A Phase 3 interventional study of Iptacopan (LNP023) in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by Novartis Pharmaceuticals. Completed at 12 sites in 8 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 6th 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 40 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.
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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.
The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibitor therapy.
This study was a multicenter, single-arm, open-label trial which was comprised of 8 weeks screening period, 24-week core treatment period and 24-week extension treatment period.
Eligible PNH patients with hemolysis (LDH > 1.5 ULN) and anemia (hemoglobin \<10 g/dL), who were naive to complement inhibitor therapy, including anti-C5 antibody treatment, received iptacopan monotherapy at a dose 200 mg orally b.i.d.
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).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
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.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
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.
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.
Participants receive LNP023 at a dose of 200 mg orally b.i.d
Drug: Iptacopan (LNP023)
Taken orally b.i.d. Dosage supplied: 200mg Dosage form: Hard gelatin capsule Route of Administration: oral
Also known as: Iptacopan
Marginal Proportion (Expressed as Percentage) 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 of ≥ 2 g/dL on three out of four measurements between Day 126 and 168 of the core 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 of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the primary analysis.
Time frame: Baseline, hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168
Marginal Proportion (Expressed as Percentage) 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 between Day 126 and 168 of the core 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 of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the analysis.
Time frame: Hemoglobin between Day 126 and Day 168 and absence of transfusions between Day 14 and Day 168
Marginal Proportion (Expressed as Percentage) of Participants Who Remain Free From Transfusions
Marginal proportion (expressed as percentage) of participants who did not require transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. The 95% CI was obtained using the bootstrap method
Time frame: Between Day 14 and Day 168
Change From Baseline in Hemoglobin Levels in the Core Treatment Period
Change from baseline in hemoglobin levels as mean of visits between Day 126 and Day 168. In order to factor out the effect of transfusions in this analysis, if a patient had a transfusion during the core treatment period, the hemoglobin (Hb) values during 30 days following the transfusion were excluded and Hb data were imputed. Change from baseline was analyzed using a mixed model of repeated measures which included age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, and baseline hemoglobin as fixed effects and the interaction between visit and baseline hemoglobin levels.
Time frame: Baseline, Day 126 to 168
Percent Change From Baseline in LDH
Percent change from baseline in lactate dehydrogenase (LDH) levels as mean of visits between Day 126 and Day 168. Percentage change from baseline was analyzed using a mixed model for repeated measures (MMRM) which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline LDH as fixed effects and visit\*baseline LDH as interaction. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the analysis.
Time frame: Baseline, Day 126 to 168
Adjusted Annualized Clinical BTH Rate in the Core Treatment Period
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events is carried out using the Wilson method. 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
Change From Baseline in Absolute Reticulocyte Count
Change from baseline in absolute reticulocyte counts as mean of visits between Day 126 and Day 168. Change from baseline was analyzed using a MMRM which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline reticulocyte counts as fixed effects and visit\*baseline reticulocyte counts as interaction.
Time frame: Baseline and mean of visits between Day 126 and 168
Change From Baseline in FACIT-Fatigue Score
Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168. 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. Change from baseline was analyzed using a Mixed Model of Repeated Measures (MMRM) which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline FACIT-Fatigue score as fixed effects and visit\*baseline FACIT-Fatigue score as interaction.
Time frame: Baseline and mean of visits between Day 126 and Day 168
Adjusted Annualized Major Adverse Vascular Events Rate in the Core Treatment Period
Adjusted annual rate is carried out using the Wilson method. 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.
Time frame: Between Day 1 and Day 168
Percentage of Patients Meeting Hematological Response Criteria Irrespective of RBC Transfusions
Patients with hematological response are those with an increase in Hb from baseline ≥ 2g/dL irrespective of red blood cell (RBC) transfusions and patients achieving Hb ≥ 12g/dL irrespective of RBC transfusions.
Time frame: Baseline, Day 336
Marginal Proportion (Expressed as Percentage) of Patients Not Receiving and Not Requiring RBC Transfusions
Requiring Red Blood Cells (RBC) transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), regardless of presence of clinical signs and/or symptoms).
Time frame: Between Day 14 and Day 336
Change From Baseline in Hemoglobin Levels
Change from baseline in Hemoglobin at Visit Day 336
Time frame: Baseline, Day 336
Change From Baseline in LDH at Visit Day 336
Change from baseline in Lactate dehydrogenase (LDH) at Visit Day 336
Time frame: Baseline, Day 336
Adjusted Annualized Clinical BTH Rate After the Start of LNP023 Treatment
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events is carried out using the Wilson method. 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 336
Change From Baseline in Absolute Reticulocyte Count at Day 336
Change from baseline in absolute reticulocyte count at visit Day 336.
