OMS906 for Paroxysmal nocturnal hemoglobinuria (PNH)

Inclusion criteria

• {"criterion_text":"- Have completed the last dosing visit of the prior OMS906-PNH-001 and OMS906-PNH-002 studies. Patients who have tolerated zaltenibart well and had an adequate clinical response will be eligible.\n- Female patients of child-bearing potential (CBP) must have a negative result from a highly sensitive urine pregnancy test prior to each dose of zaltenibart.\n- 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; If a female, must be sterile (either surgically or biologically)* or at least one year postmenopausal**, or have a monogamous partner who is surgically sterile, or have a same sex partner, or if in a heterosexual relationship, must agree to comply with the following contraception guidelines: Practice abstinence (only considered a highly effective method of contraception when it is in line with the patient’s usual and preferred lifestyle and the patient agrees to refrain from heterosexual intercourse during the entire period of risk associated with the study treatments, including during the clinical trial and for 20 weeks [140 days] following their last dose of study drug), or Use at least 1 of the following medically highly effective methods of birth control: hormonal methods (combined estrogen-andprogestogen- containing hormonal contraception associated with inhibition of ovulation [oral, intravaginal, transdermal] or progestogen only hormonal contraception associated with inhibition of ovulation [oral, injectable, implantable]), intrauterine devices, intrauterine hormone-releasing systems, bilateral salpingectomy or bilateral tubal ligation/occlusion, or a vasectomized partner. * Defined as having had a hysterectomy and/or bilateral oophorectomy at least 6 weeks prior to the Evaluation Period; or have a congenital or acquired condition that prevents child-bearing. ** Defined as at least 12 months with no menses without an alternative medical cause (confirmed with follicle stimulating hormone level [FSH] in the postmenopausal range [FSH levels ≥ 40 mIU/mL during the Evaluation Period] if the patient is not using hormonal contraception or on hormonal replacement therapy). In the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.\n- 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 that include the following: Practice abstinence (only considered a highly effective method of contraception when it is in line with the patient's usual and preferred lifestyle and the patient agrees to refrain from heterosexual intercourse during the entire period of risk associated with the study treatments, including during the clinical trial and for 20 weeks [140 days] following their last dose of study drug), or Use (or have their partner use) highly effective contraception (see Criterion #3) during heterosexual activity.\n- Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Haemophilus influenzae (for S. pneumoniae and H. influenzae vacination, if locally available and/or part of standard of care) and agree to maintain vaccination throughout the study.\n- Have provided informed consent."}

Exclusion criteria

• {"criterion_text":"- Platelet count < 30,000/μL or absolute neutrophil count < 500 cells/μL at the start of the Evaluation Period.\n- Elevation of liver function tests: any single parameter of alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) must not exceed 3 × ULN\n- History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the zaltenibart preparation.\n- Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.\n- Pregnant, planning to become pregnant, or nursing female patients.\n- 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 study.\n- Unable or unwilling to comply with the requirements of the study."}

Primary endpoints

• {"endpoint_text":"- Safety and tolerability as assessed by adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory tests.","definition_or_measurement_approach":"Assessed by adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory tests as stated in the primary endpoint text."}

Secondary endpoints

• {"endpoint_text":"- Efficacy as measured by: proportion of patients achieving Hb ≥ 12.0 g/dL assessed at 6-month intervals; proportion of patients maintaining an increase in Hb ≥ 2 g/dL, achieved in the prior study; proportion of patients who are transfusion free at Weeks 48 and 96; proportion of patients experiencing clinical BTH at Weeks 48 and 96; mean LDH and absolute reticulocyte changes from from baseline, start of the long-term extension, at Weeks 48 and 96; zaltenibart PK concentrations and PD parameters","definition_or_measurement_approach":"Measures include proportions (Hb ≥ 12.0 g/dL at 6-month intervals; Hb increase ≥ 2 g/dL maintained from prior study), transfusion-free status at Weeks 48 and 96, incidence of clinical breakthrough hemolysis (BTH) at Weeks 48 and 96, mean changes in LDH and absolute reticulocyte count from baseline at Weeks 48 and 96, and zaltenibart pharmacokinetic (PK) concentrations and pharmacodynamic (PD) parameters."}

Germany

Earliest CTIS Part Ii Submission Date

09-04-2024

Latest Decision Or Authorization Date

10-04-2026

Processing Time Days

731

Number Of Sites

2

Number Of Participants

4

Primary sponsor

Full Name

Omeros Corp.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CTI Clinical Trial and Consulting Services Europe GmbH

Responsibilities

On site monitoring; sponsorDuties codes: 1,12,15,5,6,7

Name

Pra International

Responsibilities

sponsorDuties code: 4

Name

Propharma Group LLC

Responsibilities

sponsorDuties code: 8

Name

Millmount Healthcare Limited

Responsibilities

sponsorDuties code: 14

Third parties

• {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"sponsorDuties codes: 1, 12, 15 (On site monitoring), 5, 6, 7","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pra International","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"ePRO management (sponsorDuties code: 15)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Omeros Corp.","duties_or_roles":"sponsorDuties code: 10","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Long-term lab storage (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Pharmaceutical Research Associates Group B.V.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Spain","full_name":"CTI Laboratory Services Spain S.L.","duties_or_roles":"Storage (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Propharma Group LLC","duties_or_roles":"sponsorDuties code: 8","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"sponsorDuties code: 14","organisation_type":"Pharmaceutical company"}