Paroxysmal Nocturnal HemoglobinuriaPhase 2NCT06298955
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
A Phase 2 study testing OMS906 study drug 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 long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH. Read more about this study
- Who is it for?
- Ages 18 to 99 yearsHealthy volunteers can take part
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- OMS906 study drug
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 19 Feb 2024to Apr 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Have completed the last dosing visit of the prior OMS906 PNH study.
- Female patients of child bearing potential must have a negative result from a 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 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 after last dose of study drug.
- Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Hemophilus influenza and agree to maintain vaccination throughout the study.
- Have provided informed consent
Who cannot join
- Platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at the start of the Evaluation Period.
- Elevation of liver function tests, defined as total bilirubin > 2 x ULN, direct bilirubin > 1.5 x ULN, and elevated transaminases (alanine or aspartate aminotransferase), > 2 X ULN unless due to PNH-related hemolysis.
- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
- Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.
- Pregnant, planning to become pregnant, or nursing female patients.
- 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 long-term extension.
- 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: An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
Read the detailed description
This is a multicenter, open-label, single arm study. The primary objective is to assess the long-term safety and tolerability of OMS906 in patients with PNH. Secondary objectives of this study include assessment of the long-term efficacy of OMS906 in patients with PNH. Patients will receive OMS906 5 mg/kg administered as intravenous (IV) injections at 8-week intervals.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- OMS906 study drug
- 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.
OMS906 study drugDrug
OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals
| Study group | Role | What they receive |
|---|---|---|
| OMS906 study drug | Experimental | OMS906 study drug repeat-dose 5 mg/kg IV administration at 8-week intervals. Receives: Drug: OMS906 study drug |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
Treatment-emergent adverse events, including clinically significant clinical laboratory tests, 12-lead electrocardiograms, vital signs, and physical examinations recorded as an adverse event.
Measured: 104 weeks
Secondary outcomes (11)
To assess efficacy measured by hemoglobin (Hgb).
Measured by patients achieving Hb ≥ 12.0 g/dL and by proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study, through the duration of the long-term extension.
Measured: 6 month intervals
To assess efficacy by transfusion requirements.
Measure proportion of patients who are transfusion free and mean change from baseline in transfusion frequency from the start of the long-term extension.
Measured: Weeks 48 and 96
To assess efficacy by measurement of lactate dehydrogenase (LDH).
Measure mean LDH change from baseline.
Measured: Weeks 48 and 96
To assess efficacy by measurement of reticulocyte count.
Measure mean change in reticulocyte count from baseline.
Measured: Weeks 48 and 96
To assess efficacy by measurement of clinical breakthrough hemolysis.
Measure proportion of patients experiencing clinical breakthrough hemolysis.
Measured: Weeks 48 and 96
To assess population PK Cmax of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter maximum concentration (Cmax).
Measured: Weeks 48 and 96
To assess population PK AUC of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter area under the time-concentration curve (AUC).
Measured: Weeks 48 and 96
To assess population PK terminal half life of OMS906.
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using terminal half-life parameter.
Measured: Weeks 48 and 96
To assess PD of OMS906
PD parameters include change from baseline in mature complement factor D (FD).
Measured: Weeks 48 and 96
OMS906 anti-drug antibodies (ADA).
Presence of ADA in serum will be measured.
Measured: Weeks 24, 48, 72, and 96
Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.
To assess the effect of OMS906 on Quality of Life using the FACIT fatigue scale.
Measured: Weeks 24, 48, 72, and 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start19 Feb 2024
- Primary completionDec 2026 (estimated)
- Study completionApr 2027 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Omeros Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- William Pullman, Study director, Omeros Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06298955
- Sponsor's protocol ID
- OMS906-PNH-003
- Status last verified
- Jul 2026
- First submitted
- 17 Feb 2024
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
- ClinicalTrials.gov ID
- NCT06298955
- Acronym / sponsor ID
- OMS906-PNH-003
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Omeros CorporationIndustry (pharma, biotech, devices)
- Study start
- 19 Feb 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 7 Mar 2024 (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 long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
Detailed description
This is a multicenter, open-label, single arm study. The primary objective is to assess the long-term safety and tolerability of OMS906 in patients with PNH. Secondary objectives of this study include assessment of the long-term efficacy of OMS906 in patients with PNH. Patients will receive OMS906 5 mg/kg administered as intravenous (IV) injections at 8-week intervals.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OMS906 study drug | Experimental | Drug: OMS906 study drugOMS906 study drug repeat-dose 5 mg/kg IV administration at 8-week intervals. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Have completed the last dosing visit of the prior OMS906 PNH study.
- Female patients of child bearing potential must have a negative result from a 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 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 after last dose of study drug.
- Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Hemophilus influenza and agree to maintain vaccination throughout the study.
Key exclusion criteria (7 listed)
- Platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at the start of the Evaluation Period.
- Elevation of liver function tests, defined as total bilirubin > 2 x ULN, direct bilirubin > 1.5 x ULN, and elevated transaminases (alanine or aspartate aminotransferase), > 2 X ULN unless due to PNH-related hemolysis.
- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
- Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.
- Pregnant, planning to become pregnant, or nursing female patients.
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 |
|---|---|---|
| OMS906 study drugOMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals | Drug | OMS906 study drug |
Primary outcome
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
Time frame: 104 weeks
Treatment-emergent adverse events, including clinically significant clinical laboratory tests, 12-lead electrocardiograms, vital signs, and physical examinations recorded as an adverse event.
11 secondary outcomes
To assess efficacy measured by hemoglobin (Hgb).
Time frame: 6 month intervals
To assess efficacy by transfusion requirements.
Time frame: Weeks 48 and 96
To assess efficacy by measurement of lactate dehydrogenase (LDH).
Time frame: Weeks 48 and 96
To assess efficacy by measurement of reticulocyte count.
Time frame: Weeks 48 and 96
To assess efficacy by measurement of clinical breakthrough hemolysis.
Time frame: Weeks 48 and 96
To assess population PK Cmax of OMS906.
Time frame: Weeks 48 and 96
To assess population PK AUC of OMS906.
Time frame: Weeks 48 and 96
To assess population PK terminal half life of OMS906.
Time frame: Weeks 48 and 96
To assess PD of OMS906
Time frame: Weeks 48 and 96
OMS906 anti-drug antibodies (ADA).
Time frame: Weeks 24, 48, 72, and 96
Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.
Time frame: Weeks 24, 48, 72, and 96
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2026.
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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.
William Pullman 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 study drug 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 6 inclusion and 7 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 16 Jul 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 Germany, Switzerland, Ukraine, 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?
This study is still under way. Results are not available in the source record yet.
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 overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients. (time frame: 104 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 16 Jul 2026.