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

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: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Intervention / Treatment: Drug: OMS906 study drug

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: Interventional
• Enrollment (Estimated): 25
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Omeros Clinical Trial Information; Phone Number: 206-676-5000; Email: ctinfo@omeros.com

Study Locations

• Germany
  • Aachen, Germany
    • Not yet recruiting
    • Omeros Investigational Site
  • Ulm, Germany
    • Not yet recruiting
    • Omeros Investigational Site
• Switzerland
  • Lausanne, Switzerland
    • Recruiting
    • Omeros Investigational Site
• Ukraine
  • Kyiv, Ukraine
    • Not yet recruiting
    • Omeros Investigational Site
• United Kingdom
  • Leeds, United Kingdom
    • Not yet recruiting
    • Omeros Investigational Site

Participation Criteria

Eligibility Criteria

• 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

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

Outcome Measure	Measure Description	Time Frame
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.	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

Outcome Measure	Measure Description	Time Frame
To assess efficacy measured by hemoglobin (Hgb).	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.	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).	Measure mean LDH change from baseline.	Weeks 48 and 96
To assess efficacy by measurement of reticulocyte count.	Measure mean change in reticulocyte count from baseline.	Weeks 48 and 96
To assess efficacy by measurement of clinical breakthrough hemolysis.	Measure proportion of patients experiencing clinical breakthrough hemolysis.	Weeks 48 and 96
To assess population PK Cmax of OMS906.	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.	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.	Pharmacokinetics (PK) of multiple-dose administration of OMS906 using terminal half-life parameter.	Weeks 48 and 96
To assess PD of OMS906	PD parameters include change from baseline in mature complement factor D (FD).	Weeks 48 and 96
OMS906 anti-drug antibodies (ADA).	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.	To assess the effect of OMS906 on Quality of Life using the FACIT fatigue scale.	Weeks 24, 48, 72, and 96

Collaborators and Investigators

• Sponsor: Omeros Corporation
• Investigators: Study Director: William Pullman, Omeros Corporation

Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): April 1, 2027

Study Registration Dates

• First Submitted: February 17, 2024
• First Submitted That Met QC Criteria: March 1, 2024
• First Posted (Actual): March 7, 2024

Study Record Updates

• Last Update Posted (Actual): March 7, 2024
• Last Update Submitted That Met QC Criteria: March 1, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: PNH
• Additional Relevant MeSH Terms: Urologic Diseases; Urological Manifestations; Bone Marrow Diseases; Hematologic Diseases; Urination Disorders; Anemia; Proteinuria; Anemia, Hemolytic; Myelodysplastic Syndromes; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID 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