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PNH clinical trials

View clinical trials related to PNH.

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NCT ID: NCT06449001 Not yet recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

Start date: August 23, 2024
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

NCT ID: NCT05982938 Available - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Danicopan Early Access Program

Start date: n/a
Phase:
Study type: Expanded Access

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.

NCT ID: NCT05884060 Completed - Thrombosis Clinical Trials

Retrospective Chart Review Screening Algorithm to Assess the Prevalence of PNH-clones

Start date: April 20, 2018
Phase:
Study type: Observational

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.

NCT ID: NCT05539248 Recruiting - Hemolysis Clinical Trials

A Study on the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of CAN106 in Subjects With PNH

Start date: March 25, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

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.

NCT ID: NCT05337683 Completed - PNH Clinical Trials

A Retrospective Chart Review Study to Evaluate the Impact of Eculizumab in Korean PNH Patients

Start date: December 11, 2020
Phase:
Study type: Observational

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.

NCT ID: NCT05095168 Completed - PNH Clinical Trials

Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of a Single Ascending Dose (SAD) of CAN106 Administered Intravenously (IV) in Healthy Subjects

Start date: February 22, 2021
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT04725812 Terminated - Pregnancy Related Clinical Trials

Complement Regulation to Undo Systemic Harm in Preeclampsia

CRUSH
Start date: September 13, 2021
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT04702568 Terminated - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start date: December 18, 2020
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT03593200 Completed - PNH Clinical Trials

A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH

Start date: August 16, 2018
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT03531255 Active, not recruiting - PNH Clinical Trials

Pegcetacoplan Long Term Safety and Efficacy Extension Study

Start date: August 27, 2018
Phase: Phase 3
Study type: Interventional

This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.