View clinical trials related to Hemoglobinuria, Paroxysmal.
Filter by:This is a 24-month multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study at the enrollment visit and followed prospectively for 24 (+/- 3) months. The scope of the study is to collect both retrospective and prospective data. The main part of the study will be prospective,collecting data on effectiveness, safety, patient- and clinician-reported outcomes and health care resource use.
The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following: - Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH. - Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials). - Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics. - Assist the PNH community with the development of recommendations and standards of care. - Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: - How effective is the pozelimab + cemdisiran combination? - What side effects may happen from taking the study drugs? - How much of each study drug is in your blood at different times? - Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
The purpose of the study is to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to study treatment, iptacopan/LNP023.
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 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 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)
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking. Researchers want to better understand long-term outcomes in people with these diseases. Objective: To follow people diagnosed with acquired or inherited bone marrow failure disease and study the long-term effects of the disease and its treatments on organ function. Eligibility: People aged 2 years and older who have been diagnosed with acquired or inherited bone marrow failure or Telomere Biology Disorder. First degree family members may also be able to take part in the study. Design: Participants will be screened with a medical history, physical exam, and blood tests. They may have a bone marrow biopsy and aspiration. For this, a large needle will be inserted in the hip through a small cut. Marrow will be drawn from the bone. A small piece of bone may be removed. Participants may also be screened with some of the following: Cheek swab or hair follicle sample Skin biopsy Urine or saliva sample Evaluation by disease specialists (e.g., lung, liver, heart) Imaging scan of the chest Liver ultrasounds Six-Minute Walk Test Lung function test Participants will be put into groups based on their disease. They will have visits every 1 to 3 years. At visits, they may repeat some screening tests. They may fill out yearly surveys about their medicines, transfusions, pregnancy, bleeding, and so on. They may have other specialized procedures, such as imaging scans and ultrasounds. Participation will last for up to 20 years.
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).