18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Summary of Lactate Dehydrogenase (LDH) RespondersPrimary· Week 2, week 4, week 8 and week 12
A responder was defined as a patient with at least 60% reduction in LDH compared to Baseline or LDH below the upper limit of normal at any time up to and including Week 12 for that patient.
Responders at week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
6
LNP023 50 mg Bid/200 mg Bid
5
Responders at week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
7
LNP023 50 mg Bid/200 mg Bid
5
Responders at week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
7
LNP023 50 mg Bid/200 mg Bid
5
Responders at week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
7
LNP023 50 mg Bid/200 mg Bid
5
Percent Change From Baseline in LDH LevelsSecondary· Baseline, week 2, week 4, week 8 and week 12
LDH was used as a hemolysis marker to determine the dose-response effect of iptacopan on the reduction of Paroxysmal nocturnal hemoglobinuria (PNH)-associated hemolysis.
Active hemolysis is defined by an LDH value ≥ 1.5x upper limit of normal (ULN) Baseline LDH was calculated as the average of the last three screening values prior to randomization.
Serum was used to calculate the LDH values
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-76.52
± 18.478
LNP023 50 mg Bid/200 mg Bid
-84.98
± 8.186
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-79.75
± 16.025
LNP023 50 mg Bid/200 mg Bid
-89.72
± 5.430
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-81.73
± 22.832
LNP023 50 mg Bid/200 mg Bid
-88.30
± 8.744
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-86.17
± 8.896
LNP023 50 mg Bid/200 mg Bid
-85.92
± 9.013
Change From Baseline in HemoglobinSecondary· Baseline, Week 2, week 4, week 8 and week 12
Hemoglobin was used as a hemolysis marker to determine the dose-response effect of iptacopan on the reduction of PNH-associated hemolysis. Whole blood was used to calculate the hemoglobin values.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
6.88
± 5.812
LNP023 50 mg Bid/200 mg Bid
31.83
± 10.362
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
9.61
± 11.857
LNP023 50 mg Bid/200 mg Bid
36.58
± 17.875
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
15.02
± 9.154
LNP023 50 mg Bid/200 mg Bid
38.08
± 25.956
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
23.36
± 14.378
LNP023 50 mg Bid/200 mg Bid
37.11
± 26.171
Change From Baseline in Free HemoglobinSecondary· Baseline, Week 2, week 4, week 8 and week 12
Free hemoglobin was used as a hemolysis marker to determine the dose-response effect of iptacopan on the reduction of PNH-associated hemolysis.
Whole blood was used to calculate the hemoglobin values.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-39.84
± 26.521
LNP023 50 mg Bid/200 mg Bid
-13.53
± 5.093
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-31.88
± 35.314
LNP023 50 mg Bid/200 mg Bid
-29.26
± 24.218
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-31.85
± 27.517
LNP023 50 mg Bid/200 mg Bid
-16.31
± 48.877
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-39.58
± 31.612
LNP023 50 mg Bid/200 mg Bid
-14.75
± 6.293
Change From Baseline in CarboxyhemoglobinSecondary· Baseline, Week 2, week 4, week 8 and week 12
Carboxyhemoglobin was used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan.
Whole blood was used to calculate the carboxyhemoglobin values.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-0.01
± 0.516
LNP023 50 mg Bid/200 mg Bid
-0.72
± 0.697
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-0.36
± 0.472
LNP023 50 mg Bid/200 mg Bid
-0.98
± 0.582
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-0.50
± 0.664
LNP023 50 mg Bid/200 mg Bid
-1.12
± 0.916
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-0.95
± 0.316
LNP023 50 mg Bid/200 mg Bid
-0.78
± 1.704
Change From Baseline in Absolute Reticulocyte Count (ARC)Secondary· Baseline, week 2, week 4, week 8 and week 12
Reticulocyte count was used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan.
Whole blood was used to calculate the absolute reticulocyte count.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-83.00
± 78.750
LNP023 50 mg Bid/200 mg Bid
-94.23
± 112.649
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-118.44
± 76.736
LNP023 50 mg Bid/200 mg Bid
-132.63
± 91.109
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-113.71
± 72.300
LNP023 50 mg Bid/200 mg Bid
-119.43
± 117.048
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-93.00
± 93.425
LNP023 50 mg Bid/200 mg Bid
-84.92
± 123.408
Red Blood Cell Count: Change From Baseline in ErythrocytesSecondary· Baseline, week 2, week 4, week 8 and week 12
Erythrocytes were used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan.
