A Phase 3 interventional study of LNP023 and Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Novartis Pharmaceuticals. Completed at 39 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-09.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment
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Among 16 studies in Hemoglobinuria, Paroxysmal by Novartis — 5 open to participants now, 8 under way, 2 of them phase 3. Hemoglobinuria, Paroxysmal · Novartis.
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis, this one is one of 3 that started in 2021, is one of 5 in phase 3, and was the 4th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
Browse Hemoglobinuria, Paroxysmal studies →5 of the 16 studies in Hemoglobinuria, Paroxysmal by Novartis are open now, in 8 countries. Recruiting first, the newest first:
Novartis Pharmaceuticals · Sites in Korea, Republic of
Ages 18–99
Novartis Pharmaceuticals · Sites in Italy
Ages 18+
Novartis Pharmaceuticals · Sites in United States, Brazil, Colombia and 3 more countries
Ages 2–18
Novartis Pharmaceuticals · Sites in China
Ages 18–80
Novartis Pharmaceuticals · Sites in United States
Ages 18–99
By country, each count listing its studies:
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
16 studies; the count open now beside each. This study's row is marked.
First in 2015; this study’s year is marked. 3 started in 2021.
This study's enrollment of 97 is above the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Median enrollment 28 across the 8 interventional studies, 80 across the 7 observational. Of the 7 that completed or were terminated, 6 (86%) have results posted.
This tab as a report to print or save as PDF → With an account.
This study is registered under Novartis Pharmaceuticals, one of the names counted as Novartis. Under that name alone:
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
2 of the studies in Hemoglobinuria, Paroxysmal by Novartis that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04747613Phase 3Novartis PharmaceuticalsLong-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | 2027-10-18 |
| NCT06931691Novartis PharmaceuticalsA Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China | 2027-04-21 |
Add all 2 to your calendar (.ics) → With an account.
Among the 16 studies in Hemoglobinuria, Paroxysmal by Novartis, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
This study was a multi-center, randomized, open-label, active comparator-controlled, parallel group study.
The purpose of this Phase 3 study in PNH patients presenting with residual anemia despite treatment with anti-C5 antibody, was to determine whether iptacopan is efficacious and safe for the treatment of PNH through demonstration of superiority of iptacopan compared to anti-C5 antibody treatment.
The study comprised three periods:
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Iptacopan 200mg b.i.d. hard gelatin capsule. After 24 weeks of LNP023 200mg b.i.d. treatment in the Randomized Treatment Period, participants had the option to enter the Extension Treatment Period to receive an additional 24 weeks of LNP023 200mg b.i.d.
Drug: LNP023
In the Randomized Treatment Period patients randomized to receive Anti-C5 antibody continued with the same stable regimen of Anti-C5 antibody therapy as they had received prior to randomization. For eculizumab (administered as intravenous infusion every 2 weeks), the maintenance dose was a fixed dose, whereas for ravulizumab (administered as intravenous infusion every 8 weeks), the maintenance dose was based on body weight. After 24 weeks of Anti-C5 antibody treatment in the Randomized Treatment Period, participants had the option to enter the Extension Treatment Period to receive 24 weeks of LNP023 200mg b.i.d.
