A Phase 1b Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Omeros Corporation1 site in 1 country10 target enrollmentDecember 9, 2022

ConditionsParoxysmal Nocturnal Hemoglobinuria

InterventionsOMS906 study drug

DrugsOMS906 study drug

Overview

Phase: Phase 1
Intervention: OMS906 study drug
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Sponsor: Omeros Corporation
Enrollment: 10
Locations: 1
Primary Endpoint: To assess overall safety and tolerability of repeat-dose OMS906 5 mg/kg SC administration at 4-week intervals in patients with PNH.
Status: Recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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).

Detailed Description

This is a Phase 1b, proof of concept, open-label, uncontrolled, fixed-dose study. The primary objective is to assess safety and tolerability of OMS906 in patients with PNH. Patients will receive 5 mg/kg OMS906 administered as subcutaneous (SC) injections at 4-week intervals.

Registry

clinicaltrials.gov

Start Date

December 9, 2022

End Date

June 30, 2024

Last Updated

2 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Omeros Corporation

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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 OMS
•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.

Exclusion Criteria

•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.

Arms & Interventions

OMS906 study drug

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

Intervention: OMS906 study drug

Outcomes

Primary Outcomes

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

Time Frame: 48 weeks

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

Secondary Outcomes

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 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 hemoglobin (Hgb).(48 weeks)
To assess preliminary efficacy measured by PK, PD, and ADA.(48 weeks)