Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Interventions
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Sponsors
Alexion Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female = 18 years of age 2. Treated with eculizumab for PNH for at least 6 months prior to Day 1 3. Lactate dehydrogenase (LDH) = 1.5 x upper limit of normal (ULN) at Screening 4. PNH diagnosis confirmed by documented by high-sensitivity flow cytometry 5. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment. 6. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210 7. Willing and able to give written informed consent and comply with study visit schedule Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 172 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. History of bone marrow transplantation 2. Body weight < 40 kilograms 3. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the investigator or sponsor, would preclude participation. 4. 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) 5. Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1 6. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Efficacy of ALXN1210; Secondary Objective: Safety and tolerability of ALXN1210 Additional efficacy measures ;Primary end point(s): Hemolysis as directly measured by lactate dehydrogenase percent change (LDH-PCHG);Timepoint(s) of evaluation of this end point: Week 26 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from baseline in quality of life (QoL) as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue - Percentage of patients who achieve transfusion avoidance (TA) - Proportion of patients with stabilized hemoglobin ;Timepoint(s) of evaluation of this end point: Week 26 | — |
Countries
Germany, Netherlands, Spain, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed