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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-002039-40 | EudraCT Number |
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Business decision
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The main purpose of this study was to evaluate the efficacy and safety of iptacopan in participants with autoimmune benign hematological disorders such as primary immune thrombocytopenia and primary cold agglutinin disease.
This was an open-label, single-arm (within each cohort), multi-center, non-confirmatory basket study to assess the efficacy, safety and pharmacokinetics of iptacopan in participants with autoimmune benign hematological disorders. The study was set up as a basket study to allow inclusion of new cohorts (indications). The study included 2 cohorts: primary immune thrombocytopenia (ITP) and primary cold agglutinin disease (CAD). Participants in Cohort 1 (ITP) were stratified in two groups according to high/low complement activation (i.e., based on sC5b-9 level at screening). No additional cohorts were added during the study.
The study consisted of a screening period, a 12-week treatment period (Part A), a washout (for responders)/follow-up (for non-responders) period after Part A, and, for responders only, an additional treatment extension period for up to 24 months (Part B). Non-responders who had signs of clinical benefit according to the Investigator's assessment could also continue treatment with iptacopan in Part B. The washout period prior to the start of part B lasted up to 4 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Iptacopan 200 mg BID | Experimental | Iptacopan 200 mg twice daily (BID) in participants with primary ITP and primary CAD |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Iptacopan | Drug | Iptacopan 200 mg BID given orally (capsule) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response | A study participant with ITP was considered a responder if all the below criteria were met:
| Up to 12 weeks (Part A) |
| Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response | A study participant with CAD was considered a responder if all the below criteria were met:
| Baseline, up to 12 weeks (Part A) |
| Measure | Description | Time Frame |
|---|---|---|
| Cohort 1 (ITP): Time to First Platelet Count ≥50 k/μL | The first time that a participant had a platelet count ≥50 k/μL after first dose of study treatment. Time to the first response was assessed for responders only. | Up to 12 weeks (Part A) |
| Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline |
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Inclusion Criteria:
All Cohorts:
Cohort 1 specific inclusion criteria:
Cohort 2 specific inclusion criteria:
Exclusion Criteria:
All cohorts:
Cohort 1 specific exclusion criteria:
Cohort 2 specific exclusion criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41366634 | Derived | Roth A, Barcellini W, Ademokun C, Jang J, Lozano ML, Ferreiras DV, Pascual-Izquierdo C, Chitnis S, Matviykiv S, Vitaliti A, Chen C, Katsanou V, Chawla R, Al-Samkari H. Iptacopan for Immune Thrombocytopenia and Cold Agglutinin Disease: A Global Phase 2 Basket Clinical Trial. Am J Hematol. 2026 Feb;101(2):242-254. doi: 10.1002/ajh.70147. Epub 2025 Dec 9. |
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on www.novctrd.com | View source |
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Novartis is committed to sharing with qualified external researchers, access to patient level data and supporting clinical documents from applicable 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
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The screening period began once patients had signed the study informed consent. Screening evaluations had to be completed within 8 weeks prior to the first dose of study treatment. The treatment period started on Day 1 of Part A.
Participants took part in 8 investigative sites in 6 countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 (ITP) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP) |
| FG001 | Cohort 2 (CAD) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 26, 2022 | Mar 4, 2025 |
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This is a basket study with different Cohorts.
