An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)MedDRA version: 21.1Level: LLTClassification code: 10055629Term: Paroxysmal nocturnal hemoglobinuria Class: 10038359Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2023-507413-10-00
- Lead Sponsor
- Omeros Corp.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 26
Have completed the last dosing visit of the prior OMS906 PNH studies. Patients who have tolerated OMS906 well and had an adequate clinical response will be eligible., Female patients of child-bearing potential (CBP) must have a negative result from a highly sensitive urine pregnancy test prior to each dose of OMS906., 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 an acceptable method of contraception when it is in line with the patients’ 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 acceptable 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., 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 an acceptable method of contraception when it is in line with the patients’ 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) acceptable highly effective contraception (see Criterion #3) during heterosexual activity., Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Haemophilus influenza (for S. pneumoniae and H. influenzae, if locally available and/or part of standard of care) and agree to maintain vaccination throughout the study., Have provided informed consent.
Platelet count < 30,000/µL or absolute neutrophil count < 500 cells/µL at the start of the Evaluation Period., Elevation of liver function tests, defined as total bilirubin > 2 × ULN, direct bilirubin > 1.5 × ULN, and elevated transaminases, alanine aminotransferase (ALT) or aspartate aminotransferase (AST), > 2 × ULN unless due to PNH-related hemolysis., History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation., Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis., Pregnant, planning to become pregnant, or nursing female patients., 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., Unable or unwilling to comply with the requirements of the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV<br>administration at 8-week intervals in patients with PNH.;Secondary Objective: To assess long-term efficacy by the effect on hemolysis and anemia measured by hemoglobin (Hb), LDH, reticulocyte count, and red blood cell (RBC) transfusion burden; to assess the incidence of breakthrough hemolysis; to assess population pharmacokinetics (PK) and pharmacodynamics (PD); to assess anti-drug antibodies (ADAs); to assess the effect of OMS906 on Quality of Life.;Primary end point(s): Safety and tolerability as assessed by adverse events (AEs), vital signs, electrocardiograms (ECGs), and clinical laboratory tests.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):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 breakthrough hemolysis at Weeks 48 and 96; mean LDH and reticulocyte changes from from baseline, start of the long-term extension, at Weeks 48 and 96; OMS906 population PK and PD parameters