search
Back to results

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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Recruiting
Phase
Phase 1
Locations
Ukraine
Study Type
Interventional
Intervention
OMS906 study drug
Sponsored by
Omeros Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring PNH

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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. 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. 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. Recent surgery requiring general anesthesia within the 2 weeks prior to screening or expected to have surgery requiring general anesthesia during the Treatment Period. History of any clinically significant medical, neurologic, or psychiatric disorder that in the opinion of the investigator would make the patient unsuitable for participation in the study. Treatment with any investigational medicinal product or investigational device within the 30 days (or within 5x its half-life in days, whichever is the longer period) prior to screening or participation in another concurrent clinical trial involving a therapeutic intervention. Participation in observational studies and/or registry studies is permitted. Unable or unwilling to comply with the requirements of the study.

Sites / Locations

  • Omeros Investigational SiteRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

OMS906 study drug

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).
To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).
To assess preliminary efficacy by the effect on hemolysis and anemia measured by Lactate dehydrogenase (LDH).
To assess preliminary efficacy by the effect on hemolysis and anemia measured by Lactate dehydrogenase (LDH).
To assess preliminary efficacy by the effect on hemolysis and anemia measured by red blood cell (RBC) transfusion burden.
To assess preliminary efficacy by the effect on hemolysis and anemia measured by red blood cell (RBC) transfusion burden.
To assess preliminary efficacy measured by PK, PD, and ADA.
Pharmacokinetics (PK) of multiple-dose administration of OMS906. PK parameters including maximum concentration, area under the time-concentration curve, and terminal half-life. Pharmacodynamics (PD) of multiple-dose administration of OMS906. PD parameters including change from baseline in mature complement factor D (FD) and alternative pathway (AP) activation (ex vivo rabbit RBC lysis). Presence of anti-drug antibodies (ADA) in serum will be measured.

Full Information

First Posted
May 4, 2023
Last Updated
May 25, 2023
Sponsor
Omeros Corporation
search

1. Study Identification

Unique Protocol Identification Number
NCT05889299
Brief Title
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 9, 2022 (Actual)
Primary Completion Date
October 30, 2023 (Anticipated)
Study Completion Date
June 30, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Omeros Corporation

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
PNH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
OMS906 study drug
Arm Type
Experimental
Arm Description
OMS906 study drug repeat-dose 5 mg/kg SC administration at 4-week intervals
Intervention Type
Drug
Intervention Name(s)
OMS906 study drug
Intervention Description
OMS906 study drug dose repeat-dose 5mg/kg SC administration at 4-week intervals
Primary Outcome Measure Information:
Title
To assess overall safety and tolerability of repeat-dose OMS906 5 mg/kg SC administration at 4-week intervals in patients with PNH.
Description
Number of participants with treatment-emergent adverse events assessed by CTCAE v5.0 and changes in laboratory measures, ECGs and physical examination.
Time Frame
48 weeks
Secondary Outcome Measure Information:
Title
To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).
Description
To assess preliminary efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hgb).
Time Frame
48 weeks
Title
To assess preliminary efficacy by the effect on hemolysis and anemia measured by Lactate dehydrogenase (LDH).
Description
To assess preliminary efficacy by the effect on hemolysis and anemia measured by Lactate dehydrogenase (LDH).
Time Frame
48 weeks
Title
To assess preliminary efficacy by the effect on hemolysis and anemia measured by red blood cell (RBC) transfusion burden.
Description
To assess preliminary efficacy by the effect on hemolysis and anemia measured by red blood cell (RBC) transfusion burden.
Time Frame
48 weeks
Title
To assess preliminary efficacy measured by PK, PD, and ADA.
Description
Pharmacokinetics (PK) of multiple-dose administration of OMS906. PK parameters including maximum concentration, area under the time-concentration curve, and terminal half-life. Pharmacodynamics (PD) of multiple-dose administration of OMS906. PD parameters including change from baseline in mature complement factor D (FD) and alternative pathway (AP) activation (ex vivo rabbit RBC lysis). Presence of anti-drug antibodies (ADA) in serum will be measured.
Time Frame
48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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 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. 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. 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. Recent surgery requiring general anesthesia within the 2 weeks prior to screening or expected to have surgery requiring general anesthesia during the Treatment Period. History of any clinically significant medical, neurologic, or psychiatric disorder that in the opinion of the investigator would make the patient unsuitable for participation in the study. Treatment with any investigational medicinal product or investigational device within the 30 days (or within 5x its half-life in days, whichever is the longer period) prior to screening or participation in another concurrent clinical trial involving a therapeutic intervention. Participation in observational studies and/or registry studies is permitted. Unable or unwilling to comply with the requirements of the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Omeros Clinical Trial Information
Phone
206-676-5000
Email
ctinfo@omeros.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steve Whitaker, MD
Organizational Affiliation
Omeros Corporation
Official's Role
Study Director
Facility Information:
Facility Name
Omeros Investigational Site
City
Kyiv
Country
Ukraine
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs