View clinical trials related to Hemoglobinuria.
Filter by: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).
The purpose of this study is to evaluate the safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of KP104 in complement inhibitor-naïve participants with PNH. The study will be conducted in 2 parts. Part 1 is a dose-selection study to assess escalating doses and varying dose intervals of KP104. Part 2 is a proof-of-concept (POC) study assessing the efficacy of the optimal intravenous (IV) loading dose followed by the optimal maintenance dose and regimen of KP104. Participants who complete the Initial Treatment Period and demonstrate benefit from KP104 will be eligible for a 9-month open-label extension (OLE) treatment period.
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical practice.
The purpose of this registration form is to list all Managed Access Programs (MAPs) related to LNP023, Iptacopan
The study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH and how the combination compares with 2 existing treatments, one called ravulizumab and the other called eculizumab. The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug". The study is looking at several research questions, including: - How effective is the pozelimab + cemdisiran combination compared to ravulizumab? - How effective is pozelimab + cemdisiran combination compared to eculizumab? - What side effects may happen from taking the study drugs? - How much study drugs are in your blood at different times? - Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)
The primary objective of the study is: To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy The secondary objectives of the study are to: - Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following: - Transfusion requirements and transfusion parameters - Measures of hemolysis: LDH control, breakthrough hemolysis, and inhibition of CH50 - Hemoglobin levels - Fatigue as assessed by Clinical Outcome Assessments (COAs) - Health-related quality of life (HRQoL) as assessed by COAs - Safety and tolerability - To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma - To assess the immunogenicity of pozelimab and cemdisiran
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 purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult patients with PNH not currently receiving complement inhibitor therapy.
The purpose of this study is to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH patients with residual anemia despite treatment with a C5 inhibitor.