Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria
2 other identifiers
interventional
7
1 country
3
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Oct 2009
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 23, 2009
CompletedFirst Posted
Study publicly available on registry
March 24, 2009
CompletedStudy Start
First participant enrolled
October 2, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 12, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
May 12, 2011
CompletedResults Posted
Study results publicly available
October 31, 2018
CompletedOctober 31, 2018
October 1, 2018
1.6 years
March 23, 2009
October 3, 2018
October 3, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Peak And Trough Concentrations Of Eculizumab In Serum At Week 12
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.
Pre-infusion and 1 hour post-infusion at End of Treatment (EOT) (Day 84 [Week 12]) or ET
Secondary Outcomes (4)
Number Of Participants With Treatment-emergent Adverse Events (TEAEs)
First dose of study drug (Day 0) to End of Follow-up (Week 20 [8 weeks after EOT])
Area Under The Curve (AUC) Of The Change From Baseline To Week 12 In Levels Of Lactate Dehydrogenase (LDH)
Baseline, EOT (Day 84 [Week 12]) or ET
Concentration Of Plasma-free Hemoglobin At Baseline And Week 12
Baseline, EOT (Day 84 [Week 12]) or ET
Change From Baseline In LDH Levels
Baseline, Weeks 1 to 12 or ET
Study Arms (1)
Eculizumab
EXPERIMENTALEculizumab 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (3)
Unknown Facility
Orange, California, 92868, United States
Unknown Facility
Pensacola, Florida, 32504, United States
Unknown Facility
Memphis, Tennessee, 38105, United States
Related Publications (1)
Reiss UM, Schwartz J, Sakamoto KM, Puthenveetil G, Ogawa M, Bedrosian CL, Ware RE. Efficacy and safety of eculizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria. Pediatr Blood Cancer. 2014 Sep;61(9):1544-50. doi: 10.1002/pbc.25068. Epub 2014 Apr 29.
PMID: 24777716BACKGROUND
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Alexion Pharmaceuticals Inc.
- Organization
- Alexion Pharmaceuticals Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 23, 2009
First Posted
March 24, 2009
Study Start
October 2, 2009
Primary Completion
May 12, 2011
Study Completion
May 12, 2011
Last Updated
October 31, 2018
Results First Posted
October 31, 2018
Record last verified: 2018-10