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 15 clinical trials
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, …

vaccination
eculizumab
ravulizumab
complement inhibitor
reticulocyte 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.

eculizumab
ravulizumab
anemia
antibiotics
platelet count
  • 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.

vaccination
lactate dehydrogenase
eculizumab
complement inhibitor
ravulizumab
  • 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
ravulizumab
eculizumab
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

vaccination
flow cytometry
complement inhibitor
haemophilus influenzae
neisseria meningitidis
  • 0 views
  • 09 Aug, 2022
  • 12 locations
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
lactate dehydrogenase
eculizumab
hemolysis
fatigue
  • 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
ravulizumab
hemolysis
fatigue
  • 0 views
  • 26 May, 2022
  • 1 location
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Background Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems

severe aplastic anemia
anemia
Accepts healthy volunteers
neutrophil count
granulocyte colony stimulating factor
  • 18 views
  • 24 Jul, 2022
  • 1 location
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

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 …

methotrexate
cell transplantation
tacrolimus
platelet count
bone marrow failure
  • 0 views
  • 22 Jul, 2022
  • 14 locations