Paroxysmal Nocturnal Hemoglobinuria (PNH) Haematological Disorders
Conditions
Interventions
All patients will receive OMS906 at 5 mg/kg at intervals of every 8 weeks by intravenous infusion. Patients will be followed up at 8 weekly intervals for at least 2 years, a total of 14 visits.
Sponsors
Omeros Corporation (United States)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Have completed the last dosing visit of the prior OMS906 PNH study 2. Female patients of CBP 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 (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: 3.1. 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 3.2. Use at least 1 of the following medically acceptable methods of birth control: hormonal methods (combined estrogen-and-progestogen-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, or a vasectomized partner. 3.3. Defined as having had a hysterectomy and/or bilateral oophorectomy, bilateral salpingectomy or bilateral tubal ligation/occlusion at least 6 weeks prior to the Evaluation Period; or have a congenital or acquired condition that prevents childbearing. ** 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. 4. 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: 4.1. 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 4.2. Use (or have their partner use) acceptable highly effective contraception (see Criterion No. 3) during heterosexual activity. 5. Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Haemophilus influenza and agree to maintain vaccination throughout the study. 6. Have provided informed consent.
Exclusion criteria
Exclusion criteria: 1. Platelet count 2 × ULN, direct bilirubin > 1.5 × ULN, and elevated transaminases, ALT or AST, > 2 × 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints are safety and tolerability as assessed by AEs, vital signs, 12-lead ECGs, and clinical laboratory tests assessed from evaluation visit to end of study/termination. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Efficacy as measured by: 1.1. Proportion of patients achieving Hb = 12.0 g/dL assessed at 6-month intervals 1.2. Proportion of patients maintaining an increase in Hb = 2 g/dL, achieved in the prior study, through the duration of the long-term extension assessed at 6-month intervals 1.3. Proportion of patients who are transfusion free at Weeks 48 and 96 1.4. Mean change from baseline in transfusion frequency from the start of the long-term extension at Weeks 48 and 96 1.5. Mean LDH change from baseline, from the start of the long-term extension, at Weeks 48 and 96 1.6. Mean change in reticulocyte count from baseline, from the start of the long-term extension, at Weeks 48 and 96 1.7. Proportion of patients experiencing clinical breakthrough hemolysis at Weeks 48 and 96 2. OMS906 population PK and PD (mature CFD) parameters assessed at day 1, treatment visits and at the end of study visit. 3. Incidence of ADAs in serum at Weeks 24, 48, 72, and 96 4. Change in FACIT-fatigue at Weeks 24, 48, 72, and 96 | — |
Countries
Germany, Switzerland, Ukraine, United Kingdom
Contacts
Public ContactOmeros Clinical Trial Information
Outcome results
None listed