Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects [Phase 1]
Phase 1 trial to assess the effect of a single oral dose of MY008211A on QTc interval in healthy subjects; the study is not yet recruiting a…
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CLINICAL_TRIALS trial_result · 2024-03-26Clinical Trial Update: A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria [Phase 3]
Phase 3 trial updating results for SB12 (eculizumab biosimilar) compared to Soliris in paroxysmal nocturnal hemoglobinuria (PNH) has complet…
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CLINICAL_TRIALS trial_result · 2024-01-03Clinical Trial Update: Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 trial evaluating LNP023 (iptacopan) for safety, efficacy, tolerability, pharmacokinetics, and pharmacodynamics in patients with paro…
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CLINICAL_TRIALS trial_result · 2023-12-18Clinical Trial Update: Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
Phase 2 trial evaluating multiple doses of iptacopan (LNP023) in adults with paroxysmal nocturnal hemoglobinuria to assess efficacy, safety,…
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CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment [Phase 3]
A Phase 3 study evaluating the efficacy and safety of twice-daily oral iptacopan (LNP023) in adult Paroxysmal Nocturnal Hemoglobinuria patie…
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CLINICAL_TRIALS trial_result · 2023-11-15Clinical Trial Update: Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy [Phase 3]
Phase 3 study evaluating the efficacy and safety of twice-daily oral iptacopan (LNP023) in adults with paroxysmal nocturnal hemoglobinuria w…
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CLINICAL_TRIALS trial_result · 2023-07-24Clinical Trial Update: Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) [Phase 3]
This Phase 3 study evaluates danicopan as an add-on therapy to a C5 inhibitor in Paroxysmal Nocturnal Hemoglobinuria patients with extravasc…
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CLINICAL_TRIALS trial_result · 2023-07-12Clinical Trial Update: Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) [Phase 2]
Phase 2 trial update for Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-26Clinical Trial Update: Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 trial evaluating REGN3918 for safety and efficacy in paroxysmal nocturnal hemoglobinuria has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-12Clinical Trial Update: REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. [Phase 3]
This Phase 3 trial update for REGN3918 in paroxysmal nocturnal hemoglobinuria (PNH) was terminated, with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2023-06-05Clinical Trial Update: A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition [Phase 3]
Phase 3 trial of crovalimab in paroxysmal nocturnal hemoglobinuria patients not previously treated with complement inhibition; results of th…
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CLINICAL_TRIALS trial_result · 2023-05-23Clinical Trial Update: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH [Phase 3]
Phase 3 trial evaluating ABP 959 versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria has completed and results posted.
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CLINICAL_TRIALS trial_result · 2023-03-14Clinical Trial Update: A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 long-term study of ACH-0144471 in paroxysmal nocturnal hemoglobinuria completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2022-10-27Clinical Trial Update: Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study [Phase 2]
Extension study update for RA101495 in PNH patients who completed a prior Zilucoplan/RA101495 trial; the Phase 2 study is terminated with re…
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CLINICAL_TRIALS trial_result · 2022-10-21Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH [Phase 3]
Phase 3 trial evaluating pegcetacoplan for paroxysmal nocturnal hemoglobinuria has completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-10-20Clinical Trial Update: Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab [Phase 3]
Phase 3 trial comparing subcutaneous versus intravenous ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who are currently tre…
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CLINICAL_TRIALS trial_result · 2022-09-14Clinical Trial Update: A Study on the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of CAN106 in Subjects With PNH [Phase 1, Phase 2]
Clinical trial update on CAN106 assessing safety, tolerability, efficacy, PK, and PD in subjects with paroxysmal nocturnal hemoglobinuria (P…
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CLINICAL_TRIALS trial_result · 2022-05-09Clinical Trial Update: A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 study of Ravulizumab in children and adolescents with paroxysmal nocturnal hemoglobinuria has completed and results were posted.
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CLINICAL_TRIALS trial_result · 2022-03-25Clinical Trial Update: Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 3]
Phase 3 trial evaluating APL-2 for paroxysmal nocturnal hemoglobinuria has completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-02-17Clinical Trial Update: First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH [Phase 1, Phase 2]
First-in-human study of BCX9930 in healthy volunteers and PNH patients completed, reporting Phase 1/2 results.
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CLINICAL_TRIALS trial_result · 2021-09-01Clinical Trial Update: Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH [Phase 3]
Phase 3 trial comparing Elizaria® with Soliris® in patients with Paroxysmal Nocturnal Hemoglobinuria, assessing efficacy and safety results.
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CLINICAL_TRIALS trial_result · 2021-06-25Clinical Trial Update: Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab [Phase 2]
Phase 2 study evaluating danicopan in PNH patients with inadequate response to eculizumab has completed with posted results.
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CLINICAL_TRIALS trial_result · 2021-06-02Clinical Trial Update: Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 study of danicopan in Paroxysmal Nocturnal Hemoglobinuria completed with results posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-02-12Clinical Trial Update: Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 trial update for BCD-148 and Soliris in paroxysmal nocturnal hemoglobinuria (PNH) has completed.
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CLINICAL_TRIALS trial_result · 2021-01-11Clinical Trial Update: Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. [Phase 1]
Pilot Phase 1 trial evaluating safety, preliminary efficacy, and pharmacokinetics of subcutaneous pegylated complement inhibitor pegcetacopl…