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Found 21 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 …

anemia
myelodysplastic syndromes
myelodysplastic syndrome (mds)
anemia studies
aplastic anemia
  • 194 views
  • 22 Dec, 2020
  • 1 location
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

anemia
ravulizumab
eculizumab
flow cytometry
platelet count
  • 3 views
  • 04 Oct, 2022
  • 4 locations
  • 3 views
  • 19 Oct, 2022
  • 34 locations
A Phase III, Randomized, Open-Label, Active-Controlled, Multicenter Study Evaluating The Efficacy And Safety Of Crovalimab Versus Eculizumab In Patients 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

eculizumab
neisseria meningitidis
lactate dehydrogenase
ravulizumab
paroxysmal nocturnal hemoglobinuria
  • 0 views
  • 13 Oct, 2022
  • 103 locations
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

complement c5
eculizumab
platelet count
reticulocyte count
anemia
  • 41 views
  • 07 Oct, 2022
  • 8 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
haemophilus influenzae
flow cytometry
complement inhibitor
neisseria meningitidis
  • 0 views
  • 04 Oct, 2022
  • 12 locations
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Adult Patients With Complement-Mediated Renal Disease

The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with paroxysmal nocturnal

glomerular filtration rate
haemophilus influenzae
electrocardiogram
nephropathy
body mass index
  • 0 views
  • 12 Oct, 2022
  • 1 location
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)

treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy The secondary objectives of the study are to

lactate dehydrogenase
ravulizumab
eculizumab
flow cytometry
hemolysis
  • 0 views
  • 25 Mar, 2022
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

filgrastim
g-csf
immunosuppressant
granulocyte colony stimulating factor
Accepts healthy volunteers
  • 18 views
  • 04 Oct, 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

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