Trial Outcomes & Findings for Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders (NCT NCT05086744)
NCT ID: NCT05086744
Last Updated: 2025-10-16
Results Overview
A study participant with ITP was considered a responder if all the below criteria were met: 1. Platelet count of ≥50 k/μL sustained for at least 2 consecutive weeks during the main, 12-week treatment part 2. Absence of rescue therapy or prohibited medications to treat ITP 3. Lack of treatment discontinuation
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
19 participants
Primary outcome timeframe
Up to 12 weeks (Part A)
Results posted on
2025-10-16
Participant Flow
Participants took part in 8 investigative sites in 6 countries.
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.
Participant milestones
| Measure |
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
Cohort 2 (CAD)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD)
|
|---|---|---|
|
Part A
STARTED
|
9
|
10
|
|
Part A
PD Analysis Set
|
8
|
10
|
|
Part A
ITP - sC5b-9 Low
|
5
|
0
|
|
Part A
ITP - sC5b-9 High
|
4
|
0
|
|
Part A
COMPLETED
|
4
|
9
|
|
Part A
NOT COMPLETED
|
5
|
1
|
|
Part B
STARTED
|
1
|
8
|
|
Part B
ITP - sC5b-9 High
|
1
|
0
|
|
Part B
ITP - sC5b-9 Low
|
0
|
0
|
|
Part B
COMPLETED
|
0
|
0
|
|
Part B
NOT COMPLETED
|
1
|
8
|
Reasons for withdrawal
| Measure |
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
Cohort 2 (CAD)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD)
|
|---|---|---|
|
Part A
Adverse Event
|
0
|
1
|
|
Part A
Lack of Efficacy
|
3
|
0
|
|
Part A
Protocol deviation
|
1
|
0
|
|
Part A
Subject decision
|
1
|
0
|
|
Part B
Adverse Event
|
0
|
1
|
|
Part B
Lack of Efficacy
|
0
|
1
|
|
Part B
Study terminated by sponsor
|
1
|
6
|
Baseline Characteristics
Study specific characteristic for participants in Cohort 1 only.
Baseline characteristics by cohort
| Measure |
Cohort 1 (ITP)
n=9 Participants
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
Cohort 2 (CAD)
n=10 Participants
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD)
|
Total
n=19 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
44.2 years
STANDARD_DEVIATION 20.77 • n=9 Participants
|
66.7 years
STANDARD_DEVIATION 10.01 • n=10 Participants
|
56.1 years
STANDARD_DEVIATION 19.36 • n=19 Participants
|
|
Age, Customized
18 - <65 years
|
7 Participants
n=9 Participants
|
5 Participants
n=10 Participants
|
12 Participants
n=19 Participants
|
|
Age, Customized
65 - <85 years
|
2 Participants
n=9 Participants
|
5 Participants
n=10 Participants
|
7 Participants
n=19 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=9 Participants
|
10 Participants
n=10 Participants
|
15 Participants
n=19 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=9 Participants
|
0 Participants
n=10 Participants
|
4 Participants
n=19 Participants
|
|
Race/Ethnicity, Customized
Asian
|
3 Participants
n=9 Participants
|
0 Participants
n=10 Participants
|
3 Participants
n=19 Participants
|
|
Race/Ethnicity, Customized
White
|
6 Participants
n=9 Participants
|
10 Participants
n=10 Participants
|
16 Participants
n=19 Participants
|
|
Platelets
|
14.6 platelets*10^9/liter
STANDARD_DEVIATION 10.53 • n=9 Participants • Study specific characteristic for participants in Cohort 1 only.
|
—
|
14.6 platelets*10^9/liter
STANDARD_DEVIATION 10.53 • n=9 Participants • Study specific characteristic for participants in Cohort 1 only.
|
|
Hemoglobin
|
—
|
86.7 gram/liter
STANDARD_DEVIATION 9.06 • n=10 Participants • Study specific characteristic for participants in Cohort 2 only.
|
86.7 gram/liter
STANDARD_DEVIATION 9.06 • n=10 Participants • Study specific characteristic for participants in Cohort 2 only.
|
PRIMARY outcome
Timeframe: Up to 12 weeks (Part A)Population: 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.
A study participant with ITP was considered a responder if all the below criteria were met: 1. Platelet count of ≥50 k/μL sustained for at least 2 consecutive weeks during the main, 12-week treatment part 2. Absence of rescue therapy or prohibited medications to treat ITP 3. Lack of treatment discontinuation
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=3 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=5 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
n=8 Participants
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: Participants from Cohort 2 in the PD analysis set.
