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 22 clinical trials
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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
  • 86 views
  • 22 Dec, 2020
  • 1 location
Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

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 nave, or patients currently treated

vaccination
ravulizumab
reticulocyte count
complement c5
anemia
  • 2 views
  • 31 May, 2021
  • 9 locations
A Phase 3 Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The purpose of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and efficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).

vaccination
anemia
haemophilus influenzae
influenza
blood transfusion
  • 52 views
  • 24 Jan, 2021
  • 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

ravulizumab
anemia
platelet count
hemolysis
blood transfusion
  • 0 views
  • 02 Jun, 2021
  • 35 locations
Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

The purpose of this Phase 3 study is to determine whether LNP023 is efficacious and safe for the treatment in PNH through demonstration of superiority of LNP023 compared to anti-C5 antibody

vaccination
neisseria meningitidis
ravulizumab
anemia
haemophilus influenzae
  • 0 views
  • 21 Jul, 2021
  • 35 locations
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

neutrophil count
ravulizumab
complement inhibitor
anemia
platelet count
  • 0 views
  • 03 Jun, 2021
  • 1 location
Study of Safety Efficacy Tolerability Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The main purpose of this study is to evaluate the efficacy of LNP023 in patients with PNH, showing signs of active hemolysis despite treatment with SoC (defined as an antibody with anti C5

  • 270 views
  • 31 Mar, 2021
  • 4 locations
Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).

neisseria meningitidis
ravulizumab
eculizumab
flow cytometry
  • 0 views
  • 15 Jun, 2021
  • 2 locations
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

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
neisseria meningitidis
complement inhibitor
haemophilus influenzae
flow cytometry
  • 0 views
  • 29 Jul, 2021
  • 2 locations
Unrelated And Partially Matched Related Donor PSCT w/ TCR Depletion for Patients With BMF

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.

thrombocytopenia
bone marrow failure
anemia
shwachman-diamond syndrome
antibiotics
  • 17 views
  • 25 Jan, 2021
  • 1 location