Time frame: Baseline, Day 336
Change From Baseline in FACIT-Fatigue Score
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, Day 336
Adjusted Annualized Major Adverse Vascular Events Rate After the Start of LNP023 Treatment
Adjusted annual rate is carried out using the Wilson method. A Major Adverse Vascular Events (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
Time frame: Between Day 1 and Day 336
Participants took part in 12 investigative sites in 8 countries: France(1), United Kingdom(1), Italy(1), Korea, Republic of(1), Singapore(1), China(3), Malaysia(2), Germany(2)
| Milestone | LNP023 |
|---|---|
| Started | 40 |
| Completed | 40 |
| Not completed | 0 |
| Milestone | LNP023 |
|---|---|
| Started | 40 |
| Completed | 40 |
| Not completed | 0 |
Sustained increase in hemoglobin levels (responder) is defined as an increase from baseline in hemoglobin levels of ≥ 2 g/dL on three out of four measurements between Day 126 and 168 of the core 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 of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the primary analysis.
| Percentage of responders | LNP023 |
|---|---|
| Marginal Proportion (Expressed as Percentage) of Participants With Sustained Increase in Hemoglobin Levels From Baseline of ≥ 2 g/dL in the Absence of Red Blood Cell Transfusions | 92.2 (82.5 to 100.0) |
Sustained hemoglobin levels (responder) is defined as hemoglobin levels ≥ 12 g/dL on three out of four measurements between Day 126 and 168 of the core 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 of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the analysis.
| Percentage of responders | LNP023 |
|---|---|
| Marginal Proportion (Expressed as Percentage) With Sustained Hemoglobin Levels of ≥ 12 g/dL in the Absence of Red Blood Cell Transfusions | 62.8 (47.5 to 77.5) |
Marginal proportion (expressed as percentage) of participants who did not require transfusions between Day 14 and Day 168. Requiring RBC transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), 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. The 95% CI was obtained using the bootstrap method
| Percentage of participants | LNP023 |
|---|---|
| Marginal Proportion (Expressed as Percentage) of Participants Who Remain Free From Transfusions | 97.6 (92.5 to 100.0) |
Change from baseline in hemoglobin levels as mean of visits between Day 126 and Day 168. In order to factor out the effect of transfusions in this analysis, if a patient had a transfusion during the core treatment period, the hemoglobin (Hb) values during 30 days following the transfusion were excluded and Hb data were imputed. Change from baseline was analyzed using a mixed model of repeated measures which included age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, and baseline hemoglobin as fixed effects and the interaction between visit and baseline hemoglobin levels.
| g/dL | LNP023 |
|---|---|
| Change From Baseline in Hemoglobin Levels in the Core Treatment Period | 4.28 (3.87 to 4.70) |
Percent change from baseline in lactate dehydrogenase (LDH) levels as mean of visits between Day 126 and Day 168. Percentage change from baseline was analyzed using a mixed model for repeated measures (MMRM) which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline LDH as fixed effects and visit\*baseline LDH as interaction. Results incorporated a method to handle missing data using multiple imputation. Hence, all 40 patients enrolled contributed to the analysis.
| Percent change from baseline in LDH | LNP023 |
|---|---|
| Percent Change From Baseline in LDH | -83.55 (-84.90 to -82.08) |
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events is carried out using the Wilson method. 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 |
|---|---|
| Adjusted Annualized Clinical BTH Rate in the Core Treatment Period | 0.00 (0.00 to 0.17) |
Change from baseline in absolute reticulocyte counts as mean of visits between Day 126 and Day 168. Change from baseline was analyzed using a MMRM which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline reticulocyte counts as fixed effects and visit\*baseline reticulocyte counts as interaction.
| x10^9 cells/L | LNP023 |
|---|---|
| Change From Baseline in Absolute Reticulocyte Count | -82.48 (-89.33 to -75.62) |
Change from baseline in FACIT-Fatigue scores as mean of visits between Day 126 and Day 168. 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. Change from baseline was analyzed using a Mixed Model of Repeated Measures (MMRM) which includes age (indicator variable of age ≥ 45 years), sex, history of transfusion (yes/no) prior to study treatment, visit, baseline FACIT-Fatigue score as fixed effects and visit\*baseline FACIT-Fatigue score as interaction.
| score on a scale | LNP023 |
|---|---|
| Change From Baseline in FACIT-Fatigue Score | 10.75 (8.66 to 12.84) |
Adjusted annual rate is carried out using the Wilson method. 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.
