Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Sponsors
Ra Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible for this study, subjects must meet ALL the following inclusion criteria: 1. Male or female =18 years and =65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: Subjects who meet ANY of the following exclusion criteria will be excluded from the study: 1. Platelet count 2xULN or Direct Bilirubin and Alkaline Phosphatase both >2xULN. 4. Elevation of amylase or lipase >2xULN. 5. History of meningococcal disease. 6. Current or recent systemic infection within 2 weeks prior to Screening or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Screening. 7. Pregnant, planning to become pregnant, or nursing female subjects. 8. History of bone marrow transplantation, or expected to undergo bone marrow transplantation during the 6-month treatment period. 9. Recent surgery requiring general anesthesia within 2 weeks prior to Screening or expected to have surgery requiring general anesthesia during the 6-month Treatment Period. 10. Active malignancy (except curatively resected squamous or basal cell carcinoma of the skin) requiring surgery, chemotherapy, or radiation within the prior 12 months (subjects with a history of malignancy who have undergone curative resections or otherwise not requiring a treatment for at least 12 months prior to Screening with no detectable recurrence are allowed). 11. History of any significant medical or psychiatric disorder that in the opinion of the investigator would make the subject unsuitable for participation in the study. 12. Treatment with any investigational medicinal product or investigational device within 30 days prior to Screening. 13. Participation in another concurrent clinical trial involving an investigational, therapeutic intervention (participation in observational studies and or registry studies is permitted). 14. Unable or unwilling to comply with the requirements of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To confirm the safety and tolerability of zilucoplan in subjects with PNH To confirm the efficacy of zilucoplan in subjects with PNH;Secondary Objective: Not applicable;Primary end point(s): 1.Hemoglobin stabilization defined as: maintenance of hemoglobin concentration greater than the individual set point for each subject (based on the qualifying transfusion) and avoidance of any RBC transfusion over 6 months. 2. Change in serum LDH from baseline to Week 24 (Day 168).;Timepoint(s) of evaluation of this end point: 1. 6 months 2. Day 168 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Transfusion independence: defined as subject not receiving any RBC transfusions over the 6-month study period. 2. Changes from baseline in quality of life (QOL) questionnaires (e.g., FACIT-Fatigue, EQ-5D, and WPAI:SHP).;Timepoint(s) of evaluation of this end point: 6 months | — |
Countries
Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, Finland, Georgia, Germany, Hong Kong, Italy, Korea, Republic of, Malaysia, Mexico, New Zealand, Russian Federation, Spain, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Ra Pharmaceuticals, Inc.
Outcome results
None listed