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

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

Official title:

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06298955
• Other study ID #: OMS906-PNH-003
• Status: Recruiting
• Phase: Phase 2
• Start date: February 19, 2024
• Est. completion date: April 2027

Study information

• Verified date: February 2024
• Source: Omeros Corporation
• Contact: Omeros Clinical Trial Information
• Phone: 206-676-5000
• Email: ctinfo[@]omeros.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: April 2027
• Est. primary completion date: December 2026
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• OMS906 study drug
OMS906 study drug repeat-dose 5mg/kg IV administration at 8-week intervals

Locations

Country	Name	City	State
Germany	Omeros Investigational Site	Aachen
Germany	Omeros Investigational Site	Ulm
Switzerland	Omeros Investigational Site	Lausanne
Ukraine	Omeros Investigational Site	Kyiv
United Kingdom	Omeros Investigational Site	Leeds

Sponsors (1)

Lead Sponsor	Collaborator
Omeros Corporation

Countries where clinical trial is conducted

Germany,  Switzerland,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	To assess efficacy by measurement of lactate dehydrogenase (LDH).	Measure mean LDH change from baseline.	Weeks 48 and 96
Secondary	To assess efficacy by measurement of reticulocyte count.	Measure mean change in reticulocyte count from baseline.	Weeks 48 and 96
Secondary	To assess efficacy by measurement of clinical breakthrough hemolysis.	Measure proportion of patients experiencing clinical breakthrough hemolysis.	Weeks 48 and 96
Secondary	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
Secondary	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
Secondary	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
Secondary	To assess PD of OMS906	PD parameters include change from baseline in mature complement factor D (FD).	Weeks 48 and 96
Secondary	OMS906 anti-drug antibodies (ADA).	Presence of ADA in serum will be measured.	Weeks 24, 48, 72, and 96
Secondary	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