Drug: Eculizumab · Drug: Ravulizumab
Taken Orally b.i.d. Dosage Supplied: 200 mg Dosage form: Hard gelatin capsule Route of Administration: Oral
Also known as: iptacopan
Administered as intravenous infusion every 2 weeks as per the stable regimen, the maintenance dose is a fixed dose. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion
Administered as intravenous infusion every 8 weeks, the maintenance dose is based on body weight. Dosage Supplied: 300 mg/30mL Dosage form: Concentrate solution for infusion
Marginal Proportion (Expressed as Percentages) of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 2 g/dL in the Absence of Red Blood Cell Transfusions
Sustained increase in hemoglobin levels (responder) is defined as an increase from baseline in hemoglobin levels ≥ 2 g/dL on three out of four measurements taken at the visits occurring in last six weeks (between Day 126 and 168) of the randomized treatment period, without requiring red blood cell (RBC) transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
Time frame: Baseline, hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168
Marginal Proportion (Expressed as Percentages) of Participants With Sustained Hemoglobin Levels of ≥ 12 g/dL in the Absence of Red Blood Cell Transfusions
Sustained hemoglobin levels (responder) is defined as hemoglobin levels ≥ 12 g/dL on three out of four measurements taken at the visits occurring in last six weeks (between Day 126 and 168) of the randomized treatment period, without requiring red blood cell (RBC) transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
Time frame: Hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168
Percentage of Patients Meeting Hematological Response Criterion After the Start of LNP023 Treatment
Patients with hematological response are those with ≥ 2g/dL increase in hemoglobin from baseline regardless of transfusions and patients with Hb ≥ 12g/dL regardless of transfusions. Patients in the LNP023-LNP023 group received iptacopan from Day 1 to Day 336 (48 weeks) while patients in the anti-C5 antibody-LNP023 group received iptacopan from Day 169 to Day 336 (treatment extension period - 24 weeks).
Time frame: Up to 48 weeks
Number of Patients Not Requiring RBC Transfusions After the Start of LNP023 Treatment
Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
Time frame: Up to 48 weeks
Change From Baseline in Hemoglobin at Visit Day 336
Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
Time frame: Baseline, Day 336
Change From Baseline in FACIT-Fatigue Questionnaire at Day 336
The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
Time frame: Baseline, Day 336
Adjusted Annualized Clinical BTH Rate After the Start of LNP023 Treatment
This endpoint is considering clinical BTH events after the start of LNP023 treatment. Therefore, results are presented in a single arm on LNP023 since it includes all patients in the Combined Full analysis set. Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events are from negative binomial model. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.The breakthrough is defined clinical if either there is a decrease in hemoglobin levels equal to or more than 2 g/dL (compared to the latest assessment, or within 15 days) or if patients present signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia or any other significant clinical PNH-related signs \& symptoms, in presence of laboratory evidence of intravascular hemolysis.
Time frame: Up to 336 Days
Adjusted Annualized Major Adverse Vascular Events Rate After the Start of LNP023 Treatment
This endpoint is considering clinical BTH events after the start of LNP023 treatment. Therefore, results are presented in a single arm on LNP023 since it includes all patients in the Combined Full analysis set. Adjusted annualized Major Adverse Vascular Events (MAVEs incl. thrombosis) rate. A MAVE is defined as: acute peripheral vascular occlusion, amputation (non-traumatic; nondiabetic), cerebral arterial occlusion/cerebrovascular accident, cerebral venous occlusion, dermal thrombosis, gangrene (non-traumatic; nondiabetic), hepatic/portal vein thrombosis (Budd-Chiari syndrome), mesenteric/visceral arterial, thrombosis or infarction, mesenteric/visceral vein thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial thrombosis, renal vein thrombosis, thrombophlebitis / deep vein thrombosis, transient ischemic attack, unstable angina or other. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.
Time frame: Up to 336 Days
Marginal Proportion (Expressed as Percentages) of Participants Who Remain Free From Transfusions
Marginal proportion (expressed as percentages) of participants who did not require transfusions between Day 14 and Day 168. Requiring red blood cell transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
Time frame: Between Day 14 and Day 168
Change From Baseline in Hemoglobin Between Day 126 and 168
Change from baseline in hemoglobin levels as mean of visits between Day 126 and Day 168. For this analysis, in order to factor out the effect of transfusions, if a patient had a transfusion during the randomized treatment period, then the hemoglobin values 30 days following the transfusion were excluded and hemoglobin data were imputed.
Time frame: Baseline and mean of visits between Day 126 and 168
Change From Baseline in FACIT-Fatigue Questionnaire in the Randomized Treatment Period
The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best.
