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

Phase 2

Recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: OMS906 study drug

• Registration Number: NCT06298955
• Lead Sponsor: Omeros Corporation

Brief Summary

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.

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 Timeline

• Status Verified: 2024-02
• Study First Submitted: 2024-02-17
• Study Start: 2024-02-19
• Study First Submitted QC: 2024-03-01
• Last Update Submitted: 2024-03-01
• Study First Posted: 2024-03-07
• Last Update Posted: 2024-03-07
• Primary Completion: 2026-12
• Study Completion: 2027-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 25

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.	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 Outcome Measures

Name	Time	Method
To assess efficacy measured by hemoglobin (Hgb).	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.
To assess efficacy by transfusion requirements.	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.
To assess efficacy by measurement of lactate dehydrogenase (LDH).	Weeks 48 and 96	Measure mean LDH change from baseline.
To assess efficacy by measurement of reticulocyte count.	Weeks 48 and 96	Measure mean change in reticulocyte count from baseline.
To assess efficacy by measurement of clinical breakthrough hemolysis.	Weeks 48 and 96	Measure proportion of patients experiencing clinical breakthrough hemolysis.
To assess population PK Cmax of OMS906.	Weeks 48 and 96	Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter maximum concentration (Cmax).
To assess population PK AUC of OMS906.	Weeks 48 and 96	Pharmacokinetics (PK) of multiple-dose administration of OMS906 using PK parameter area under the time-concentration curve (AUC).
To assess population PK terminal half life of OMS906.	Weeks 48 and 96	Pharmacokinetics (PK) of multiple-dose administration of OMS906 using terminal half-life parameter.
To assess PD of OMS906	Weeks 48 and 96	PD parameters include change from baseline in mature complement factor D (FD).
OMS906 anti-drug antibodies (ADA).	Weeks 24, 48, 72, and 96	Presence of ADA in serum will be measured.
Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.	Weeks 24, 48, 72, and 96	To assess the effect of OMS906 on Quality of Life using the FACIT fatigue scale.

Trial Locations

Locations (1)

Omeros Investigational Site; 🇬🇧 Leeds, United Kingdom