Phase 4
Completed N=7
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Hemoglobinuria, Paroxysmal
Source: ClinicalTrials.gov NCT00867932 ↗
Enrolled (actual)
7
Serious AEs
28.6%
Results posted
Oct 2018
Primary outcomePrimary: Peak And Trough Concentrations Of Eculizumab In Serum At Week 12 — 192.5; 425.4 μg/mL
◆ Published Evidence
Established
42citations · ~4 / year
Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria.
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.
Linked Publications
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Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Peak And Trough Concentrations Of Eculizumab In Serum At Week 12 |
192.5; 425.4 | — |
| SECONDARY Number Of Participants With Treatment-emergent Adverse Events (TEAEs) |
7; 2; 4; 1; 2; 2 | — |
| SECONDARY Area Under The Curve (AUC) Of The Change From Baseline To Week 12 In Levels Of Lactate Dehydrogenase (LDH) |
-60634 | — |
| SECONDARY Concentration Of Plasma-free Hemoglobin At Baseline And Week 12 |
17.70; 7.44 | — |
| SECONDARY Change From Baseline In LDH Levels |
1020; -672; -763; -752; -761; -747 | — |
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT00867932) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.