Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2021-01-08Clinical Trial Update: A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH [Phase 1]
Phase I study evaluating the safety of pegcetacoplan (APL-2) as an add-on to standard of care in paroxysmal nocturnal hemoglobinuria (PNH).
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CLINICAL_TRIALS trial_result · 2020-12-28Clinical Trial Update: A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) [Phase 3]
A Phase 3 safety and immunogenicity trial of long-term eculizumab in patients with Paroxysmal Nocturnal Hemoglobinuria has completed.
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CLINICAL_TRIALS trial_result · 2020-12-22Clinical Trial Update: A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH [Phase 2]
Phase IIa trial evaluating subcutaneous pegcetacoplan (APL-2) for safety, efficacy, and pharmacokinetics in patients with paroxysmal nocturn…
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CLINICAL_TRIALS trial_result · 2020-03-30Clinical Trial Update: A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH [Phase 1, Phase 2]
Phase 1/2 trial investigating ALN-CC5 in healthy volunteers and patients with Paroxysmal Nocturnal Hemoglobinuria (PNH); study completed wit…
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CLINICAL_TRIALS trial_result · 2020-03-10Clinical Trial Update: Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients [Phase 2]
Phase 2 trial of Zilucoplan (RA101495) for Paroxysmal Nocturnal Hemoglobinuria has completed with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2020-02-26Clinical Trial Update: Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab [Phase 2]
Phase 2 trial assessing safety and efficacy of Zilucoplan (RA101495) for PNH patients inadequately responding to eculizumab; study completed…
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CLINICAL_TRIALS trial_result · 2019-07-12Clinical Trial Update: E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 2]
Phase 2 extension study results for safety and efficacy of eculizumab in patients with paroxysmal nocturnal hemoglobinuria have been complet…
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CLINICAL_TRIALS trial_result · 2019-03-21Clinical Trial Update: ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab [Phase 3]
Phase 3 trial comparing ravulizumab (ALXN1210) with eculizumab in adults with paroxysmal nocturnal hemoglobinuria who are already receiving …
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CLINICAL_TRIALS trial_result · 2019-03-08Clinical Trial Update: Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH [Phase 4]
Phase 4 clinical trial evaluating sirolimus with low-dose warfarin for refractory paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS trial_result · 2019-02-18Clinical Trial Update: Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 open-label, multiple ascending dose study of ravulizumab (ALXN1210) in participants with paroxysmal nocturnal hemoglobinuria (PNH) h…
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CLINICAL_TRIALS trial_result · 2019-02-12Clinical Trial Update: ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
A Phase 3 trial compares ravulizumab (ALXN1210) with eculizumab in treatment-naïve adults with paroxysmal nocturnal hemoglobinuria; results …
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CLINICAL_TRIALS trial_result · 2019-01-18Clinical Trial Update: Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 study evaluated high-dose cyclophosphamide in patients with severe aplastic anemia and paroxysmal nocturnal hemoglobinuria. The tr…
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CLINICAL_TRIALS trial_result · 2018-10-31Clinical Trial Update: Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 4]
Phase 4 pharmacokinetics/pharmacodynamics study of eculizumab in pediatric/adolescent paroxysmal nocturnal hemoglobinuria has completed with…
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CLINICAL_TRIALS trial_result · 2018-03-13Clinical Trial Update: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients [Phase 2]
Phase 2 clinical trial of eculizumab in patients with paroxysmal nocturnal hemoglobinuria has completed, with results posted detailing safet…
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CLINICAL_TRIALS trial_result · 2018-01-30Clinical Trial Update: Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1, Phase 2]
A dose-escalation study of ALXN1210 IV in people with paroxysmal nocturnal hemoglobinuria (PNH) has completed Phase 1/2 with results posted.
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CLINICAL_TRIALS trial_result · 2017-10-18Clinical Trial Update: Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study
Clinical trial update indicating that a study on human anti-human antibodies in Paroxysmal nocturnal hemoglobinuria (PNH) has completed.
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
This document reports a Phase 3 clinical trial evaluating eculizumab for paroxysmal nocturnal hemoglobinuria, with the trial status listed a…
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CLINICAL_TRIALS trial_result · 2017-06-05Clinical Trial Update: Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 extension study of eculizumab in transfusion-dependent paroxysmal nocturnal hemoglobinuria has completed and posted results in 2017.
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CLINICAL_TRIALS trial_result · 2017-03-10Clinical Trial Update: Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders [Phase 2]
Phase 2 trial evaluating myeloablative conditioning with hyperfractionated total body irradiation plus thiotepa and fludarabine followed by …
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CLINICAL_TRIALS trial_result · 2016-05-03Clinical Trial Update: Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies [Phase 2]
Phase 2 clinical trial update on mismatched family member donor stem cell transplantation in children and young adults with high-risk hemato…
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CLINICAL_TRIALS trial_result · 2016-03-03Clinical Trial Update: Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors [Phase 2]
Phase 2 results from a trial of peripheral blood stem cell transplantation using haploidentical related donors were completed, led by the Un…
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CLINICAL_TRIALS trial_result · 2015-12-31Clinical Trial Update: Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome [Phase 2]
Phase 2 trial evaluating donor stem cell transplantation after a busulfan/fludarabine/methylprednisolone/antithymocyte globulin conditioning…
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CLINICAL_TRIALS trial_result · 2014-10-28Clinical Trial Update: Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease
This update summarizes a completed clinical trial investigating microvascular and cardiac dysfunction in Paroxysmal Nocturnal Hemoglobinuria…
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CLINICAL_TRIALS trial_result · 2014-05-22Clinical Trial Update: Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1]
A Phase 1 study evaluated the safety and pharmacokinetics of TT30 in patients with paroxysmal nocturnal hemoglobinuria. The trial was termin…
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CLINICAL_TRIALS trial_result · 2013-11-11Clinical Trial Update: Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes [Phase 2]
Phase 2 trial update of an allogeneic mixed chimerism stem cell transplant using Campath (alemtuzumab) for hemoglobinopathies and bone marro…