View clinical trials related to Hemoglobinuria, Paroxysmal.
Filter by:Primary objective - The tolerability and safety of SAR443809 Secondary - The PK parameters of SAR443809 - The PD activity of SAR443809 - The immunogenicity of SAR443809
The aim of this non-interventional secondary use of data study is to evaluate hematological response in patients with paroxysmal nocturnal hemoglobinuria and anemia in the 6-month period after initiation of anti-C5 antibody treatment using real-world data obtained from multiple datasets. The results will be used to contextualize results from the APPOINT-PNH (NCT04820530) trial with iptacopan.
This was a single-center, single-dose, open-label clinical study. 12 subjects were randomly assigned in a 1:1 ratio to one of the following dosing sequences (sequence 1: AB; Sequence 2: BA). Each dosing sequence consisted of two cycles, one dose per cycle, with a 5-day washout period between doses.
This is a single-center, randomized, double-blind, placebo-controlled, dose-escalation, and multiple-dose study to evaluate safety, tolerability, PK and PD of MY008211A Tablets in healthy subjects.
The trial is the first human trial. The safety, tolerability, PK and PD of MY008211A Tablets will be evaluated in healthy subjects.
The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in healthy volunteers. The study will be conducted in 2 parts: Part 1, the single ascending dose (SAD) is the first in human (FIH) study of KP104 and Part 2, multiple ascending dose (MAD).
This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis. This observational study consists of two parts, one part is retrospective study which aims to collect medical chart data to calculate the mean change or mean incidence rates of LDH, hemoglobin, PNH-related symptoms and PNH-related events over 6 months. The other part is cross-sectional study to detect the total C5 level in PUMCH at the latest follow-up visit in eligible PNH patients with high-risk hemolysis, to show the difference between eligible PNH patients and healthy people and to explore the related clinical factor influencing high-level total C5 using logistic regression model.
The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy The secondary objectives of the study are: - To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of CH50 - To evaluate the effect of the combination treatment on the stability of LDH during the transition period from eculizumab monotherapy to combination with pozelimab and cemdisiran - To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements - To evaluate the effect of the combination treatment on hemoglobin levels - To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life (HRQoL) - To assess the concentrations of total pozelimab and eculizumab in serum; and total cemdisiran and C5 protein in plasma - To assess the immunogenicity of pozelimab and cemdisiran - To assess safety after dose intensification - To evaluate the long-term safety and efficacy of the combination treatment in an optional open-label extension period (OLEP)
The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibitor therapy.
The primary objective of the study is to evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP) The secondary objectives of the study are: - To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of total complement hemolysis activity (CH50) - To evaluate the effect of the combination treatment on hemoglobin levels - To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements - To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life - To assess the concentrations of total pozelimab in serum and total complement component (C) 5 and cemdisiran in plasma - To assess immunogenicity to pozelimab and cemdisiran - To evaluate the long-term safety and efficacy of pozelimab and cemdisiran in an optional open-label extension period (OLEP) - To assess safety after treatment intensification with pozelimab and cemdisiran