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Found 363 clinical trials
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 with eculizumab who still experience anemia and reticulocytosis, or patients currently treated with ALXN2040 (danicopan) as monotherapy. After signing consent, …

anemia
neutrophil count
platelet count
paroxysmal nocturnal hemoglobinuria
reticulocyte count
  • 2 views
  • 31 May, 2021
  • 9 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 treatment in adult PNH patients presenting with residual anemia despite treatment with anti-C5 therapy

anemia
haemophilus influenzae
flow cytometry
eculizumab
vaccination
  • 0 views
  • 19 Sep, 2021
  • 42 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.

anemia
hemolysis
platelet count
antibiotics
blood transfusion
  • 0 views
  • 02 Jun, 2021
  • 35 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).

anemia
thrombosis
fatigue
influenza
paroxysmal nocturnal hemoglobinuria
  • 52 views
  • 24 Jan, 2021
  • 7 locations
A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT

Treatment options for older adults with Acute Myeloid Leukaemia (AML) and Myelodysplasia (MDS) are limited. Although stem cell transplantation remains one of the most effective treatments it is

remission
hla-a
busulfan
secondary acute myeloid leukemia
blast cells
  • 0 views
  • 24 Jan, 2021
  • 1 location
A Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following subjects: Subjects receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the …

luspatercept
cancer
ace-536
hysterectomy
growth factor
  • 83 views
  • 27 Jan, 2021
  • 125 locations
Matched Unrelated vs. Haploidentical Donor for Allogeneic Stem Cell Transplantation in Patients With Acute Leukemia

Primary objective of this open label, two-arm, multicenter, multinational, randomized trial is to compare anti-leukemic activity of allogeneic stem cell transplantation for patients with acute leukemia in complete remission between a 10/10 HLA matched unrelated donor and a haploidentical donor. The hypothesis: Haploidentical stem cell transplantation with post cyclophosphamide induces …

remission
myeloid leukemia
cell transplantation
cyclophosphamide
leukemia
  • 1 views
  • 03 May, 2021
  • 24 locations
Predictive Clinical and Biological Parameters in Acute Leukemia Myelodysplastic Syndromes and Myeloproliferative Disorders-HEMATO-BIO-IPC-2013-015

HEMATO-BIO-IPC-2013-015 is a monocenter prospective longitudinal study. Our aim is to define predictive clinical and biological factors in acute leukemia, myelodysplastic syndromes and myeloproliferative disorders by using genomics, genetics and epigenetics, in vitro and in vivo drug sensitivity studies,and translational immonulogy and immunomonitoring studies. HEMATO-BIO primary outcome measure is to …

remission
myeloproliferative disorder
myelodysplastic syndromes
bone marrow procedure
cancer
  • 13 views
  • 22 Jan, 2021
  • 1 location
Trial to Test the Effects of Adding 1 of 2 New Treatment Agents to Commonly Used Chemotherapy Combinations

The AML18 Trial will evaluate several relevant therapeutic questions in Acute Myeloid Leukaemia (AML), as defined by the WHO, and High Risk Myelodysplastic Syndrome. The trial is primarily designed for patients over 60 years considered fit for an intensive chemotherapeutic approach, but younger patients who may not be considered suitable …

remission
residual tumor
myelodysplastic syndrome with excess blasts-2
mylotarg
azacitidine
  • 381 views
  • 23 Jan, 2021
  • 83 locations
A Phase 1 Study of AMG 330 in Subjects With Myeloid Malignancies

The purpose of this First-in-Human Phase 1 study is to determine if AMG 330 given as a continuous IV infusion is safe and tolerable in adult subjects that have myeloid malignancies, and to determine the maximum tolerated dose and/or a biologically active dose. The study will be conducted in multiple …

cancer
refractory acute myeloid leukemia (aml)
leukemia
acute promyelocytic leukemia
  • 0 views
  • 11 Jul, 2021
  • 19 locations