Records containing: Paroxysmal Nocturnal Hemoglobinuria (PNH)

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CLINICAL_TRIALS study record update · 2026-04-24
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
CLINICAL_TRIALS study record update · 2026-04-22
A Phase 1, Randomized, Double Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Participants Followed by a Phase 2a Open Label Study in Participants With PNH and Residual Anemia to Evaluate the Safety, Tolerability, PK and PD of ADX-038
CLINICAL_TRIALS study record update · 2026-04-16
An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to <18 Years of Age
CLINICAL_TRIALS study record update · 2026-04-16
Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
CLINICAL_TRIALS study record update · 2026-04-16
REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
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CLINICAL_TRIALS study record update · 2026-03-17
A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy
CLINICAL_TRIALS study record update · 2026-02-04
A Phase II Clinical Study Evaluating SLN12140 in Complement Inhibitor-Naïve Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria
CLINICAL_TRIALS study record update · 2025-12-15
To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of LP-005 in Healthy Volunteers
CLINICAL_TRIALS study record update · 2025-12-05
Managed Access Programs for LNP023, Iptacopan
CLINICAL_TRIALS study record update · 2025-09-03
A Single-dose, Open-label, Phase I Study Comparing the Pharmacokinetics, Safety, and Pharmacodynamics of HSK39297 in Subjects With Mild and Moderate Hepatic Impairment and Normal Hepatic Function
CLINICAL_TRIALS study record update · 2025-08-06
A Prospective Study of the Spectrum of Haematological Disorders in Patients With Extrahepatic Portal Vein Obstruction
CLINICAL_TRIALS study record update · 2025-07-29
A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis
CLINICAL_TRIALS study record update · 2025-06-29
A Multicenter, Randomized, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）
CLINICAL_TRIALS study record update · 2025-06-11
An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria
CLINICAL_TRIALS study record update · 2025-05-29
An Extension Clinical Study of BCD-148 (JSC BIOCAD) for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
CLINICAL_TRIALS study record update · 2025-05-04
CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
CLINICAL_TRIALS study record update · 2025-04-18
An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)
CLINICAL_TRIALS study record update · 2025-04-09
Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH)
CLINICAL_TRIALS study record update · 2025-03-07
A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects With Inadequate Response to C5 Inhibitor Therapy
CLINICAL_TRIALS study record update · 2025-02-28
Study on Mass Balance of [14C]HSK39297 in Healthy Chinese Adult Male Subjects
CLINICAL_TRIALS study record update · 2025-02-27
Phase Ib Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VSA012 Injection in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH) Who Are Complement Inhibitor Naïve or Have Not Received Complement Inhibitor Therapy Recently
CLINICAL_TRIALS study record update · 2025-01-03
A Randomized, Double-Blind, Multicenter, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria
CLINICAL_TRIALS study record update · 2024-08-09
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
CLINICAL_TRIALS study record update · 2023-11-28
A Randomized, Multicenter, Active-comparator Controlled, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia, Despite Treatment With an Intravenous Anti-C5 Antibody.
CLINICAL_TRIALS study record update · 2023-11-15
A Multicenter, Single-arm, Open-label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily Iptacopan in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy