Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Interventions
Trade Name: Ultomiris
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Current Spons
Sponsors
Alexion Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female = 18 years of age 2. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry 3. Presence of 1 or more of the following PNH-related signs or symptoms within 3 months of Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath (dyspnea), anemia (hemoglobin =65 years) yes F.1.3.1 Number of subjects for this age range 31
Exclusion criteria
Exclusion criteria: 1. Treatment with a complement inhibitor at any time 2. History of bone marrow transplantation 3. Body weight < 40 kilograms 4. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1 5. Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater. 6. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation 7. Unstable medical conditions (eg, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The purpose of this study is to assess ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor.;Secondary Objective: - safety and tolerability of ALXN1210 - efficacy - PK/PD and immunogenicity - long-term safety and efficacy - evaluate the safety and efficacy in patients who switch from eculizumab to ALXN1210 in the Extension Period - quantify identified specific safety concerns during treatment with ALXN1210, including meningococcal infections, serious hemolysis after drug discontinuation in PNH, immunogenicity, serious infections, malignancies and hematologic abnormalities, and during pregnancy and breastfeeding. Roll-over PNH Patients From Other Ongoing Studies of ALXN1210 IV: - Long-term safety of patients receiving ALXN1210 after roll over into Study ALXN1210-PNH-301;Primary end point(s): - Percentage of patients who achieve transfusion avoidance (TA) - Normalization of lactate dehydrogenase (LDH) levels ;Timepoint(s) of evaluation of this end point: 26 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Percentage change from baseline in lactate dehydrogenase ( LDH) levels - Change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue - Percentage of patients with breakthrough hemolysis - Percentage of patients with stabilized hemoglobin Roll-over Cohort: -evaluate the long-term safety;Timepoint(s) of evaluation of this end point: 26 weeks Roll-over Cohort: -by incidence of treatment-emergent adverse events and SAEs, laboratory assessments, and proportion of patients who develop antidrug antibodies (ADAs) | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, Colombia, Czechia, Czech Republic, Denmark, Estonia, Finland, France, Germany, Italy, Japan, Korea, Democratic People's Republic of, Malaysia, Mexico, Netherlands, Poland, Portugal, Russian Federation, Singapore, Spain, Sweden, Taiwan, Thailand, Turkey, United Kingdom, United States Minor Outlying Islands
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed