Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

July 14, 2026 updated by: Omeros Corporation

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
25

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Omeros Clinical Trial Information
  • Phone Number: 206-676-5000
  • Email: ctinfo@omeros.com

Study Locations

  • Germany
      • Aachen, Germany
        • Omeros Investigational Site
      • Ulm, Germany
        • Omeros Investigational Site
  • Switzerland
      • Lausanne, Switzerland
        • Omeros Investigational Site
  • Ukraine
      • Kyiv, Ukraine
        • Omeros Investigational Site
  • United Kingdom
      • Leeds, United Kingdom
        • Omeros Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  1. Have completed the last dosing visit of the prior OMS906 PNH study.
  2. Female patients of child bearing potential must have a negative result from a highly sensitive urine pregnancy test prior to each dose of OMS906.
  3. 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.
  4. 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.
  5. Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Hemophilus influenza and agree to maintain vaccination throughout the study.
  6. Have provided informed consent

Exclusion Criteria:

  1. Platelet count <30,000/µL or absolute neutrophil count <500 cells/µL at the start of the Evaluation Period.
  2. 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.
  3. History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.
  4. Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.
  5. Pregnant, planning to become pregnant, or nursing female patients.
  6. 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.
  7. Unable or unwilling to comply with the requirements of the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: OMS906 study drug
OMS906 study drug repeat-dose 5 mg/kg IV administration at 8-week intervals.
Drug: OMS906 study drug
OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
104 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
To assess efficacy measured by hemoglobin (Hgb).
Time Frame: 6 month intervals
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.
6 month intervals
To assess efficacy by transfusion requirements.
Time Frame: Weeks 48 and 96
Measure proportion of patients who are transfusion free and mean change from baseline in transfusion frequency from the start of the long-term extension.
Weeks 48 and 96
To assess efficacy by measurement of lactate dehydrogenase (LDH).
Time Frame: Weeks 48 and 96
Measure mean LDH change from baseline.
Weeks 48 and 96
To assess efficacy by measurement of reticulocyte count.
Time Frame: Weeks 48 and 96
Measure mean change in reticulocyte count from baseline.
Weeks 48 and 96
To assess efficacy by measurement of clinical breakthrough hemolysis.
Time Frame: Weeks 48 and 96
Measure proportion of patients experiencing clinical breakthrough hemolysis.
Weeks 48 and 96
To assess population PK Cmax of OMS906.
Time Frame: Weeks 48 and 96
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter maximum concentration (Cmax).
Weeks 48 and 96
To assess population PK AUC of OMS906.
Time Frame: Weeks 48 and 96
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter area under the time-concentration curve (AUC).
Weeks 48 and 96
To assess population PK terminal half life of OMS906.
Time Frame: Weeks 48 and 96
Pharmacokinetics (PK) of multiple-dose administration of OMS906 using terminal half-life parameter.
Weeks 48 and 96
To assess PD of OMS906
Time Frame: Weeks 48 and 96
PD parameters include change from baseline in mature complement factor D (FD).
Weeks 48 and 96
OMS906 anti-drug antibodies (ADA).
Time Frame: Weeks 24, 48, 72, and 96
Presence of ADA in serum will be measured.
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
To assess the effect of OMS906 on Quality of Life using the FACIT fatigue scale.
Weeks 24, 48, 72, and 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Omeros Corporation

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: William Pullman, Omeros Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 19, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 17, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 1, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 7, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 14, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • OMS906-PNH-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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