Immune Thrombocytopenia (ITP)Phase 2NCT05086744
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders
A Phase 2 study testing Iptacopan in people with Immune Thrombocytopenia (ITP) and Cold Agglutinin Disease (CAD), run by Novartis Pharmaceuticals at 8 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 120 years
- Conditions the study is for
- Immune Thrombocytopenia (ITP), Cold Agglutinin Disease (CAD)
- What is being tested?
- Iptacopan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 8 sitesin 6 countries
- Study dates
- 21 Dec 2021to 17 May 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 120 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All Cohorts:
- Written informed consent
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections was required and vaccination against Haemophilus influenzae infection is recommended prior to the start of treatment.
- Weight of at least 35 kg
- Cohort 1 specific inclusion criteria:
- Participants with a diagnosis of persistent or chronic primary ITP
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- Participants must have received at least 1 unique prior therapy administered with the intention to treat ITP
- Sustained thrombocytopenia
- Cohort 2 specific inclusion criteria:
- Participants with a diagnosis of primary CAD
- Participants must have received at least 1 unique prior therapy administered with the intention to treat CAD
- Laboratory evidence of ongoing hemolysis
- Sustained anemia
Who cannot join
- All cohorts:
- Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations
- Past or concomitant use of medications prohibited by the protocol
- Known or suspected hereditary or acquired complement deficiency
- History of primary or secondary immunodeficiency, including a positive HIV test result
- Chronic infection with Hepatitis B or C virus
Show all 22Show fewer
- History of recurrent invasive infections caused by encapsulated organisms, including Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae
- Presence or suspicion of any active infection within 14 days prior to first study drug administration.
- Any medical condition deemed likely to interfere with the participant's participation in the study
- Any malignant disease diagnosed within the past 5 years, with the exception of localized non-melanoma skin cancer, in situ cervical cancer, or, for CAD, a low-grade lymphoproliferative disorder.
- History of bone marrow/hematopoietic stem cell or solid organ transplantation.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of investigational drug and for 1 week after last iptacopan dose
- Active severe bleeding or history of intracranial hemorrhage.
- Liver disease, or liver injury as indicated by abnormal liver function tests.
- Severe concurrent comorbidities of unstable medical conditions.
- Cohort 1 specific exclusion criteria:
- Secondary ITP, as may arise in the setting of certain autoimmune disorders, immunodeficiency syndromes, infections, malignancies, and drug treatments
- No ITP-directed background therapy permitted, with the exception of either a thrombopoietin receptor agonist or low-dose corticosteroid, as long as stable dosage for at least 4 weeks prior to first iptacopan dose
- Abnormal coagulation screening labs
- Cohort 2 specific exclusion criteria:
- Secondary cold agglutinin syndrome, as may arise in the setting of certain infections, autoimmune disorders, and malignancies (with the exception of a low-grade lymphoproliferative disorder)
- No CAD-directed background therapy permitted
Trial locations and contacts
8 sites in 6 countries.
8 sites in 6 countries
Spain3 sites
Novartis Investigative Site
Barcelona, Catalonia, 08035
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Murcia, 30008
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: An Open-label, Multi-center, Phase 2 Basket Study to Assess Efficacy, Safety and Pharmacokinetics of Iptacopan (LNP023) in Participants With Autoimmune Benign Hematological Disorders
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.
Read the detailed description
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.
- Being tested
- Iptacopan
- Keywords
- LNP023, Iptacopan, Immune thrombocytopenia, Cold agglutinin disease, Autoimmune benign hematological disorders
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Anemia, Hemolytic, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IptacopanDrug
Also called Investigational new drug, company code: LNP023
Iptacopan 200 mg BID given orally (capsule)
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan 200 mg BID | Experimental | Iptacopan 200 mg twice daily (BID) in participants with primary ITP and primary CAD Receives: Drug: Iptacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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:
- Platelet count of ≥50 k/μL sustained for at least 2 consecutive weeks during the main, 12-week treatment part
- Absence of rescue therapy or prohibited medications to treat ITP
- Lack of treatment discontinuation
Measured: 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:
- Hemoglobin level increase of ≥1.5 g/dL above baseline sustained for at least 2 consecutive weeks during the main, 12-week treatment part
- Absence of rescue therapy or prohibited medications to treat CAD
- Lack of treatment discontinuation
Measured: Baseline, up to 12 weeks (Part A)
Secondary outcomes (17)
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.