Time frame: Baseline, mean of visits between Day 126 and Day 168
Change From Baseline in Absolute Reticulocyte Count in the Randomized Treatment Period
Change from baseline in absolute reticulocyte count as mean of visits between Day 126 and Day 168
Time frame: Baseline and mean of visits between Day 126 and 168
Ratio to Baseline in Log-transformed LDH in the Randomized Treatment Period
Average of the Lactate dehydrogenase (LDH) log transformed ratio to baseline in each treatment estimated between Day 126 and Day 168.The log transformation used refers to the natural log (base of e).
Time frame: Baseline and mean of visits between Day 126 and 168
Adjusted Annualized Clinical BTH Rate in the Randomized Treatment Period
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events are from negative binomial model. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment. The breakthrough is defined clinical if either there is a decrease in hemoglobin levels equal to or more than 2 g/dL (compared to the latest assessment, or within 15 days) or if patients present signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia or any other significant clinical PNH-related signs \& symptoms, in presence of laboratory evidence of intravascular hemolysis.
Time frame: Between Day 1 and Day 168
Adjusted Annualized Major Adverse Vascular Events Rate in the Randomized Treatment Period
Adjusted annualized Major Adverse Vascular Events (MAVEs incl. thrombosis) rate. A MAVE is defined as: acute peripheral vascular occlusion, amputation (non-traumatic; nondiabetic), cerebral arterial occlusion/cerebrovascular accident, cerebral venous occlusion, dermal thrombosis, gangrene (non-traumatic; nondiabetic), hepatic/portal vein thrombosis (Budd-Chiari syndrome), mesenteric/visceral arterial, thrombosis or infarction, mesenteric/visceral vein thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial thrombosis, renal vein thrombosis, thrombophlebitis / deep vein thrombosis, transient ischemic attack, unstable angina or other. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.
Time frame: Between Day 1 and Day 168
Change From Baseline in Absolute Reticulocyte Count at Day 336
Change from baseline in absolute reticulocyte count at visit Day 336. Patients randomized to anti-C5 antibody were switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
Time frame: Baseline and Day 336
Ratio to Baseline in Log-transformed LDH at Visit Day 336
Average of the Lactate dehydrogenase (LDH) log transformed ratio to baseline at visit Day 336.The log transformation used refers to the natural log (base of e). Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
Time frame: Baseline and Day 336
Participants took part in 39 investigative sites in 12 countries: Netherlands(1), Germany(5), France(3), Japan(7), Korea, Republic of(1), Italy(7), Spain(3), Taiwan(2), United Kingdom(2), Czech Republic(1), United States(5), Brazil(2)
| Milestone | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Started | 62 | 35 |
| Completed | 62 | 35 |
| Not completed | 0 | 0 |
| Milestone | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Started | 61 | 34 |
| Full analysis set | 62 | 35 |
| Combined full analysis set | 62 | 34 |
| Completed | 61 | 34 |
| Not completed | 0 | 0 |
Sustained increase in hemoglobin levels (responder) is defined as an increase from baseline in hemoglobin levels ≥ 2 g/dL on three out of four measurements taken at the visits occurring in last six weeks (between Day 126 and 168) of the randomized treatment period, without requiring red blood cell (RBC) transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
| Percentage of responders | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Marginal Proportion (Expressed as Percentages) of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 2 g/dL in the Absence of Red Blood Cell Transfusions | 82.3 (73.4 to 90.2) | 2.0 (1.1 to 4.0) |
Sustained hemoglobin levels (responder) is defined as hemoglobin levels ≥ 12 g/dL on three out of four measurements taken at the visits occurring in last six weeks (between Day 126 and 168) of the randomized treatment period, without requiring red blood cell (RBC) transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
| Percentage of responders | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Marginal Proportion (Expressed as Percentages) of Participants With Sustained Hemoglobin Levels of ≥ 12 g/dL in the Absence of Red Blood Cell Transfusions | 68.8 (58.4 to 78.9) | 1.8 (0.9 to 4.0) |
Patients with hematological response are those with ≥ 2g/dL increase in hemoglobin from baseline regardless of transfusions and patients with Hb ≥ 12g/dL regardless of transfusions. Patients in the LNP023-LNP023 group received iptacopan from Day 1 to Day 336 (48 weeks) while patients in the anti-C5 antibody-LNP023 group received iptacopan from Day 169 to Day 336 (treatment extension period - 24 weeks).