Whole blood was used to calculate erythrocytes values.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.49
± 0.194
LNP023 50 mg Bid/200 mg Bid
1.02
± 0.407
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.74
± 0.401
LNP023 50 mg Bid/200 mg Bid
1.26
± 0.619
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
1.16
± 0.396
LNP023 50 mg Bid/200 mg Bid
1.38
± 0.796
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
1.38
± 0.531
LNP023 50 mg Bid/200 mg Bid
1.38
± 0.770
Change From Baseline in C3 Fragment Deposition on PNH RBCSecondary· Baseline, week 2, week 4, week 8 and week 12
C3 fragment deposition on paroxysmal nocturnal hemoglobinuria red blood cell (PNH RBC) was used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan. Accumulation of C3 fragments on red blood cells make them prone to phagocytosis causing extravascular hemolysis.
Whole blood was used to calculate C3 fragment deposition on PNH RBC values.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-1.31
± 0.932
LNP023 50 mg Bid/200 mg Bid
-1.88
± 1.457
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-1.43
± 1.217
LNP023 50 mg Bid/200 mg Bid
-2.74
± 2.584
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-1.85
± 1.293
LNP023 50 mg Bid/200 mg Bid
-2.90
± 2.654
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-1.65
± 1.221
LNP023 50 mg Bid/200 mg Bid
-2.75
± 3.002
Mean Haptoglobin LevelsSecondary· Baseline, week 2, week 4, week 8 and week 12
Haptoglobin levels were used as a hemolysis marker to determine the effect of iptacopan on the reduction of PNH-associated hemolysis.
Serum was used to calculate haptoglobin levels.
Baseline
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.05
± 0.000
LNP023 50 mg Bid/200 mg Bid
0.05
± 0.000
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.27
± 0.251
LNP023 50 mg Bid/200 mg Bid
0.41
± 0.499
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.24
± 0.255
LNP023 50 mg Bid/200 mg Bid
0.49
± 0.490
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.38
± 0.456
LNP023 50 mg Bid/200 mg Bid
0.41
± 0.374
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
0.25
± 0.356
LNP023 50 mg Bid/200 mg Bid
0.18
± 0.225
Change From Baseline in Total BilirubinSecondary· Baseline, week 2, week 4, week 8 and week 12
Bilirubin levels were used as a hemolysis marker to determine the effect of iptacopan on the reduction of PNH-associated hemolysis.
Serum was used to calculate bilirubin levels.
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-19.81
± 7.855
LNP023 50 mg Bid/200 mg Bid
-23.93
± 16.735
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-19.95
± 10.620
LNP023 50 mg Bid/200 mg Bid
-25.93
± 15.992
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-20.24
± 12.612
LNP023 50 mg Bid/200 mg Bid
-25.53
± 16.471
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
-21.61
± 5.998
LNP023 50 mg Bid/200 mg Bid
-23.17
± 18.195
Mean Platelets CountSecondary· Baseline, week 2, week 4, week 8 and week 12
Platelet counts were used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan.
Whole blood was used to calculate platelets count.
Baseline
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
190.45
± 59.938
LNP023 50 mg Bid/200 mg Bid
156.42
± 52.321
Week 2
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
223.71
± 76.707
LNP023 50 mg Bid/200 mg Bid
158.20
± 62.683
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
203.17
± 68.520
LNP023 50 mg Bid/200 mg Bid
138.20
± 57.085
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
184.43
± 50.856
LNP023 50 mg Bid/200 mg Bid
141.20
± 46.569
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
189.33
± 54.617
LNP023 50 mg Bid/200 mg Bid
140.75
± 59.618
Mean Ferritin LevelsSecondary· Baseline, Week 4, Week 8 and week 12
Ferritin levels were used as a marker of intravascular and extravascular hemolysis to assess the effect of iptacopan.
Serum was used to calculate ferritin levels.
Baseline
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
176.97
± 404.254
LNP023 50 mg Bid/200 mg Bid
258.90
± 418.114
Week 4
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
174.21
± 431.042
LNP023 50 mg Bid/200 mg Bid
287.88
± 517.901
Week 8
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
178.31
± 443.346
LNP023 50 mg Bid/200 mg Bid
304.74
± 521.535
Week 12
Group
Value
95% CI
LNP023 25 mg Bid/100 mg Bid
203.22
± 454.296
LNP023 50 mg Bid/200 mg Bid
320.40
± 526.783
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were reported from first dose of study treatment until end of study treatment plus 7 days post treatment, up to a maximum duration of 115 weeks..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This was a Phase II randomized, open-label, multicenter, efficacy, safety, pharmacokinetic and pharmacodynamic study assessing four iptacopan doses in adult Paroxysmal nocturnal hemoglobinuria (PNH) patients with active hemolysis who were not on eculizumab or any other complement inhibitor less than 3 months prior to first iptacopan dose. Active hemolysis was defined by a lactate dehydrogenase (LDH) value ≥ 1.5 × ULN.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
NCT06934967 — Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
· Phase 3
· recruiting
NCT06931691 — A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
· recruiting
NCT04557462 — A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in P
· Phase 3
· recruiting
NCT04578834 — Study of Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients
· Phase 3
· completed
NCT04558918 — Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antib
· Phase 3
· completed
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 18 June 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03896152.