Measured: Up to 12 weeks (Part A)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Pre-dose on Day 15, 29 and 57 of Part A
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2021
- Study start21 Dec 2021
- Primary completion20 Sep 2023
- Study completion17 May 2024
Why it stopped: Business decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a basket study with different Cohorts.
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05086744
- Sponsor's protocol ID
- CLNP023L12201
- Other IDs
- 2021-002039-40
- Status last verified
- Oct 2025
- First submitted
- 28 Sep 2021
- Last updated
- 16 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
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.
PubMed 41366634
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multi-center, Phase 2 Basket Study to Assess Efficacy, Safety and Pharmacokinetics of Iptacopan (LNP023) in Participants With Autoimmune Benign Hematological Disorders
- ClinicalTrials.gov ID
- NCT05086744
- Acronym / sponsor ID
- CLNP023L12201
- Keywords
- LNP023, Iptacopan, Immune thrombocytopenia, Cold agglutinin disease, Autoimmune benign hematological disorders
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business decision
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 21 Dec 2021 (actual)
- Primary completion
- 20 Sep 2023 (actual)
- Study completion
- 17 May 2024 (actual)
- First posted
- 21 Oct 2021 (actual)
- Results
- Posted 19 Mar 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a basket study with different Cohorts.
| Arm | Type | Receives |
|---|---|---|
| Iptacopan 200 mg BID | Experimental | Drug: IptacopanIptacopan 200 mg twice daily (BID) in participants with primary ITP and primary CAD |
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Written informed consent
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections was required and vaccination against Haemophilus influenzae infection is recommended prior to the start of treatment.
- Weight of at least 35 kg
- Participants with a diagnosis of persistent or chronic primary ITP
- Participants must have received at least 1 unique prior therapy administered with the intention to treat ITP
Key exclusion criteria (22 listed)
- Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations
- Past or concomitant use of medications prohibited by the protocol
- Known or suspected hereditary or acquired complement deficiency
- History of primary or secondary immunodeficiency, including a positive HIV test result
- Chronic infection with Hepatitis B or C virus
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IptacopanAlso: Investigational new drug, company code: LNP023Iptacopan 200 mg BID given orally (capsule) | Drug | Iptacopan 200 mg BID |
Primary outcome
Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response
Time frame: Up to 12 weeks (Part A)
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
Cohort 2 (CAD): Number of Participants With a Clinically Meaningful Response
Time frame: Baseline, up to 12 weeks (Part A)
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
17 secondary outcomes
Cohort 1 (ITP): Time to First Platelet Count ≥50 k/μL
Time frame: Up to 12 weeks (Part A)
Cohort 2 (CAD): Time to First Hemoglobin Level ≥1.5 g/dL Above Baseline
Time frame: 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
Time frame: 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
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 1 (ITP): Magnitude of Platelet Count Increase From Baseline
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 2 (CAD): Magnitude of Hemoglobin Increase From Baseline
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 1 (ITP): Need for Rescue Therapy During Part A
Time frame: Up to 12 weeks (Part A)
Cohort 2 (CAD): Need for Rescue Therapy During Part A
Time frame: Up to 12 weeks (Part A)
Cohort 2 (CAD): Change From Baseline in Lactate Dehydrogenase (LDH)
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 2 (CAD): Change From Baseline in Total Bilirubin
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 2 (CAD): Change From Baseline in Reticulocyte Count
Time frame: Baseline, up to 12 weeks (Part A)
Cohort 2 (CAD): Change From Baseline in Haptoglobin
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Pre-dose on Day 15, 29 and 57 of Part A
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41366634Roth 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.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Iptacopan in people with Immune Thrombocytopenia (ITP) and Cold Agglutinin Disease (CAD), run by Novartis Pharmaceuticals at 8 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Spain, United States, Germany, Italy, South Korea and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cohort 1 (ITP): Number of Participants With a Clinically Meaningful Response (time frame: Up to 12 weeks (Part A)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05086744, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2025.