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Hemoglobinuria clinical trials

View clinical trials related to Hemoglobinuria.

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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: NCT06412497 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Start date: June 5, 2024
Phase: Phase 2
Study type: Interventional

A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and/or clonal hematopoiesis.

NCT ID: NCT06411626 Not yet recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Home Reported Outcomes in PNH

Start date: June 15, 2024
Phase:
Study type: Observational

The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.

NCT ID: NCT06326814 Completed - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults

Start date: October 11, 2021
Phase: Phase 1
Study type: Interventional

Primary objective - The tolerability and safety of SAR443809 Secondary - The PK parameters of SAR443809 - The PD activity of SAR443809 - The immunogenicity of SAR443809

NCT ID: NCT06312644 Not yet recruiting - Pregnancy Clinical Trials

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Start date: March 29, 2024
Phase:
Study type: Observational [Patient Registry]

The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.

NCT ID: NCT06298955 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria

Start date: February 19, 2024
Phase: Phase 2
Study type: Interventional

The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.

NCT ID: NCT06294301 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Study of Single and Multiple Doses of LP-005 in Healthy Adult Participants

Start date: November 19, 2023
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate safety, tolerability, immunogenicity, pharmacokinetics, pharmacodynamics, and efficacy of LP-005 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 LP-005 and Part 2, multiple ascending dose (MAD).

NCT ID: NCT06238544 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Start date: February 6, 2024
Phase: Phase 2
Study type: Interventional

This is a multicenter, single-arm, open-label study. Patients with Paroxysmal Nocturnal Hemoglobinuria who had previously received and completed the HRS-5965 study well included. All eligible subjects received HRS-5965 tablets until the end of treatment in this study.

NCT ID: NCT06154512 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

A Real-world, Multi-center, Prospective, Observational Study for PNH in China

ReWoPNH
Start date: November 10, 2023
Phase:
Study type: Observational [Patient Registry]

As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of disease characteristics and clinical outcomes. Eculizumab was recently approved by NMPA. The availability of Eculizumab in China pro-vides people living with PNH with a new treatment option that can reduce disease symptoms and prevent the dysregulated complement system from causing further damage. A Phase Ⅳ study is necessary to understand the natural history of disease and the clinical outcomes with different medical interventions.

NCT ID: NCT06134414 Not yet recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start date: December 2024
Phase: Phase 2
Study type: Interventional

The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.