| Percentage of participants | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| ≥2 g/dL increase in Hb from baseline irrespective of RBC transfusions | 86.4 | 72.4 |
| Hb ≥12 g/dL irrespective of RBC transfusions | 67.8 | 58.6 |
Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
| Participants | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Since Day 1 of LNP023 treatment | 51 | 31 |
| Since Day 14 of LNP023 treatment | 57 | 32 |
Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
| g/dL | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in Hemoglobin at Visit Day 336 | 3.35 (3.04 to 3.67) | 3.36 (2.94 to 3.79) |
The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best. Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
| score on a scale | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in FACIT-Fatigue Questionnaire at Day 336 | 9.80 (8.04 to 11.56) | 10.96 (8.58 to 13.34) |
Marginal proportion (expressed as percentages) of participants who did not require transfusions between Day 14 and Day 168. Requiring red blood cell transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level ≤ 9 g/dL with signs /and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤ 7 g/dL, regardless of presence of clinical signs and/or symptoms). The term 'marginal proportion' can be interpreted as the population average probability of being a responder for each treatment group. These values include adjustment for baseline covariates and missing data has also been taken into account.
| Percentage of participants | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Marginal Proportion (Expressed as Percentages) of Participants Who Remain Free From Transfusions | 94.8 (88.1 to 100.0) | 25.9 (11.6 to 42.4) |
Change from baseline in hemoglobin levels as mean of visits between Day 126 and Day 168. For this analysis, in order to factor out the effect of transfusions, if a patient had a transfusion during the randomized treatment period, then the hemoglobin values 30 days following the transfusion were excluded and hemoglobin data were imputed.
| g/dL | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in Hemoglobin Between Day 126 and 168 | 3.60 (3.33 to 3.88) | -0.06 (-0.45 to 0.34) |
The FACIT-Fatigue is a 13-item questionnaire with support for its validity and reliability in PNH that assesses patient self-reported fatigue and its impact on daily activities and function. All FACIT scales are scored so that a high score is better. As each of the 13 items of the FACIT-F Scale ranges from 0-4, the range of possible scores is 0-52, with 0 being the worst possible score and 52 the best.
| score on a scale | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in FACIT-Fatigue Questionnaire in the Randomized Treatment Period | 8.59 (6.72 to 10.47) | 0.31 (-2.20 to 2.81) |
Change from baseline in absolute reticulocyte count as mean of visits between Day 126 and Day 168
| x10^9 cells/L | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in Absolute Reticulocyte Count in the Randomized Treatment Period | -115.81 (-126.40 to -105.23) | 0.34 (-13.04 to 13.72) |
Average of the Lactate dehydrogenase (LDH) log transformed ratio to baseline in each treatment estimated between Day 126 and Day 168.The log transformation used refers to the natural log (base of e).
| ln(ratio) | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Ratio to Baseline in Log-transformed LDH in the Randomized Treatment Period | 0.96 (0.90 to 1.03) | 0.98 (0.89 to 1.07) |
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events are from negative binomial model. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment. The breakthrough is defined clinical if either there is a decrease in hemoglobin levels equal to or more than 2 g/dL (compared to the latest assessment, or within 15 days) or if patients present signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia or any other significant clinical PNH-related signs \& symptoms, in presence of laboratory evidence of intravascular hemolysis.