| MAVE events/year | LNP023 |
|---|---|
| Adjusted Annualized Major Adverse Vascular Events Rate in the Core Treatment Period | 0.00 (0.00 to 0.17) |
Patients with hematological response are those with an increase in Hb from baseline ≥ 2g/dL irrespective of red blood cell (RBC) transfusions and patients achieving Hb ≥ 12g/dL irrespective of RBC transfusions.
| Percentage of participants | LNP023 |
|---|---|
| ≥2 g/dL increase in Hb from baseline irrespective of RBC transfusions | 97.4 |
| Hb ≥ 12g/dL irrespective of transfusions | 79.5 |
Requiring Red Blood Cells (RBC) transfusions refers to any patient receiving transfusions or meeting protocol defined criteria (Hemoglobin level of ≤9 g/dL (≤8 g/dL for Chinese population) with signs and or symptoms of sufficient severity to warrant a transfusion or Hemoglobin of ≤7 g/dL (≤6 g/dL for Chinese population), regardless of presence of clinical signs and/or symptoms).
| Percentage of participants | LNP023 |
|---|---|
| Marginal Proportion (Expressed as Percentage) of Patients Not Receiving and Not Requiring RBC Transfusions | 97.5 (92.5 to 100.0) |
Change from baseline in Hemoglobin at Visit Day 336
| g/dL | LNP023 |
|---|---|
| Change From Baseline in Hemoglobin Levels | 5.09 ± 2.010 |
Change from baseline in Lactate dehydrogenase (LDH) at Visit Day 336
| U/L | LNP023 |
|---|---|
| Change From Baseline in LDH at Visit Day 336 | -1393.3 ± 652.15 |
Adjusted annualized rate of clinical breakthrough hemolysis (BTH) events is carried out using the Wilson method. 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 |
|---|---|
| Adjusted Annualized Clinical BTH Rate After the Start of LNP023 Treatment | 0.05 (0.01 to 0.17) |
Change from baseline in absolute reticulocyte count at visit Day 336.
| x10^9 cells/L | LNP023 |
|---|---|
| Change From Baseline in Absolute Reticulocyte Count at Day 336 | -76.55 ± 50.149 |
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 |
|---|---|
| Change From Baseline in FACIT-Fatigue Score | 10.4 ± 10.14 |
Adjusted annual rate is carried out using the Wilson method. A Major Adverse Vascular Events (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
| MAVE events/year | LNP023 |
|---|---|
| Adjusted Annualized Major Adverse Vascular Events Rate After the Start of LNP023 Treatment | 0.00 (0.00 to 0.09) |
Collected over Adverse events of LNP023 group were reported from first dose of study treatment until the end of study treatment plus up to 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. | 0/40 (0%) | 8/40 (20%) | 24/40 (60%) |
| Event | LNP023 200mg b.i.d. |
|---|---|
| COVID-19Infections and infestations | 2/40 |
| Breakthrough haemolysisBlood and lymphatic system disorders | 1/40 |
| CataractEye disorders | 1/40 |
| InfectionInfections and infestations | 1/40 |
| PneumoniaInfections and infestations | 1/40 |
| Pneumonia bacterialInfections and infestations | 1/40 |
| Type 2 diabetes mellitusMetabolism and nutrition disorders | 1/40 |
| Malignant melanomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/40 |
| Event | LNP023 200mg b.i.d. |
|---|---|
| HeadacheNervous system disorders | 12/40 |
| COVID-19Infections and infestations | 7/40 |
| Upper respiratory tract infectionInfections and infestations | 7/40 |
| DiarrhoeaGastrointestinal disorders | 6/40 |
| Abdominal painGastrointestinal disorders | 3/40 |
| ConstipationGastrointestinal disorders | 3/40 |
| VomitingGastrointestinal disorders | 3/40 |
| PyrexiaGeneral disorders | 3/40 |
| Iron deficiencyMetabolism and nutrition disorders | 3/40 |
| Age, Continuous(years) | LNP023 |
|---|---|
| Mean | 42.1 ± 15.85 |
| Sex: Female, Male(Participants) | LNP023 |
|---|---|
| Female | 17 |
| Male | 23 |
| Race/Ethnicity, Customized(Participants) | LNP023 |
|---|---|
| White | 12 |
| Black or African American | 1 |
| Asian | 27 |
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 Jul 2021 to primary completion in Nov 2022, 15 months.
Results were first posted in Nov 2023, 1 year 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 15 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