View clinical trials related to PNH.
Filter by:This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.
The present study is a non-interventional retrospective chart review study assessing the prevalence of PNH-clones in patients with PNH risk-factors aged ≥14 years and treated at our hospital. The objective of this study is to develop a PNH screening tool on the hospital Electronic Health Record (EHR) system. An algorithm defining PNH risk groups is developed.
The purpose of the study is to evaluate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of CAN106 administered intravenously to subjects with PNH who have not previously been treated with a complement inhibitor.
This study is a retrospective chart review study, and the main objective of this study is to identify the impact of eculizumab treatment and the improvement of complications before and after eculizumab treatment, and to evaluate the safety during the treatment and the clinical unmet needs during the treatment in PNH patients in real-world.
This is a single site, single dose escalation study in healthy subject with CAN106. The study is to assess the safety and tolerability of single escalating doses of CAN106; to characterize the PK and PD profile of CAN106; and to evaluate the immunogenicity of CAN106 injection.
This is a Phase II, single arm, open-label study to determine if treatment with eculizumab prolongs pregnancy compared to historical controls in women with preeclampsia between 23-30 weeks gestation.
This study is designed to evaluate the long-term safety of daily oral treatment with BCX9930 in subjects who have participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allows continued access to BCX9930 for enrolled subjects. The study will also evaluate the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the subject's satisfaction with the medication.
This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Patients who are positive of PNH-type cells are followed up for the percentage of PNH-type cells regularly for examining how it change.