| BTH events/year | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Adjusted Annualized Clinical BTH Rate in the Randomized Treatment Period | 0.07 (0.02 to 0.31) | 0.67 (0.26 to 1.72) |
Adjusted annualized Major Adverse Vascular Events (MAVEs incl. thrombosis) rate. A MAVE is defined as: acute peripheral vascular occlusion, amputation (non-traumatic; nondiabetic), cerebral arterial occlusion/cerebrovascular accident, cerebral venous occlusion, dermal thrombosis, gangrene (non-traumatic; nondiabetic), hepatic/portal vein thrombosis (Budd-Chiari syndrome), mesenteric/visceral arterial, thrombosis or infarction, mesenteric/visceral vein thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial thrombosis, renal vein thrombosis, thrombophlebitis / deep vein thrombosis, transient ischemic attack, unstable angina or other. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.
| MAVE events/year | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Adjusted Annualized Major Adverse Vascular Events Rate in the Randomized Treatment Period | 0.03 (0.00 to 0.25) | 0.00 (0.00 to 0.00) |
Change from baseline in absolute reticulocyte count at visit Day 336. Patients randomized to anti-C5 antibody were switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
| x10^9 cells/L | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Change From Baseline in Absolute Reticulocyte Count at Day 336 | -106.26 (-117.57 to -94.96) | -107.95 (-123.18 to -92.73) |
Average of the Lactate dehydrogenase (LDH) log transformed ratio to baseline at visit Day 336.The log transformation used refers to the natural log (base of e). Patients randomized to anti-C5 treatment switched to LNP023 (iptacopan) on Day 169 and were treated until Day 336 (treatment extension period).
| ln(ratio) | LNP023 200mg b.i.d. | Anti-C5 Antibody |
|---|---|---|
| Ratio to Baseline in Log-transformed LDH at Visit Day 336 | 1.11 (1.02 to 1.22) | 0.99 (0.88 to 1.11) |
This endpoint is considering clinical BTH events after the start of LNP023 treatment. Therefore, results are presented in a single arm on LNP023 since it includes all patients in the Combined Full analysis set. Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events are from negative binomial model. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.The breakthrough is defined clinical if either there is a decrease in hemoglobin levels equal to or more than 2 g/dL (compared to the latest assessment, or within 15 days) or if patients present signs or symptoms of gross hemoglobinuria, painful crisis, dysphagia or any other significant clinical PNH-related signs \& symptoms, in presence of laboratory evidence of intravascular hemolysis.
| BTH events/year | Overall LNP023 200 mg b.i.d During the Entire Study |
|---|---|
| Adjusted Annualized Clinical BTH Rate After the Start of LNP023 Treatment | 0.11 (0.05 to 0.23) |
This endpoint is considering clinical BTH events after the start of LNP023 treatment. Therefore, results are presented in a single arm on LNP023 since it includes all patients in the Combined Full analysis set. Adjusted annualized Major Adverse Vascular Events (MAVEs incl. thrombosis) rate. A MAVE is defined as: acute peripheral vascular occlusion, amputation (non-traumatic; nondiabetic), cerebral arterial occlusion/cerebrovascular accident, cerebral venous occlusion, dermal thrombosis, gangrene (non-traumatic; nondiabetic), hepatic/portal vein thrombosis (Budd-Chiari syndrome), mesenteric/visceral arterial, thrombosis or infarction, mesenteric/visceral vein thrombosis or infarction, myocardial infarction, pulmonary embolus, renal arterial thrombosis, renal vein thrombosis, thrombophlebitis / deep vein thrombosis, transient ischemic attack, unstable angina or other. A patient with multiple occurrences of an event under one treatment is counted only once for that treatment.
