Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT05889299 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: OMS906-PNH-002
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

PNH

Outcome Measures

Primary Outcomes (1)

• To assess overall safety and tolerability of repeat-dose OMS906 5 mg/kg SC administration at 4-week intervals in patients with PNH.

  Number of participants with treatment-emergent adverse events assessed by CTCAE v5.0 and changes in laboratory measures, ECGs and physical examination.

  48 weeks

Secondary Outcomes (4)

• To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).

  48 weeks

• To assess preliminary efficacy by the effect on hemolysis and anemia measured by Lactate dehydrogenase (LDH).

  48 weeks

• To assess preliminary efficacy by the effect on hemolysis and anemia measured by red blood cell (RBC) transfusion burden.

  48 weeks

• To assess preliminary efficacy measured by PK, PD, and ADA.

  48 weeks

Study Arms (1)

OMS906 study drug

EXPERIMENTAL

OMS906 study drug repeat-dose 5 mg/kg SC administration at 4-week intervals

Drug: OMS906 study drug

Interventions

OMS906 study drug DRUG

OMS906 study drug dose repeat-dose 5mg/kg SC administration at 4-week intervals

OMS906 study drug

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.
• 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. Patients who have not received this vaccination at the time of screening may be vaccinated at any time prior to 2 weeks before the first study drug administration.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Omeros Corporation: lead

Study Sites (1)

Omeros Investigational Site

Kyiv, Ukraine

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Steve Whitaker, MD

  Omeros Corporation

  STUDY DIRECTOR

Central Study Contacts

Omeros Clinical Trial Information

CONTACT

206-676-5000; ctinfo@omeros.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2023
• First Posted: June 5, 2023
• Study Start: December 9, 2022
• Primary Completion: October 30, 2023
• Study Completion: June 30, 2024
• Last Updated: June 5, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

UA (1)