Phase 2 N=1 Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH)
Paroxysmal Nocturnal Haemoglobinuria (PNH)
Primary: Measurement of Ratio of LDH to the Upper Limit of Normal (ULN) — 5.6; 1.9 Ratio of LDH:ULN (250 U/L)
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=8 A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change From Baseline in LDH Level at Week 25 — -683.29 U/L
Phase 3 N=25 Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage Change From Baseline in LDH at Week 12 — -76.5 percentage change — p=<0.001
Phase 3 N=80 Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria
Primary: Least Squares (LS) Mean Change From Baseline to Week 16 in Hemoglobin (Hb) Level During the RCP — 2.37; -1.47 Grams per deciliter (g/dL) — p=<0.0001
Phase 2 N=24 Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Percentage of Participants Who Achieved Adequate Control of Intravascular Hemolysis — 75.0 Percentage of Participants
Phase 2 N=10 Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage of Participants With Reduction in Serum Lactate Dehydrogenase (LDH) Levels Within the First 4 Weeks of LFG316 Treatment as Measured by Response Rate — 10; 0…
Phase 3 N=187 Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Hemoglobinuria, Nocturnal
Primary: Incidence of Treatment-emergent Adverse Events — 182 Participants
Phase 2 N=29 Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change From Baseline in Lactate Dehydrogenase — 1845.1; 398.7; -1446.4 Units/Liter — p=<0.0001
Phase 2 N=1 Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Measurement of Serum Lactate Dehydrogenase (LDH) — 2913; 3056; 1453; 564 U/L
Phase 3 N=42 A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH
Paroxysmal Nocturnal Hemoglobinuria
Primary: LDH Level at Week 27 (Parallel Comparison) — 205.69; 193.53 U/L
Phase 3 N=53 A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Paroxysmal Nocturnal Hemoglobinuria
Primary: Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization — 30; 0 Participants — p=<0.0001
Phase 2 N=26 Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria · PNH
Primary: Percent Change In LDH Levels From Baseline To Day 253 And Day 281 — -72.85; -78.12; -84.96; -87.63 Percent Change — p=<0.0001
Phase 3 N=13 A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
Primary: Maximum Observed Serum Concentration (Cmax) Of Ravulizumab — 725.40; 884.63; 1161.60; 1612.50 μg/mL
Phase 2 N=6 Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
Paroxysmal Nocturnal Hemoglobinuria
Primary: OLTP: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 6 Participants
Phase 4 N=18 Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage of Participants Who Experienced Free C5-associated BTH — 0 percentage of participants
Phase 2 N=16 Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis
Primary: Percent Change From Baseline in Lactate Dehydrogenase (LDH) Level at Day 92 — -53.59; -25.56; -43.58 Percent change from Baseline
Phase 2 N=10 Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Change From Baseline In Serum LDH Levels At Day 28 — 1416; 444.3; -971.7 international units per liter (IU/L)
Phase 2 N=13 Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH
Primary: Percent Change In Lactate Dehydrogenase (LDH) Levels From Baseline To Day 169 — -85.952; -84.736 percent change
Phase 2 N=13 Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
Primary: Summary of Lactate Dehydrogenase (LDH) Responders — 6; 5; 7; 5 Participants
Phase 3 N=50 A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
Primary: Lactate Dehydrogenase (U/L) at Week 26 — 284.20; 249.72 U/L
Phase 2 N=24 Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
Paroxysmal Nocturnal Hemoglobinuria
Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) — 66.7; 41.7; 16.7; 0 Percentage of participants
Phase 2 N=12 Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Change From Baseline In Hemoglobin At Week 24 — 7.94; 10.33; 2.39 g/dL
Phase 2 N=27 E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change From Baseline in Lactate Dehydrogenase — -15.9 Units/Liter — p=<0.001
Phase 4 N=7 Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Hemoglobinuria, Paroxysmal
Primary: Peak And Trough Concentrations Of Eculizumab In Serum At Week 12 — 192.5; 425.4 μg/mL
Phase 1 N=23 Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects.
Paroxysmal Nocturnal Hemoglobinuria
Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity — 2; 18; 2; 9 Participants
Phase 3 N=136 Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Paroxysmal Nocturnal Hemoglobinuria
Primary: Ctrough Serum Concentration of Ravulizumab — 457.58; 578.70 micrograms/milliliter (µg/mL) — p=<0.0001
Phase 3 N=52 Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change in Hb Levels as Mean of Visits Between Day 126 and Day 168 Compared to Baseline Tested for Non-inferiority — 2.01 g/dL — p=<0.0001
Phase 2 N=4 A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
PNH
Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity — 3; 2; 1; 0 Participants
Phase 2 N=26 Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. — -695.2; 216.7 U/L
Phase 3 N=86 Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)
Paroxysmal Nocturnal Hemoglobinuria
Primary: Change From Baseline in Hgb at Week 12 — 29.40; 4.96; 28.08; 4.62 g/L — p=0.0007