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The first time that a participant had a hemoglobin level ≥1.5 g/dL above baseline after first dose of study treatment. Time to the first response was assessed for responders only. |
| Baseline, up to 12 weeks (Part A) |
| Cohort 1 (ITP): Duration of Time During Which Platelet Count Remains ≥50 k/μL Without the Use of Rescue Therapy | The duration of response corresponds to the duration of time during which a participant's platelet count remains ≥50 k/μL without the use of rescue therapy. The duration of response was considered as cumulative if there were non-continuous periods of response. Duration of response was analyzed for responders only. | Up to 12 weeks (Part A) |
| Cohort 2 (CAD): Duration of Time During Which Hemoglobin Level Remains ≥1.5 g/dL Above Baseline Without the Use of Rescue Therapy | The duration of response corresponds to the duration of time during which a participant's hemoglobin level remained ≥1.5 g/dL above baseline without the use of rescue therapy. The duration of response was considered as cumulative if there were non-continuous periods of response. Duration of response was analyzed for responders only. | Baseline, up to 12 weeks (Part A) |
| Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline | The magnitude of increase in platelet count compared to baseline was derived for each participant at each visit and time point. Best response across all visits is presented (highest value). The following categories were used: absolute platelet counts increase <50, ≥50 and <100, ≥100 and <150, and ≥150 k/uL. This endpoint is only applicable to participants without rescue therapy in the treatment period. | Baseline, up to 12 weeks (Part A) |
| Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline | The magnitude of increase in hemoglobin level compared to baseline was derived for each participant at each visit and time point. Best response across all visits is presented (highest value). The following categories were used: Hb increase from baseline by <1, ≥1 and <1.5, ≥1.5 and <2, and ≥2 g/dL. This endpoint is only applicable to participants without rescue therapy in the treatment period. | Baseline, up to 12 weeks (Part A) |
| Cohort 1 (ITP): Need for Rescue Therapy During Part A | Rescue therapy was defined as any therapy with ITP indication that started on or after Day 1. Rescue therapy, if indicated, could be initiated at the Investigator's discretion. From Day 1 onwards, if rescue therapy was needed before response criteria were met, the participant was treated as a non-responder. Conversely, use of rescue therapy after the primary endpoint was met, would not impact the response status with respect to that endpoint. For ITP, rescue therapy generally consisted of corticosteroids, intravenous immunoglobulins or anti-Rho(D) immunoglobulin and may had been indicated in case of worsening thrombocytopenia and/or signs or symptoms of bleeding. | Up to 12 weeks (Part A) |
| Cohort 2 (CAD): Need for Rescue Therapy During Part A | Rescue therapy was defined as any therapy with CAD indication that started on or after Day 1. Rescue therapy, if indicated, could be initiated at the Investigator's discretion. From Day 1 onwards, if rescue therapy was needed before response criteria were met, the participant was treated as a non-responder. Conversely, use of rescue therapy after the primary endpoint was met, would not impact the response status with respect to that endpoint. For CAD, rescue therapy generally consisted of plasmapheresis, intravenous immunoglobulins (IVIG) and/or red blood cell transfusions and may had been indicated in case of worsening anemia and/or critical hemolysis. | Up to 12 weeks (Part A) |
| Cohort 2 (CAD): Change From Baseline in Lactate Dehydrogenase (LDH) | LDH was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Baseline, up to 12 weeks (Part A) |
| Cohort 2 (CAD): Change From Baseline in Total Bilirubin | Total bilirubin was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Baseline, up to 12 weeks (Part A) |
| Cohort 2 (CAD): Change From Baseline in Reticulocyte Count | Reticulocyte count was measured in blood samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Baseline, up to 12 weeks (Part A) |
| Cohort 2 (CAD): Change From Baseline in Haptoglobin | Haptoglobin was measured in serum or plasma samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Baseline, up to 12 weeks (Part A) |
| Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B | Number of participants with AEs (any adverse events regardless of seriousness) and serious adverse events (SAEs), including changes from baseline in vital signs, electrocardiograms and laboratory results qualifying and reported as AEs. AE grades to characterize the severity of the AEs were based on the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. For CTCAE v5.0, Grade 1 = mild; Grade 2 = moderate; Grade 3 = severe; Grade 4 = life-threatening; Grade 5 = death related to AE. The on-treatment period is defined from the day of first administration of study drug up to 7 days after the last administration of study drug. | From first dose of study treatment to 7 days after last dose, up to approximately 43 weeks (Cohort 1) and 103 weeks (Cohort 2) |
| Cohort 1 and 2: Maximum Observed Plasma Concentration (Cmax) of Iptacopan | Pharmacokinetic (PK) parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. Cmax is defined as the maximum (peak) observed concentration following a dose. | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
