A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria...
Paroxysmal Nocturnal HemoglobinuriaThe purpose of this study was to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ravulizumab in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH).
Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After...
Acute Myelogenous LeukemiaAcute Lymphocytic Leukemia11 moreThe purpose of this study is to compare the effects (good and bad) of the medication basiliximab in combination with cyclosporine (investigational therapy) for the prevention of a complication of bone marrow transplantation known as graft-versus-host disease (GVHD). GVHD is a complication in which the cells of the transplanted bone marrow react against organs and tissues.
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal...
HemoglobinuriaParoxysmalThe 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.
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating...
Accelerated Phase Chronic Myelogenous LeukemiaAcute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome100 moreThis clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow failure disorders. Giving low doses of chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect)
Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients...
Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)The main purpose of this study is to evaluate the efficacy of LNP023 in patients with PNH, showing signs of active hemolysis despite treatment with SoC (defined as an antibody with anti C5 activity).
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian...
Paroxysmal Nocturnal HemoglobinuriaA safety and immunogenicity study in long-term treatment of Eculizumab (JSC "GENERIUM", Russian Federation) in patients with paroxysmal nocturnal hemoglobinuria, who have been involved earlier in the clinical trials of Eculizumab (JSC "GENERIUM", Russian Federation).
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With...
Paroxysmal Nocturnal HemoglobinuriaThe primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Paroxysmal Nocturnal Hemoglobinuria (PNH)The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.
Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients...
Paroxysmal Nocturnal Hemoglobinuria PNHTo determine whether LFG316 can induce a hematological response, as measured by reduction in hemolytic activity, in patients with PNH.
Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)...
PNHThis study evaluated the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of multiple intravenous (IV) doses of ALXN1210 administered to participants with PNH who have not previously been treated with complement inhibitor.