherapy. The primary objective is to assess overall safety and tolerability of repeat-dose IV OMS906 administration at 8-week intervals in patients with PNH.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 3 mg/kg IV OMS906 with Ravulizumab IV | Experimental | Biological: OMS906Up to 6 doses of 3 mg/kg at 8-week intervals. All patients will receive 3 doses of OMS906 of 3 mg/kg Intravenous (IV) at 8-week intervals. Clinical responders at Week 24 will receive an additional 3 doses of OMS906 only at 8-week intervals at 5 mg/kg (monotherapy). Incomplete responders may receive an additional 3 doses of OMS906 with ravulizumab at 8-week intervals. Non responders will not receive additional OMS906. |
| 5 mg/kg IV OMS906 with Ravulizumab IV | Experimental | Biological: OMS906Up to 6 doses of 5 mg/kg at 8-week intervals. All patients will receive 3 doses of OMS906 of 5 mg/kg Intravenous (IV) at 8-week intervals. Clinical responders at Week 24 will receive an additional 3 doses of OMS906 only at 8-week intervals (monotherapy). Incomplete responders may receive an additional 3 doses of OMS906 with ravulizumab at 8-week intervals. Non responders will not receive additional OMS906. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Confirmed diagnosis of PNH by flow cytometry with PNH clone size of > 10% red blood cells (RBCs) and/or granulocytes.
- Male or female adults 18 years and older.
- Completed informed consent procedures.
- In relation to ravulizumab treatment prescribed in accordance with its marketing authorization and summary of product characteristics (SmPC):
- Female patients of child-bearing potential must have a negative highly sensitive urine pregnancy test at screening and prior to each dose of OMS906.
Key exclusion criteria (13 listed)
- History of major organ transplant or hematopoietic stem cell/marrow transplant.
- Platelet count < 30,000/µL or absolute neutrophil count < 500 cells/µL at Screening.
- Anemia, as evidenced by hemoglobin < 10.5 g/dL, attributable to any other medical condition apart from PNH.
- Elevation of liver function tests, defined as total bilirubin > 2×ULN, direct bilirubin > 1.5× upper limit of normal (ULN), and elevated transaminases, alanine aminotransferase (ALT) or aspartate aminotransferase (AST), > 2×ULN unless due to PNH related hemolysis.
- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
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 | 3 mg/kg IV OMS906 with Ravulizumab IV, 5 mg/kg IV OMS906 with Ravulizumab IV |
Primary outcome
To assess the overall OMS906 administration at 8-week intervals in PNH patients.
Time frame: 56 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
7 secondary outcomes
Incidence of patients with hemoglobin increase ≥ 2.0 g/dL from baseline (Response criterion) baseline on adjunctive treatment and sustained during monotherapy.
Time frame: 56 weeks
Reticulocyte count
Time frame: 56 weeks
Lactate dehydrogenase (LDH)
Time frame: 56 weeks
Transfusion requirements
Time frame: 56 weeks
Pharmacokinetics (PK) of multiple-dose administration of OMS906
Time frame: 56 weeks
Pharmacodynamics (PD) of multiple-dose administration of OMS906
Time frame: 56 weeks
OMS906 anti-drug antibodies (ADA)
Time frame: 56 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 1 Oct 2025.
- Individual participant dataSharing planned: No
Related trials for Paroxysmal Nocturnal Hemoglobinuria
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Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
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RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
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Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
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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.
Edward Philpot, MD study director
Omeros Corporation
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OMS906 in people with Paroxysmal Nocturnal Hemoglobinuria, run by Omeros Corporation at 5 sites in 4 countries.
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 18 inclusion and 13 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 1 Oct 2025. 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 Germany, Greece, Switzerland, United Kingdom. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 OMS906 administration at 8-week intervals in PNH patients. (time frame: 56 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 1 Oct 2025.