A study participant with CAD was considered a responder if all the below criteria were met: 1. Hemoglobin level increase of ≥1.5 g/dL above baseline sustained for at least 2 consecutive weeks during the main, 12-week treatment part 2. Absence of rescue therapy or prohibited medications to treat CAD 3. Lack of treatment discontinuation
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=10 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response
|
5 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks (Part A)Population: 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.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: Participants from Cohort 2 in the PD analysis set who were responders.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=5 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline
|
29.0 days
Interval 23.0 to 63.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks (Part A)Population: 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.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: Participants from Cohort 2 in the PD analysis set who were responders.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=5 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Duration of Time During Which Hemoglobin Level Remains ≥1.5 g/dL Above Baseline Without the Use of Rescue Therapy
|
56.0 days
Interval 28.0 to 63.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=1 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=2 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
n=3 Participants
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline
Absolute platelet counts increase <50 k/uL
|
1 Participants
|
2 Participants
|
3 Participants
|
|
Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline
Absolute platelet counts increase >=50 k/uL and <100 k/uL
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline
Absolute platelet counts increase >=100 k/uL and <150 k/uL
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline
Absolute platelet counts increase >=150 k/uL
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: Participants from Cohort 2 in the PD analysis set who did not use rescue therapy in the treatment period of Part A.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline
Hb increase by <1.0 g/dL
|
2 Participants
|
—
|
—
|
|
Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline
Hb increase by >=1.0 g/dL and <1.5 g/dL
|
1 Participants
|
—
|
—
|
|
Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline
Hb increase by =>1.5 g/dL and <2 g/dL
|
2 Participants
|
—
|
—
|
|
Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline
Hb increase by =>2 g/dL
|
4 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 12 weeks (Part A)Population: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=3 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=5 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
n=8 Participants
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 (ITP): Need for Rescue Therapy During Part A
No
|
1 Participants
|
2 Participants
|
3 Participants
|
|
Cohort 1 (ITP): Need for Rescue Therapy During Part A
Yes
|
2 Participants
|
3 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Up to 12 weeks (Part A)Population: Participants from Cohort 2 in the PD analysis set.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=10 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Need for Rescue Therapy During Part A
Yes
|
1 Participants
|
—
|
—
|
|
Cohort 2 (CAD): Need for Rescue Therapy During Part A
No
|
9 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: 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.
LDH was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=6 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Change From Baseline in Lactate Dehydrogenase (LDH)
|
-277.83 Units/liter (U/L)
Standard Deviation 88.966
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: 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.
Total bilirubin was measured in serum samples to assess the effect of treatment with iptacopan on relevant disease biomarkers.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=8 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Change From Baseline in Total Bilirubin
|
-15.63 micromole/liter (μmol/L)
Standard Deviation 12.979
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: 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.
Reticulocyte count was measured in blood samples to assess the effect of treatment with iptacopan on relevant disease biomarkers.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=8 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Change From Baseline in Reticulocyte Count
|
-37.45 reticulocytes * 10^9/liter
Standard Deviation 17.205
|
—
|
—
|
SECONDARY outcome
Timeframe: Baseline, up to 12 weeks (Part A)Population: 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.
Haptoglobin was measured in serum or plasma samples to assess the effect of treatment with iptacopan on relevant disease biomarkers.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 2 (CAD): Change From Baseline in Haptoglobin
|
0.12 gram/liter (g/L)
Standard Deviation 0.236
|
—
|
—
|
SECONDARY outcome
Timeframe: From first dose of study treatment to 7 days after last dose, up to approximately 43 weeks (Cohort 1) and 103 weeks (Cohort 2)Population: All participants who received at least one dose of study treatment.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=10 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
AEs
|
7 Participants
|
9 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Treatment-related AEs
|
2 Participants
|
3 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Severe AEs
|
1 Participants
|
0 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Treatment-related severe AEs
|
1 Participants
|
0 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
SAEs
|
0 Participants
|
1 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Treatment-related SAEs
|
0 Participants
|
0 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Fatal SAEs
|
0 Participants
|
0 Participants
|
—
|
|
Cohort 1 and 2: Number of Participants With AEs and SAEs During the On-treatment Period in Part A and B
Treatment-related fatal SAEs
|
0 Participants
|
0 Participants
|
—
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part APopulation: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=7 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 and 2: Maximum Observed Plasma Concentration (Cmax) of Iptacopan
Day 15
|
3190.0 ng/mL
Standard Deviation 465.00
|
4800.0 ng/mL
Standard Deviation 838.00
|
—
|
|
Cohort 1 and 2: Maximum Observed Plasma Concentration (Cmax) of Iptacopan
Day 57
|
2940.0 ng/mL
Standard Deviation 1020.00
|
4420.0 ng/mL
Standard Deviation 1300.00
|
—
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part APopulation: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=7 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 and 2: Time to Maximum Observed Plasma Concentration (Tmax) of Iptacopan
Day 15
|
2 hours
Interval 0.75 to 5.15
|
1 hours
Interval 1.0 to 2.0
|
—
|
|
Cohort 1 and 2: Time to Maximum Observed Plasma Concentration (Tmax) of Iptacopan
Day 57
|
2 hours
Interval 2.0 to 2.0
|
1.97 hours
Interval 1.0 to 2.0
|
—
|
SECONDARY outcome
Timeframe: Pre-dose, 0.5, 2, 4 and 6 hours after iptacopan administration on Day 15 and Day 57 of Part APopulation: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=7 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
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
Day 15
|
24200.0 hr*ng/mL
Standard Deviation 6110.00
|
31100.0 hr*ng/mL
Standard Deviation 5830.00
|
—
|
|
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
Day 57
|
21300.0 hr*ng/mL
Standard Deviation 5280.00
|
28200.0 hr*ng/mL
Standard Deviation 6880.00
|
—
|
SECONDARY outcome
Timeframe: Pre-dose on Day 15, 29 and 57 of Part APopulation: 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.
