PAROXYSMAL NOCTURNAL HEMOGLOBINURIA MedDRA version: 14.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Sponsors
ALEXION PHARMACEUTICALS, INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Subjects between 2-17 years; •Diagnosis of PNH; •subjects with = 5% glycosylphosphatidylinositol (GPI) deficient red blood cells or granulocytes as confirmed by flow cytometry; •Written Informed Consent from parent/guardian; •Negative pregnancy Test for Women of Child Bearing Potential; •Sexually active females must document reliable and medically approved method of birth control; •Patient must be vaccinated against N.meningitides at least 14 days prior to to study drug initiation or receive antibiotics for 14 days after the vaccination; Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: •Prior Eculizumab treatment; •Presence of suspicion of active bacterial infection; •Participation in another concurrent clinical trial; •History of meningococcal disease; •Pregnant, breast feeding or intending to conceive during study including Follow Up period. •Any other condition that can confound the outcome of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: PK/PD ;Secondary Objective: Safety and Efficacy ;Primary end point(s): To study PK/PD of Eculizumab doses in pediatric and adolescent PNH patients ;Timepoint(s) of evaluation of this end point: PK and PD samples will be collected before and after completing the administration of the investigational product at each visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety and Efficacy determination of the chosen dosing regimen based on the age of the patient;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Countries
United States
Contacts
Public ContactEuropean Clinical Trial Information
ALEXION EUROPE SAS
Outcome results
None listed