Hemoglobinuria, ParoxysmalNCT06903234
Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry
An observational study of Hemoglobinuria, Paroxysmal, run by Novartis Pharmaceuticals at 1 site in Switzerland.
Trial summary
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Hemoglobinuria, Paroxysmal
- What is being tested?
- Iptacopan
- How many people
- 200 participants
- Where
- 1 siteSwitzerland
- Study dates
- 31 Mar 2025to 1 Oct 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adult patients with PNH who are enrolled in the IPIG PNH Registry, newly treated with iptacopan.
Who may be able to join
- Signed informed consent to participate in the IPIG PNH Registry
- PNH confirmed by flow cytometry
- Incident users of iptacopan
- Aged at least 18 years at the iptacopan initiation
Who cannot join
- Participation in an interventional clinical trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the Non-interventional IPIG PNH Registry
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
Read the detailed description
This multinational, non-interventional, descriptive single-arm cohort study is based on secondary analysis of data collected within the iptacopan silo of the IPIG PNH Registry (data on iptacopan-treated patients made available to Novartis). This is a non-interventional study utilizing secondary data and is considered a "registry-based study." The IPIG PNH Registry (CT.gov NCT06524726), the parent registry, includes a dedicated drug silo to collect data from patients using iptacopan in routine care.
- Conditions
- Hemoglobinuria, Paroxysmal
- Being tested
- Iptacopan
- Keywords
- Paroxysmal nocturnal hemoglobinuria, Iptacopan
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IptacopanDrug
Also called Fabhalta
Adult patients with PNH treated with iptacopan
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan | Adult patients with PNH treated with iptacopan in routine care. Receives: Drug: Iptacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with infections caused by encapsulated bacteria
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Secondary outcomes (14)
Number of patients with serious infections caused by encapsulated bacteria and all serious infection
To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)
To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection
To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection
To describe the short- and long-term risk of the serious infections in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae) and all serious infection.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia
To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)
To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)
To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)
To describe the short- and long-term risk of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause
To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of death due to any cause (event probability as a function of time)
To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause events per 100 patients -years (incidence rates)
To describe the short- and long-term risk of all-cause mortality in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit
To describe the number of patients receiving mandatory and recommended vaccinations against encapsulated bacteria.
Measured: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious hemolysis following discontinuation of iptacopan
To describe the risk of serious hemolysis following discontinuation of iptacopan in patients with PNH treated with iptacopan in routine clinical practice.
Measured: From the iptacopan discontinuation up to 14 days
Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes
To describe the frequency of use of iptacopan during pregnancy in PNH patients, characteristics of pregnancies exposed to iptacopan and frequency of selected pregnancy and birth outcomes.
Measured: From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2025
- Study start31 Mar 2025
- Primary completion1 Oct 2029 (estimated)
- Study completion1 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemoglobinuria, ParoxysmalStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06903234
- Sponsor's protocol ID
- CLNP023C12003
- Other IDs
- EUPAS1000000457
- Status last verified
- Jun 2026
- First submitted
- 24 Mar 2025
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the Non-interventional IPIG PNH Registry
- ClinicalTrials.gov ID
- NCT06903234
- Acronym / sponsor ID
- CLNP023C12003
- Conditions
- Hemoglobinuria, Paroxysmal
- Keywords
- Paroxysmal nocturnal hemoglobinuria, Iptacopan
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 31 Mar 2025 (actual)
- Primary completion
- 1 Oct 2029 (estimated)
- Study completion
- 1 Oct 2029 (estimated)
- First posted
- 30 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an observational single-arm descriptive cohort study based on the secondary use of data collected on iptacopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
Detailed description
This multinational, non-interventional, descriptive single-arm cohort study is based on secondary analysis of data collected within the iptacopan silo of the IPIG PNH Registry (data on iptacopan-treated patients made available to Novartis). This is a non-interventional study utilizing secondary data and is considered a "registry-based study." The IPIG PNH Registry (CT.gov NCT06524726), the parent registry, includes a dedicated drug silo to collect data from patients using iptacopan in routine care.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Iptacopan | – | Drug: IptacopanAdult patients with PNH treated with iptacopan in routine care. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adult patients with PNH who are enrolled in the IPIG PNH Registry, newly treated with iptacopan.
Key inclusion criteria (4 listed)
- Signed informed consent to participate in the IPIG PNH Registry
- PNH confirmed by flow cytometry
- Incident users of iptacopan
- Aged at least 18 years at the iptacopan initiation
Key exclusion criteria (1 listed)
- Participation in an interventional clinical trial
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: FabhaltaAdult patients with PNH treated with iptacopan | Drug | Iptacopan |
Primary outcome
Number of patients with infections caused by encapsulated bacteria
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Cumulative incidence of infections (event probability as a function of time), caused by encapsulated bacteria
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Number of patients with infections events per 100 participants -years (incidence rates) caused by encapsulated bacteria
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
Number of infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
To describe the risk of infections caused by encapsulated bacteria in patients with PNH treated with iptacopan in routine clinical practice. Infections caused by encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae).
14 secondary outcomes
Number of patients with serious infections caused by encapsulated bacteria and all serious infection
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of serious infections, caused by encapsulated bacteria and all serious infection (event probability as a function of time)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious infections events per 100 patients -years (incidence rates) caused by encapsulated bacteria and all serious infection
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of serious infections episodes per 100 patients -years (occurrence rates) caused by encapsulated bacteria and all serious infection
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, Major adverse vascular events (MAVEs), serious adverse events (SAEs), hyperlipidemia and thrombocytopenia
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia (event probability as a function of time)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia events per 100 patients -years (incidence rates)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of potential breakthrough hemolysis, solid tumors, hematological malignancies, MAVEs, SAEs, hyperlipidemia and thrombocytopenia episodes per 100 patients -years (occurrence rates)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Cumulative incidence of death due to any cause (event probability as a function of time)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with death due to any cause events per 100 patients -years (incidence rates)
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae at each study visit
Time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.
Number of patients with serious hemolysis following discontinuation of iptacopan
Time frame: From the iptacopan discontinuation up to 14 days
Number of patients who became pregnant during treatment with iptacopan, exposure characteristics (e.g. trimester of exposure) and birth outcomes
Time frame: From the Last Menstrual Period to pregnancy outcome (in case of live birth, up to 12 months post delivery)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemoglobinuria, Paroxysmal, run by Novartis Pharmaceuticals at 1 site in Switzerland.
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 4 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Number of patients with infections caused by encapsulated bacteria (time frame: From initiation of iptacopan until discontinuation + 3 days, or end of follow-up, up to 5 years.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Jun 2026.