Clinical trial NCT06298955: Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
Official title: An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: PNH
Lead sponsor: Omeros Corporation
Sponsor class: INDUSTRY
First posted: 2024-03-07
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: To assess overall safety and tolerability of OMS906 administration at 8-week intervals in PNH patients.
timeFrame: 104 weeks
Secondary outcomes:
measure: To assess efficacy measured by hemoglobin (Hgb).
timeFrame: 6 month intervals
measure: To assess efficacy by transfusion requirements.
timeFrame: Weeks 48 and 96
measure: To assess efficacy by measurement of lactate dehydrogenase (LDH).
timeFrame: Weeks 48 and 96
measure: To assess efficacy by measurement of reticulocyte count.
timeFrame: Weeks 48 and 96
measure: To assess efficacy by measurement of clinical breakthrough hemolysis.
timeFrame: Weeks 48 and 96
measure: To assess population PK Cmax of OMS906.
timeFrame: Weeks 48 and 96
measure: To assess population PK AUC of OMS906.
timeFrame: Weeks 48 and 96
measure: To assess population PK terminal half life of OMS906.
timeFrame: Weeks 48 and 96
measure: To assess PD of OMS906
timeFrame: Weeks 48 and 96
measure: OMS906 anti-drug antibodies (ADA).
timeFrame: Weeks 24, 48, 72, and 96
measure: Assess the change in Functional Assessment of Chronic Illness Therapy (FACIT) fatigue score.
timeFrame: Weeks 24, 48, 72, and 96
Other outcomes:
Locations:
city: Aachen
country: Germany
facility: Omeros Investigational Site
city: Ulm
country: Germany
facility: Omeros Investigational Site
city: Lausanne
country: Switzerland
facility: Omeros Investigational Site
city: Kyiv
country: Ukraine
facility: Omeros Investigational Site
city: Leeds
country: United Kingdom
facility: Omeros Investigational Site
Study officials:
affiliation: Omeros Corporation
name: William Pullman
role: STUDY_DIRECTOR
References: