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: ALXN1210
Current Sponsor code: ALXN1210
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 4
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 or history of unexplained, recurrent infection 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;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 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 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 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 | — |
Countries
Canada, Finland, France, Germany, Korea, Republic of, Russian Federation, Spain, Sweden, Taiwan, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
ALEXION EUROPE SAS
Outcome results
None listed