| Cohort 1 and 2: Time to Maximum Observed Plasma Concentration (Tmax) of Iptacopan | PK parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. Tmax is defined as the time to reach maximum (peak) observed concentration following a dose. Actual sampling times were considered for the calculation of PK parameters. | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
| Cohort 1 and 2: Area Under the Plasma Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of Iptacopan | PK parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. The linear trapezoidal method was used for area under the curve (AUC) calculation. | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
| Cohort 1 and 2: Trough Plasma Concentration (Ctrough) of Iptacopan | Ctrough is defined as the concentration reached immediately before the next dose is administered. All drug concentrations below the lower limit of quantification were treated as zero for the calculation of PK parameters. | Pre-dose on Day 15, 29 and 57 of Part A |
| Essen |
| 45147 |
| Germany |
| Novartis Investigative Site | Milan | MI | 20122 | Italy |
| Novartis Investigative Site | Seoul | 06351 | South Korea |
| Novartis Investigative Site | Barcelona | Catalonia | 08035 | Spain |
| Novartis Investigative Site | Madrid | 28009 | Spain |
| Novartis Investigative Site | Murcia | 30008 | Spain |
| Novartis Investigative Site | London | W12 0HS | United Kingdom |
| PD Analysis Set | All participants who received any study drug and with no protocol deviations with relevant impact on pharmacodynamics (PD) data |
|
| ITP - sC5b-9 Low | Participants with primary ITP and low complement activation (low sC5b-9 levels) |
|
| ITP - sC5b-9 High | Participants with primary ITP and high complement activation (high sC5b-9 levels) |
|
| COMPLETED |
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| NOT COMPLETED |
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|
| Part B |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 (ITP) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP) |
| BG001 | Cohort 2 (CAD) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Platelets | Platelet count in blood at baseline for participants in Cohort 1 | Study specific characteristic for participants in Cohort 1 only. | Mean | Standard Deviation | platelets*10^9/liter |
| |||||||||||||
| Hemoglobin | Hemoglobin in blood at baseline for participants in Cohort 2 | Study specific characteristic for participants in Cohort 2 only. | Mean | Standard Deviation | gram/liter |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response | A study participant with ITP was considered a responder if all the below criteria were met:
| Participants from Cohort 1 in the Pharmacodynamic (PD) analysis set. Results are presented by sC5b-9 stratification group (sC5b-9 high and sC5b-9 low) and overall. | Posted | Count of Participants | Participants | Up to 12 weeks (Part A) |
|
|
| ||||||||||||||||||||||||||||||||
| Primary | Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response | A study participant with CAD was considered a responder if all the below criteria were met:
| Participants from Cohort 2 in the PD analysis set. | Posted | Count of Participants | Participants | Baseline, up to 12 weeks (Part A) |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Cohort 1 (ITP): Time to First Platelet Count ≥50 k/μL | The first time that a participant had a platelet count ≥50 k/μL after first dose of study treatment. Time to the first response was assessed for responders only. | Participants from Cohort 1 in the Pharmacodynamic (PD) analysis set who were responders. Results are presented by sC5b-9 stratification group (sC5b-9 high and sC5b-9 low) and overall. | Posted | Up to 12 weeks (Part A) |
|
| |||||||||||||||||||||||||||||||||||
| Secondary | Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline | The first time that a participant had a hemoglobin level ≥1.5 g/dL above baseline after first dose of study treatment. Time to the first response was assessed for responders only. | Participants from Cohort 2 in the PD analysis set who were responders. | Posted | Median | Full Range | days | Baseline, up to 12 weeks (Part A) |
|
| ||||||||||||||||||||||||||||||||
| Secondary | Cohort 1 (ITP): Duration of Time During Which Platelet Count Remains ≥50 k/μL Without the Use of Rescue Therapy | The duration of response corresponds to the duration of time during which a participant's platelet count remains ≥50 k/μL without the use of rescue therapy. The duration of response was considered as cumulative if there were non-continuous periods of response. Duration of response was analyzed for responders only. | Participants from Cohort 1 in the Pharmacodynamic (PD) analysis set who were responders. Results are presented by sC5b-9 stratification group (sC5b-9 high and sC5b-9 low) and overall. | Posted | Up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Duration of Time During Which Hemoglobin Level Remains ≥1.5 g/dL Above Baseline Without the Use of Rescue Therapy | The duration of response corresponds to the duration of time during which a participant's hemoglobin level remained ≥1.5 g/dL above baseline without the use of rescue therapy. The duration of response was considered as cumulative if there were non-continuous periods of response. Duration of response was analyzed for responders only. | Participants from Cohort 2 in the PD analysis set who were responders. | Posted | Median | Full Range | days | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline | The magnitude of increase in platelet count compared to baseline was derived for each participant at each visit and time point. Best response across all visits is presented (highest value). The following categories were used: absolute platelet counts increase <50, ≥50 and <100, ≥100 and <150, and ≥150 k/uL. This endpoint is only applicable to participants without rescue therapy in the treatment period. | Participants from Cohort 1 in the Pharmacodynamic (PD) analysis set who did not use rescue therapy in the treatment period of Part A. Results are presented by sC5b-9 stratification group (sC5b-9 high and sC5b-9 low) and overall. | Posted | Count of Participants | Participants | No | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline | The magnitude of increase in hemoglobin level compared to baseline was derived for each participant at each visit and time point. Best response across all visits is presented (highest value). The following categories were used: Hb increase from baseline by <1, ≥1 and <1.5, ≥1.5 and <2, and ≥2 g/dL. This endpoint is only applicable to participants without rescue therapy in the treatment period. | Participants from Cohort 2 in the PD analysis set who did not use rescue therapy in the treatment period of Part A. | Posted | Count of Participants | Participants | No | Baseline, up to 12 weeks (Part A) |
|
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| Secondary | Cohort 1 (ITP): Need for Rescue Therapy During Part A | Rescue therapy was defined as any therapy with ITP indication that started on or after Day 1. Rescue therapy, if indicated, could be initiated at the Investigator's discretion. From Day 1 onwards, if rescue therapy was needed before response criteria were met, the participant was treated as a non-responder. Conversely, use of rescue therapy after the primary endpoint was met, would not impact the response status with respect to that endpoint. For ITP, rescue therapy generally consisted of corticosteroids, intravenous immunoglobulins or anti-Rho(D) immunoglobulin and may had been indicated in case of worsening thrombocytopenia and/or signs or symptoms of bleeding. | Participants from Cohort 1 in the Pharmacodynamic (PD) analysis set. Results are presented by sC5b-9 stratification group (sC5b-9 high and sC5b-9 low) and overall. | Posted | Count of Participants | Participants | Up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Need for Rescue Therapy During Part A | Rescue therapy was defined as any therapy with CAD indication that started on or after Day 1. Rescue therapy, if indicated, could be initiated at the Investigator's discretion. From Day 1 onwards, if rescue therapy was needed before response criteria were met, the participant was treated as a non-responder. Conversely, use of rescue therapy after the primary endpoint was met, would not impact the response status with respect to that endpoint. For CAD, rescue therapy generally consisted of plasmapheresis, intravenous immunoglobulins (IVIG) and/or red blood cell transfusions and may had been indicated in case of worsening anemia and/or critical hemolysis. | Participants from Cohort 2 in the PD analysis set. | Posted | Count of Participants | Participants | Up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Change From Baseline in Lactate Dehydrogenase (LDH) | LDH was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Participants from Cohort 2 in the PD analysis set with an available value for the outcome measure at both baseline and end of treatment in Part A. | Posted | Mean | Standard Deviation | Units/liter (U/L) | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Change From Baseline in Total Bilirubin | Total bilirubin was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Participants from Cohort 2 in the PD analysis set with an available value for the outcome measure at both baseline and end of treatment in Part A. | Posted | Mean | Standard Deviation | micromole/liter (μmol/L) | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Change From Baseline in Reticulocyte Count | Reticulocyte count was measured in blood samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Participants from Cohort 2 in the PD analysis set with an available value for the outcome measure at both baseline and end of treatment in Part A. | Posted | Mean | Standard Deviation | reticulocytes * 10^9/liter | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 2 (CAD): Change From Baseline in Haptoglobin | Haptoglobin was measured in serum or plasma samples to assess the effect of treatment with iptacopan on relevant disease biomarkers. | Participants from Cohort 2 in the PD analysis set with an available value for the outcome measure at both baseline and end of treatment in Part A. | Posted | Mean | Standard Deviation | gram/liter (g/L) | Baseline, up to 12 weeks (Part A) |
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| Secondary | Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B | Number of participants with AEs (any adverse events regardless of seriousness) and serious adverse events (SAEs), including changes from baseline in vital signs, electrocardiograms and laboratory results qualifying and reported as AEs. AE grades to characterize the severity of the AEs were based on the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. For CTCAE v5.0, Grade 1 = mild; Grade 2 = moderate; Grade 3 = severe; Grade 4 = life-threatening; Grade 5 = death related to AE. The on-treatment period is defined from the day of first administration of study drug up to 7 days after the last administration of study drug. | All participants who received at least one dose of study treatment. | Posted | Count of Participants | Participants | From first dose of study treatment to 7 days after last dose, up to approximately 43 weeks (Cohort 1) and 103 weeks (Cohort 2) |
