Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
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|ClinicalTrials.gov Identifier: NCT00867932|
Recruitment Status : Completed
First Posted : March 24, 2009
Results First Posted : October 31, 2018
Last Update Posted : October 31, 2018
|Condition or disease||Intervention/treatment||Phase|
|Hemoglobinuria, Paroxysmal||Drug: Eculizumab||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria|
|Actual Study Start Date :||October 2, 2009|
|Actual Primary Completion Date :||May 12, 2011|
|Actual Study Completion Date :||May 12, 2011|
Eculizumab was administered as an IV infusion for 12 weeks. All participants weighed more than 45 kg and received the following weight-based dosing regimen: induction/loading = 600 milligram (mg) weekly x 4; maintenance = 900 mg at Week 5; 900 mg every 2 weeks.
5 mg/mL solution in 5% Dextrose
Other Name: Soliris®
- Peak And Trough Concentrations Of Eculizumab In Serum At Week 12 [ Time Frame: Pre-infusion and 1 hour post-infusion at End of Treatment (EOT) (Day 84 [Week 12]) or ET ]Serum concentrations of eculizumab were measured by using a validated enzyme-linked immunosorbent assay (ELISA) method developed at Alexion Pharmaceuticals Bioanalytical Laboratory. The range of the analytical assay was 10 to 600 microgram per milliliter (μg/mL). Peak concentrations were not measured at the early termination (ET) visit.
- Number Of Participants With Treatment-emergent Adverse Events (TEAEs) [ Time Frame: First dose of study drug (Day 0) to End of Follow-up (Week 20 [8 weeks after EOT]) ]A TEAE was defined as any adverse event (AE) not present prior to exposure to eculizumab or any event already present that worsened in either intensity or frequency following exposure to eculizumab. A serious TEAE was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. Related TEAEs were considered by investigators to be definitely, probably, or possibly related to administration of the study drug. Relationship is ordered as follows: unrelated, possibly related, probably related, or definitely related. TEAEs and TEAE severity were classified in accordance with the Medical Dictionary for Regulatory Activities (MedDRA) 13.0 dictionary. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
- Area Under The Curve (AUC) Of The Change From Baseline To Week 12 In Levels Of Lactate Dehydrogenase (LDH) [ Time Frame: Baseline, EOT (Day 84 [Week 12]) or ET ]The AUC of LDH was calculated by using the change of LDH from baseline values for each participant up to Week 12. For those participants with missing LDH values, the last observation carried forward method (LOCF) was used to impute missing values. Individual AUC values of LDH were summarized and tabulated.
- Concentration Of Plasma-free Hemoglobin At Baseline And Week 12 [ Time Frame: Baseline, EOT (Day 84 [Week 12]) or ET ]Plasma-free hemoglobin was determined for each participant by using standard laboratory assays. The values of plasma-free hemoglobin were summarized by visit.
- Change From Baseline In LDH Levels [ Time Frame: Baseline, Weeks 1 to 12 or ET ]Levels of LDH were determined by using standard laboratory assays. LDH values and the change of LDH from baseline were summarized by visit.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00867932
|United States, California|
|Orange, California, United States, 92868|
|United States, Florida|
|Pensacola, Florida, United States, 32504|
|United States, Tennessee|
|Memphis, Tennessee, United States, 38105|