Paroxysmal Nocturnal Hemoglobinuria
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CLINICAL_TRIALS trial_result · 2025-08-20Clinical Trial Update: A Clinical Study to Evaluate the Effect of Food on the Pharmacokinetic Profiles of HRS-5965 Capsules in Healthy Subjects [Phase 1]
Phase 1 study evaluating the effect of food on the pharmacokinetics of HRS-5965 capsules in healthy subjects has completed. Conditions of in…
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CLINICAL_TRIALS trial_result · 2025-07-30Clinical Trial Update: A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
A Phase 2 clinical trial of MY008211A in adult patients with paroxysmal nocturnal hemoglobinuria has completed.
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CLINICAL_TRIALS trial_result · 2025-06-29Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH） [Phase 2]
Phase 2 clinical trial evaluating the efficacy and safety of HSK39297 in patients with paroxysmal nocturnal hemoglobinuria has completed.
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CLINICAL_TRIALS trial_result · 2025-06-25Clinical Trial Update: Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy [Phase 2]
This Phase 2 trial assesses the combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who switched from…
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CLINICAL_TRIALS trial_result · 2025-06-12Clinical Trial Update: Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
A completed clinical trial update reports real-world use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria.
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CLINICAL_TRIALS trial_result · 2025-05-29Clinical Trial Update: An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN [Phase 3]
An extension Phase 3 study of BCD-148 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who previously participated in BCD-148-2/NO…
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CLINICAL_TRIALS trial_result · 2025-05-15Clinical Trial Update: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
Post-authorization safety study evaluating iptacopan in adults with paroxysmal nocturnal hemoglobinuria using data from the IPIG PNH registr…
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CLINICAL_TRIALS trial_result · 2025-05-04Clinical Trial Update: Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms [Phase 2]
Phase 2 results for coversin in paroxysmal nocturnal hemoglobinuria patients who are resistant to eculizumab due to C5 polymorphisms; the tr…
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CLINICAL_TRIALS trial_result · 2025-04-16Clinical Trial Update: Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China [Phase 3]
This clinical trial update reports on a Phase 3 study evaluating eculizumab in adult participants with paroxysmal nocturnal hemoglobinuria i…
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CLINICAL_TRIALS trial_result · 2025-04-09Clinical Trial Update: Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 [Phase 3]
This Clinical Trials update notes completion of a Phase 3 study of rVA576 for treating paroxysmal nocturnal hemoglobinuria (PNH).
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CLINICAL_TRIALS trial_result · 2025-03-12Clinical Trial Update: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study [Phase 3]
Phase 3 long-term safety and efficacy surveillance trial of Coversin (rVA576) in paroxysmal nocturnal hemoglobinuria was terminated with res…
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CLINICAL_TRIALS trial_result · 2025-03-07Clinical Trial Update: BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy [Phase 2]
Phase 2 study of BCX9930 in Paroxysmal nocturnal hemoglobinuria patients with inadequate response to C5 inhibitors has been terminated, with…
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CLINICAL_TRIALS trial_result · 2025-03-03Clinical Trial Update: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies [NA]
A hematopoietic stem cell transplant trial for high-risk hemoglobinopathies has been terminated; the conditions studied include sickle cell …
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CLINICAL_TRIALS trial_result · 2025-02-28Clinical Trial Update: Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects [Phase 1]
Phase 1 mass-balance study of radiolabeled HSK39297 in healthy Chinese adult male volunteers has been completed, with Paroxysmal nocturnal h…
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CLINICAL_TRIALS trial_result · 2025-01-28Clinical Trial Update: A Retrospective Non-Interventional Study to Evaluate the Impact of Eculizumab in Korean PNH Patients
A retrospective non-interventional study evaluates the impact of eculizumab in Korean patients with paroxysmal nocturnal hemoglobinemia (PNH…
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CLINICAL_TRIALS trial_result · 2025-01-27Clinical Trial Update: Observational of PNH Type Cells in Korean Patients With Bone Marrow Failure Syndrome and Having Hemolytic PNH
This completed observational clinical trial from Handok Inc. investigates PNH type cells in Korean patients with bone marrow failure syndrom…
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CLINICAL_TRIALS trial_result · 2025-01-16Clinical Trial Update: Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy [Phase 2]
A Phase 2 clinical trial evaluated the combination of pozelimab and cemdisiran in adults with paroxysmal nocturnal hemoglobinuria who previo…
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CLINICAL_TRIALS trial_result · 2025-01-08Clinical Trial Update: Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 1]
This Phase 1 dose-escalation trial of BCX10013 in Paroxysmal nocturnal hemoglobinuria has completed its dose-escalation portion.
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CLINICAL_TRIALS trial_result · 2025-01-03Clinical Trial Update: BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy [Phase 2]
A Phase 2 trial evaluating BCX9930 for Paroxysmal Nocturnal Hemoglobinuria in participants not receiving other complement inhibitors was ter…
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CLINICAL_TRIALS trial_result · 2024-12-13Clinical Trial Update: A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) [Phase 2]
Phase 2 long-term safety study update for BCX9930 in participants with Paroxysmal nocturnal hemoglobinuria. The trial was terminated and res…
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CLINICAL_TRIALS trial_result · 2024-11-01Clinical Trial Update: Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
A Phase 2 proof-of-concept study evaluating the efficacy, safety, and pharmacokinetics of LFG316 in patients with Paroxysmal Nocturnal Hemog…
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CLINICAL_TRIALS trial_result · 2024-10-15Clinical Trial Update: Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 2]
This update reports the completion of a Phase 2 proof-of-concept trial evaluating the efficacy and safety of HRS-5965 in patients with parox…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria [Phase 3]
Phase 3 study evaluating the efficacy and safety of the combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab i…
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CLINICAL_TRIALS trial_result · 2024-08-12Clinical Trial Update: Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab [Phase 4]
Phase 4 study evaluating Ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who are currently on high-dose eculizumab; results p…
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy [Phase 2]
A Phase 2 study evaluating the oral Factor D inhibitor ALXN2050 as monotherapy in paroxysmal nocturnal hemoglobinuria was terminated, with r…