Hemoglobinuria, Paroxysmal Clinical Trial
Official title:
An Open-Label Multi-Center Study of Eculizumab in Children and Adolescents With a Diagnosis of Paroxysmal Nocturnal Hemoglobinuria
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.
This was an open-label, multi-center study of eculizumab administered to approximately 6 to 8 pediatric and adolescent participants aged 2 to 17 years with PNH. There were 3 periods in this study (screening, treatment, and post-treatment) with the treatment period having 2 dosing phases (induction and maintenance). If all screening criteria were met, the participant was eligible to enter the treatment period of the study after receiving Neisseria meningitidis (N. men), Streptococcus pneumoniae (S. pneumo), and Haemophilus influenzae (H. influ) vaccinations at least 14 days prior to first dose of study drug, or was vaccinated and received treatment with appropriate antibiotics until 14 days after the vaccinations. Participants received eculizumab intravenously (IV) based on their weight. Eculizumab was administered via an IV infusion at a rate of 5 to 10 milliliters (mL) per kilogram (kg) per hour (hr) (mL/kg/hr) for at least 25 minutes. The planned duration of treatment was 12 weeks with a 4-week induction phase and an 8-week maintenance phase. At the Investigator's and parents/legal guardian's discretion, participants who completed this study with eculizumab could continue treatment with commercially available eculizumab (Soliris®) and were followed in the Soliris® PNH Registry. Participants who stopped study participation before study completion or who did not continue with Soliris® treatment at the completion of the study were followed for 8 weeks and monitored for signs and symptoms of serious hemolysis. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00997386 -
Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States
|
Phase 2 | |
| Completed |
NCT00975975 -
Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer
|
Phase 2 | |
| Completed |
NCT00122330 -
Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
|
Phase 3 | |
| Completed |
NCT00721864 -
The Molecular Biology of Paroxysmal Nocturnal Hemoglobinuria (PNH)
|
N/A | |
| Completed |
NCT00152139 -
Stem Cell Transplantation for Patients With Hematologic Malignancies
|
Phase 3 | |
| Active, not recruiting |
NCT01642979 -
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria
|
Phase 2 | |
| Active, not recruiting |
NCT01760096 -
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)
|
Phase 2 | |
| Completed |
NCT00618969 -
Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors
|
Phase 2 | |
| Completed |
NCT00122304 -
Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
|
Phase 3 | |
| Completed |
NCT00098280 -
Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
|
Phase 3 | |
| Recruiting |
NCT01224483 -
Multicenter Retrospective Analysis About the Clinical Characteristics of Korean Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
|
N/A | |
| Active, not recruiting |
NCT00315419 -
Identifying Characteristics of Bone Marrow Failure Syndromes
|
N/A | |
| Approved for marketing |
NCT00438789 -
The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol
|
Phase 3 |