Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 18.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female patients = 18 years of age 2. PNH diagnosis confirmed by documented high-sensitivity flow cytometry (red blood cells [RBCs] and/or granulocytes) 3. Mean lactate dehydrogenase (LDH) =3 × upper limit of normal, based on 2 measurements from separate blood samples collected at least 1 day apart during screening 4. Willing and able to give written informed consent and comply with the study visit schedule 5. Documented meningococcal vaccination not more than 3 years prior to dosing 6. Female patients who consider themselves postmenopausal must provide evidence at screening of menopause status, based on a combination of amenorrhea for at least 1 year and increased serum follicle-stimulating hormone level (> 30 IU/L) on at least 2 occasions (eg, in the absence of hormone replacement therapy, dietary phytoestrogens) or estradiol concentration =65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: 1. Treatment with a complement inhibitor at any time 2. Platelet count < 30,000/mm3 (30 × 109 /L) at screening 3. Absolute neutrophil count < 500/µL (0.5 × 109 /L) at screening 4. History of bone marrow transplantation 5. History of Neisseria meningitidis infection; history of unexplained, recurrent infection; or infection requiring treatment with systemic antibiotics within the last 90 days prior to dosing on Day 1 6. Female patients who are planning to become pregnant, or are pregnant or breastfeeding
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy, safety, and tolerability of multiple doses of ALXN1210 administered intravenously (IV) to complement inhibitor treatment-naïve patients with PNH;Secondary Objective: The secondary objectives of the trial are: - To characterize the pharmacokinetic (PK) and pharmacodynamic (PD) effects of multiple doses of ALXN1210 administered IV to complement inhibitor treatment-naïve patients with PNH - To investigate the immunogenicity of ALXN1210 administered IV to complement inhibitor treatment-naïve patients with PNH;Primary end point(s): Change in LDH levels from baseline to Day 253;Timepoint(s) of evaluation of this end point: Day 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy endpoints: - Changes in hemolysis-related hematologic parameters - Changes in clinical manifestations Safety endpoints: - Change from baseline in the need for blood transfusions - Change from baseline in disease-associated biomarkers - Change from baseline in quality of life - Change from baseline in major adverse vascular events (MAVEs) Immunogenicity: Measurement of antidrug antibodies (ADA) Pharmacokinetic/Pharmacodynamic Safety;Timepoint(s) of evaluation of this end point: Day 1, 7, 15, 22, 29, 43, 57, 85, 113, 127, 141, 169, 197, 211, 225, 253 | — |
Countries
Canada, Finland, France, Germany, Russian Federation, Spain, Sweden, Taiwan, United Kingdom
Contacts
Public ContactEuropean Clinical Trial Information
ALEXION EUROPE SAS
Outcome results
None listed