Hemoglobinuria, Paroxysmal Clinical Trials

A listing of Hemoglobinuria, Paroxysmal medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 24 clinical trials
Featured trial
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused with a Reduced Dose of Non-Mobilized Donor T-Cells

Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell …

myelodysplastic syndrome (mds)
anemia studies
anemia
myelodysplastic syndromes
aplastic anemia
  • 190 views
  • 22 Dec, 2020
  • 1 location
Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

The study will evaluate the efficacy and safety of the oral Factor D (FD) inhibitor ALXN2050 (ACH-0145228) monotherapy in patients with PNH that are treatment naïve, or patients currently treated with eculizumab who still experience anemia and reticulocytosis, or patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent, …

complement inhibitor
reticulocyte count
anemia
vaccination
neutrophil count
  • 41 views
  • 17 Jun, 2022
  • 7 locations
Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

The main objective of this study is to evaluate the efficacy of danicopan as add-on therapy to a complement component 5 (C5) inhibitor (eculizumab or ravulizumab) in participants with PNH who have clinically evident EVH.

blood transfusion
anemia
hemolysis
antibiotics
danicopan
  • 1 views
  • 13 Jul, 2022
  • 69 locations
A Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors. (COMMODORE 1)

A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 250 participants.

complement inhibitor
vaccination
high sensitivity
lactate dehydrogenase
flow cytometry
  • 0 views
  • 10 Aug, 2022
  • 101 locations
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed Novartis-sponsored Phase 2 or 3 studies with iptacopan

haemophilus influenzae
neisseria meningitidis
  • 2 views
  • 20 Jun, 2022
  • 24 locations
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

(adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).

flow cytometry
complement inhibitor
platelet count
ravulizumab
anemia
  • 0 views
  • 21 Jul, 2022
  • 3 locations
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (APPOINT-PNH)

The purpose of this Phase 3 study is to determine whether iptacopan is efficacious and safe for the treatment of PNH patients who are naive to complement inhibitor therapy

flow cytometry
complement inhibitor
haemophilus influenzae
neisseria meningitidis
vaccination
  • 0 views
  • 09 Aug, 2022
  • 12 locations
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (ACCESS 2)

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 …

flow cytometry
hemolysis
fatigue
ravulizumab
lactate dehydrogenase
  • 0 views
  • 25 Mar, 2022
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy The secondary objectives of the study are: To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: …

flow cytometry
fatigue
hemolysis
eculizumab
lactate dehydrogenase
  • 4 views
  • 29 Apr, 2022
  • 1 location
Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy (ACCESS-1)

treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor

flow cytometry
complement inhibitor
fatigue
ravulizumab
hemolysis
  • 0 views
  • 26 May, 2022
  • 1 location