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NCT Number: NCT06298955

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes

Age range

18 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Omeros Investigational Site, Aachen, Germany

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About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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.

Treatment and study plan

OMS906 study drug

Drug

OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. To assess efficacy by measurement of lactate dehydrogenase (LDH).

    Time frame: Weeks 48 and 96

    Measure mean LDH change from baseline.

  4. To assess efficacy by measurement of reticulocyte count.

    Time frame: Weeks 48 and 96

    Measure mean change in reticulocyte count from baseline.

  5. To assess efficacy by measurement of clinical breakthrough hemolysis.

    Time frame: Weeks 48 and 96

    Measure proportion of patients experiencing clinical breakthrough hemolysis.

  6. 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).

  7. 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).

  8. 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.

  9. To assess PD of OMS906

    Time frame: Weeks 48 and 96

    PD parameters include change from baseline in mature complement factor D (FD).

  10. OMS906 anti-drug antibodies (ADA).

    Time frame: Weeks 24, 48, 72, and 96

    Presence of ADA in serum will be measured.

  11. 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.

Sponsors and collaborators

Lead sponsor

Omeros Corporation

Industry

Registry information

Official study title

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Important dates

Study start
2024
Primary completion
2026
Study completion
2027
First posted
Mar 7, 2024
Registry last updated
Jul 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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