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| Secondary | Cohort 1 and 2: Maximum Observed Plasma Concentration (Cmax) of Iptacopan | Pharmacokinetic (PK) parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. Cmax is defined as the maximum (peak) observed concentration following a dose. | Participants in the PK analysis set (PAS) who had an available value for the outcome measure on the assessed study day. PAS consisted of all participants with at least one available valid PK concentration measurement, who received any study drug and with no protocol deviations or AEs which may have impacted iptacopan's PK. | Posted | Mean | Standard Deviation | ng/mL | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
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| Secondary | Cohort 1 and 2: Time to Maximum Observed Plasma Concentration (Tmax) of Iptacopan | PK parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. Tmax is defined as the time to reach maximum (peak) observed concentration following a dose. Actual sampling times were considered for the calculation of PK parameters. | Participants in the PK analysis set (PAS) who had an available value for the outcome measure on the assessed study day. PAS consisted of all participants with at least one available valid PK concentration measurement, who received any study drug and with no protocol deviations or AEs which may have impacted iptacopan's PK. | Posted | Median | Full Range | hours | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
|
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| Secondary | Cohort 1 and 2: Area Under the Plasma Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) of Iptacopan | PK parameters were calculated based on iptacopan plasma concentrations by using non-compartmental methods. The linear trapezoidal method was used for area under the curve (AUC) calculation. | Participants in the PK analysis set (PAS) who had an available value for the outcome measure on the assessed study day. PAS consisted of all participants with at least one available valid PK concentration measurement, who received any study drug and with no protocol deviations or AEs which may have impacted iptacopan's PK. | Posted | Mean | Standard Deviation | hr*ng/mL | Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part A |
|
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| Secondary | Cohort 1 and 2: Trough Plasma Concentration (Ctrough) of Iptacopan | Ctrough is defined as the concentration reached immediately before the next dose is administered. All drug concentrations below the lower limit of quantification were treated as zero for the calculation of PK parameters. | Participants in the PK analysis set (PAS) who had an available value for the outcome measure on the assessed study day. PAS consisted of all participants with at least one available valid PK concentration measurement, who received any study drug and with no protocol deviations or AEs which may have impacted iptacopan's PK. | Posted | Mean | Standard Deviation | ng/mL | Pre-dose on Day 15, 29 and 57 of Part A |
|
|
From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 (ITP) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP) | 0 | 9 | 1 | 9 | 7 | 9 |
| EG001 | Cohort 2 (CAD) | Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD) | 0 | 10 | 1 | 10 | 9 | 10 |
| EG002 | All Patients | All patients in the study | 0 | 19 | 2 | 19 | 16 | 19 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Blood creatinine increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (27.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Neutrophilia | Blood and lymphatic system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Thyroglossal cyst | Congenital, familial and genetic disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Gingival bleeding | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Haemorrhoids | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Fungal foot infection | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (27.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (27.0) | Systematic Assessment |
| |
| Immunisation reaction | Injury, poisoning and procedural complications | MedDRA (27.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Blood iron increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Reverse tri-iodothyronine increased | Investigations | MedDRA (27.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Breast cancer recurrent | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Head discomfort | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Depressed mood | Psychiatric disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Hair texture abnormal | Skin and subcutaneous tissue disorders | MedDRA (27.0) | Systematic Assessment |
| |
| Petechiae | Skin and subcutaneous tissue disorders | MedDRA (27.0) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. Novartis does not prohibit any investigator from publishing. Any publications from a single site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 | Novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 21, 2024 | Mar 4, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D000744 | Anemia, Hemolytic, Autoimmune |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
Not provided
Not provided
| ID | Term |
|---|---|
| C000730766 | iptacopan |
| D015507 | Drugs, Investigational |
| ID | Term |
|---|---|
| D004364 | Pharmaceutical Preparations |
Not provided
Not provided
| Study terminated by sponsor |
|
|
|
|
|
|
|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP) |
|
|
|
|
|
|
|
| Units | Counts |
|---|
| Participants |
|
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