| MAVE events/year | Overall LNP023 200 mg b.i.d During the Entire Study |
|---|---|
| Adjusted Annualized Major Adverse Vascular Events Rate After the Start of LNP023 Treatment | 0.04 (0.01 to 0.13) |
Collected over Adverse events of LNP023 group were reported from first dose of study treatment until the end of study treatment plus 30 days, up to a maximum duration of 48 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| LNP023 200mg b.i.d. (Randomized Treatment Period) | 0/62 (0%) | 6/62 (9.7%) | 34/62 (54.8%) |
| Anti-C5 Antibody (Randomized Treatment Period) | 0/35 (0%) | 5/35 (14.3%) | 21/35 (60%) |
| LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) | 0/62 (0%) | 9/62 (14.5%) | 43/62 (69.4%) |
| Any LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) | 0/96 (0%) | 13/96 (13.5%) | 62/96 (64.6%) |
| Event | LNP023 200mg b.i.d. (Randomized Treatment Period) | Anti-C5 Antibody (Randomized Treatment Period) | LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) | Any LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) |
|---|---|---|---|---|
| COVID-19Infections and infestations | 1/62 | 2/35 | 1/62 | 1/96 |
| Breakthrough haemolysisBlood and lymphatic system disorders | 0/62 | 1/35 | 0/62 | 0/96 |
| Extravascular haemolysisBlood and lymphatic system disorders | 0/62 | 1/35 | 0/62 | 0/96 |
| JaundiceHepatobiliary disorders | 0/62 | 1/35 | 0/62 | 0/96 |
| Arthritis bacterialInfections and infestations | 0/62 | 1/35 | 0/62 | 0/96 |
| Intervertebral discitisInfections and infestations | 0/62 | 1/35 | 0/62 | 0/96 |
| Pseudomonal sepsisInfections and infestations | 0/62 | 1/35 | 0/62 | 0/96 |
| Influenza A virus test positiveInvestigations | 0/62 | 1/35 | 0/62 | 0/96 |
| Acute kidney injuryRenal and urinary disorders | 0/62 | 1/35 | 0/62 | 0/96 |
| BilirubinuriaRenal and urinary disorders | 0/62 | 1/35 | 0/62 | 0/96 |
| Event | LNP023 200mg b.i.d. (Randomized Treatment Period) | Anti-C5 Antibody (Randomized Treatment Period) | LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) | Any LNP023 200mg b.i.d. (Randomized Treatment Period + Extension Treatment Period) |
|---|---|---|---|---|
| COVID-19Infections and infestations | 4/62 | 7/35 | 17/62 | 25/96 |
| HeadacheNervous system disorders | 11/62 | 1/35 | 12/62 | 14/96 |
| Breakthrough haemolysisBlood and lymphatic system disorders | 2/62 | 6/35 | 6/62 | 7/96 |
| DiarrhoeaGastrointestinal disorders | 9/62 | 2/35 | 10/62 | 12/96 |
| NasopharyngitisInfections and infestations | 7/62 | 3/35 | 9/62 | 12/96 |
| NauseaGastrointestinal disorders | 6/62 | 1/35 | 8/62 | 11/96 |
| Urinary tract infectionInfections and infestations | 4/62 | 1/35 | 7/62 | 7/96 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 5/62 | 1/35 | 7/62 | 7/96 |
| Blood lactate dehydrogenase increasedInvestigations | 4/62 | 3/35 | 6/62 | 6/96 |
| PyrexiaGeneral disorders | 2/62 | 3/35 | 4/62 | 5/96 |
| Age, Continuous(years) | LNP023 200mg b.i.d. | Anti-C5 Antibody | Total |
|---|---|---|---|
| Mean | 51.7 ± 16.94 | 49.8 ± 16.69 | 51.0 ± 16.79 |
| Sex: Female, Male(Participants) | LNP023 200mg b.i.d. | Anti-C5 Antibody | Total |
|---|---|---|---|
| Female | 43 | 24 | 67 |
| Male | 19 | 11 | 30 |
| Race/Ethnicity, Customized(Participants) | LNP023 200mg b.i.d. | Anti-C5 Antibody | Total |
|---|---|---|---|
| White | 48 | 26 | 74 |
| Black or African American | 2 | 2 | 4 |
| Asian | 12 | 7 | 19 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Ran from Jan 2021 to primary completion in Sep 2022, 20 months.
Results were first posted in Nov 2023, 1 year 2 months after primary completion.
Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's 20 months is below that.
Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Iptacopan