Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Status: Completed
• Trial end date: 2011-05-12
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals
• Treatments: Eculizumab

Criteria

Inclusion Criteria:

• Participants between 2 and 17 years of age;

• Diagnosed with PNH;

• Participants with ≥ 5% glycosylphosphatidylinositol-deficient red blood cells or granulocytes as confirmed by flow cytometry;

• Participants must have shown evidence of hemolytic anemia as documented by lactate dehydrogenase greater than the upper limit of normal or at least 1 transfusion in the past 2 years for anemia or anemia related symptoms;

• Written informed consent from a parent/guardian;

• Negative pregnancy test for females of child bearing potential at screening;

• Sexually active females must have documented a reliable and medically approved method of contraception;

• Participant must have been vaccinated against N. men, S. pneumo, and H. influ at least 14 days prior to study drug initiation or received antibiotics for 14 days after the vaccinations.

Exclusion Criteria:

• Prior eculizumab treatment;

• Presence or suspicion of active bacterial infection at baseline;

• Participation in another concurrent clinical study within at least 30 days prior to screening;

• History of meningococcal/pneumococcal/gonococcal disease;

• Pregnant, breast feeding, or intending to conceive during the study including the safety follow-up visits;

• Any other condition that could increase the participant's risk or confound the outcome of the study.