Paroxysmal Nocturnal HemoglobinuriaPRIORITY- PNHNCT07036718
NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 5 sites in Russia.
Trial summary
This is a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- How many people
- 6 participants
- Where
- 5 sitesRussia
- Study dates
- 29 Jul 2025to 25 Dec 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Enrollment of female and male patients with PNH
Who may be able to join
- Age ≥18 years at the start of iptacopan therapy.
- The patient is undergoing treatment with iptacopan.
- The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.
- Provision of written informed consent.
Who cannot join
- 1. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Russia5 sites
Novartis Investigative Site
Grozny, 364047
Novartis Investigative Site
Moscow, 129110
Novartis Investigative Site
Murmansk, 183047
Novartis Investigative Site
Syktyvkar, Komi, 167904
Novartis Investigative Site
Yuzhno-Sakhalinsk, 693000
About this study
Official title: A Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of Iptacopan in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) in Routine Clinical Practice in the Russian Federation
This is a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, 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.
| Study group | Role | What they receive |
|---|---|---|
| iptacopan | Adults with PNH who started the iptacopan therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with an increase in Hb level ≥ 20 g/l
Number of patients who achieved an increase in Hb level ≥ 20 g/l over 6 months after iptacopan treatment initiation and were Red Blood Cells (RBC) transfusion independent.
Measured: 6 months
Secondary outcomes (8)
Number of patients with an increase in Hb level ≥ 120 g/L
Number of patients who achieved an increase in Hb level ≥ 120 g/L at 12 months after initiation of iptacopan treatment and were RBC transfusion independent
Measured: 12 months
Number of patients with an increase in Hb level ≥ 20 g/L
Number of patients who achieved an increase in Hb level ≥ 20 g/L at 12 months after initiation of iptacopan treatment and was RBC transfusion independent
Measured: 12 months
Change from baseline in reticulocyte count (10⁹/L)
Change from baseline in reticulocyte count (10⁹/L) over 12 months after initiation of iptacopan treatment
Measured: 12 months
Change from baseline in LDH levels (U/L)
Change from baseline in LDH levels (U/L) over 12 months after initiation of iptacopan treatment
Measured: 12 months
Number of patients with an absence of administration of packed-RBC transfusions
Number of patients with an absence of administration of packed-RBC transfusions at 12 months after initiation of iptacopan treatment
Measured: 12 months
Change from baseline in the Hb level (g/L)
Change from baseline in the Hb level (g/L) at 3, 6 and 12 months after initiation of iptacopan treatment
Measured: Baseline, month 3, month 6 and month 12
Number of breakthrough hemolysis (BTH)
Number of breakthrough hemolysis (BTH) cases 12 months following the initiation of iptacopan therapy
Measured: 12 months
Percentage of patients by BMI
Percentage of patients by Body mass index (BMI) (<25, 25.1-29.9, >30)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2025
- Study start29 Jul 2025
- Primary completion25 Dec 2025
- Study completion25 Dec 2025
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07036718
- Sponsor's protocol ID
- CLNP023C1RU03
- Acronym
- PRIORITY- PNH
- Status last verified
- Jul 2026
- First submitted
- 17 Jun 2025
- Last updated
- 17 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Non-interventional Study to Evaluate the Efficacy and Safety of Iptacopan in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) in Routine Clinical Practice in the Russian Federation
- ClinicalTrials.gov ID
- NCT07036718
- Acronym / sponsor ID
- PRIORITY- PNH / CLNP023C1RU03
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH, Paroxysmal nocturnal hemoglobinuria, Iptacopan
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 29 Jul 2025 (actual)
- Primary completion
- 25 Dec 2025 (actual)
- Study completion
- 25 Dec 2025 (actual)
- First posted
- 25 Jun 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 a prospective, non-interventional, multicenter study to evaluate the efficacy and safety of iptacopan in patients with PNH in real-world settings in Russia.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| iptacopan | – | –Adults with PNH who started the iptacopan therapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Enrollment of female and male patients with PNH
Key inclusion criteria (4 listed)
- Age ≥18 years at the start of iptacopan therapy.
- The patient is undergoing treatment with iptacopan.
- The treating physician decided to prescribe iptacopan based on the Summary of Product Characteristics during routine clinical practice, regardless of study participation.
- Provision of written informed consent.
Key exclusion criteria (1 listed)
- 1. Any situations where iptacopan is contraindicated in accordance with the Summary of Product Characteristics.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of patients with an increase in Hb level ≥ 20 g/l
Time frame: 6 months
Number of patients who achieved an increase in Hb level ≥ 20 g/l over 6 months after iptacopan treatment initiation and were Red Blood Cells (RBC) transfusion independent.
8 secondary outcomes
Number of patients with an increase in Hb level ≥ 120 g/L
Time frame: 12 months
Number of patients with an increase in Hb level ≥ 20 g/L
Time frame: 12 months
Change from baseline in reticulocyte count (10⁹/L)
Time frame: 12 months
Change from baseline in LDH levels (U/L)
Time frame: 12 months
Number of patients with an absence of administration of packed-RBC transfusions
Time frame: 12 months
Change from baseline in the Hb level (g/L)
Time frame: Baseline, month 3, month 6 and month 12
Number of breakthrough hemolysis (BTH)
Time frame: 12 months
Percentage of patients by BMI
Time frame: 12 months
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2026.
- Link to study resultsLink provided by the sponsor.
- 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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Paroxysmal Nocturnal Hemoglobinuria, run by Novartis Pharmaceuticals at 5 sites in Russia.
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: stopped early, as of the sponsor's last update on 17 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Russia. 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?
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
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 an increase in Hb level ≥ 20 g/l (time frame: 6 months). 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 17 Aug 2026.