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.
Outcome measures
| Measure |
Cohort 1 (ITP) - sC5b-9 High
n=8 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and high complement activation (i.e., high sC5b-9 levels)
|
Cohort 1 (ITP) - sC5b-9 Low
n=9 Participants
Iptacopan 200 mg b.i.d. in participants with primary ITP and low complement activation (i.e., low sC5b-9 levels)
|
Cohort 1 (ITP)
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
|---|---|---|---|
|
Cohort 1 and 2: Trough Plasma Concentration (Ctrough) of Iptacopan
Day 57
|
1680.0 ng/mL
Standard Deviation 758.00
|
1410.0 ng/mL
Standard Deviation 320.00
|
—
|
|
Cohort 1 and 2: Trough Plasma Concentration (Ctrough) of Iptacopan
Day 15
|
1670.0 ng/mL
Standard Deviation 1320.00
|
1980.0 ng/mL
Standard Deviation 1720.00
|
—
|
|
Cohort 1 and 2: Trough Plasma Concentration (Ctrough) of Iptacopan
Day 29
|
1670.0 ng/mL
Standard Deviation 927.00
|
1330.0 ng/mL
Standard Deviation 337.00
|
—
|
Adverse Events
Cohort 1 (ITP)
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Cohort 2 (CAD)
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
All Patients
Serious events: 2 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1 (ITP)
n=9 participants at risk
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
Cohort 2 (CAD)
n=10 participants at risk
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD)
|
All Patients
n=19 participants at risk
All patients in the study
|
|---|---|---|---|
|
Investigations
Blood creatinine increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Nervous system disorders
Haemorrhage intracranial
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
Other adverse events
| Measure |
Cohort 1 (ITP)
n=9 participants at risk
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary immune thrombocytopenia (ITP)
|
Cohort 2 (CAD)
n=10 participants at risk
Iptacopan 200 mg twice daily (b.i.d.) in participants with primary cold agglutinin disease (CAD)
|
All Patients
n=19 participants at risk
All patients in the study
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Leukocytosis
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Blood and lymphatic system disorders
Neutrophilia
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Congenital, familial and genetic disorders
Thyroglossal cyst
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Ear and labyrinth disorders
Tinnitus
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Ear and labyrinth disorders
Vertigo positional
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Gingival bleeding
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Haematochezia
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Nausea
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
15.8%
3/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
General disorders
Asthenia
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
30.0%
3/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
15.8%
3/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
General disorders
Pyrexia
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Bronchitis
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
COVID-19
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Diverticulitis
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Ear infection
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Fungal foot infection
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Gastroenteritis
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Oral herpes
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Infections and infestations
Upper respiratory tract infection
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Injury, poisoning and procedural complications
Contusion
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Injury, poisoning and procedural complications
Immunisation reaction
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Investigations
Blood creatinine increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Investigations
Blood iron increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Investigations
Reverse tri-iodothyronine increased
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer recurrent
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Nervous system disorders
Dizziness
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Nervous system disorders
Head discomfort
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Nervous system disorders
Headache
|
22.2%
2/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
30.0%
3/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
26.3%
5/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Psychiatric disorders
Depressed mood
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Renal and urinary disorders
Renal impairment
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Reproductive system and breast disorders
Heavy menstrual bleeding
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
20.0%
2/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
15.8%
3/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
22.2%
2/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.5%
2/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Skin and subcutaneous tissue disorders
Eczema
|
11.1%
1/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Skin and subcutaneous tissue disorders
Hair texture abnormal
|
0.00%
0/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
10.0%
1/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
5.3%
1/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
33.3%
3/9 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
0.00%
0/10 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
15.8%
3/19 • From first dose of study treatment to 30 days after last dose, up to approximately 46 weeks (Cohort 1) and 106 weeks (Cohort 2)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER