Paroxysmal Nocturnal Hemoglobinuria (PNH)REAL-CARENCT07229235
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
An observational study of Paroxysmal Nocturnal Hemoglobinuria (PNH), run by Novartis Pharmaceuticals at 12 sites in Italy.
Trial summary
This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes. The primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- How many people
- 50 participants
- Where
- 12 sitesItaly
- Study dates
- 13 Jan 2026to 31 Aug 2028
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023
Who may be able to join
- signed informed consent: patient must provide written informed before any study assessment is collected; in case of deceased patients, informed consent is not required as per Art. 110, comma 1 of Legislative Decree n° 196, dated 30 June 2003 as amended by D.lgs. 101/2018 and the "Autorizzazione generale al trattamento dei dati personali effettuato per scopi di ricerca scientifica" n° 9/2016
- Male and female,
- ≥ 18 years of age,
- documented diagnosis of PNH,
- followed in Italian clinical sites,
- on treatment with iptacopan prescribed as per routine medical care (i.e. patients naïve to treatment for whom the decision to start this treatment has already been made on the basis of clinical practice and according to SmPC and AIFA criteria and regardless of inclusion in this study or patients already treated under the Managed Access Program (MAP))
Who cannot join
- 1.Concomitant participation in an interventional clinical study related to PNH or its treatment.
- No other inclusion/exclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
12 sites in 1 country
Italy12 sites, 12 recruiting
- Recruiting
Novartis Investigative Site
Alessandria, AL, 15121
- Recruiting
Novartis Investigative Site
Avellino, AV, 83100
- Recruiting
Novartis Investigative Site
Bassano del Grappa, VI, 36061
- Recruiting
Novartis Investigative Site
Brescia, BS, 25123
- Recruiting
Novartis Investigative Site
Florence, FI, 50134
- Recruiting
Novartis Investigative Site
Genova, GE, 16132
- Recruiting
Novartis Investigative Site
Milan, MI, 20122
- Recruiting
Novartis Investigative Site
Milan, MI, 20132
- Recruiting
Novartis Investigative Site
Palermo, PA, 90146
- Recruiting
Novartis Investigative Site
Roma, RM, 00161
- Recruiting
Novartis Investigative Site
Roma, RM, 00168
- Recruiting
Novartis Investigative Site
Torino, TO, 10126
About this study
Official title: REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.
The primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 months post-initiation, and the proportion of patients who remain free from red blood cell (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, from Day 14 through Month 12 after starting iptacopan.
Read the detailed description
This is a local, multicenter, observational study with retrospective, retro-prospective and prospective cohorts. Adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023 will be enrolled. Primary data will be collected prospectively; secondary data will be retrieved retrospectively from patients previously treated under MAP. A mix of primary and secondary data will be collected in retro-prospective patients. Patients will be indexed on the date of iptacopan initiation and will be followed up for 24 months or until iptacopan discontinuation (due to occurrence of an adverse event, lack of efficacy etc), clinical decision, death, administrative issues, consent withdrawal or loss to follow-up, whichever comes first. The enrollment period is planned to last 15 months, with potential adjustments based on enrollment progress. No treatment decision will be influenced by study participation.
No extra visits, examinations, or procedures are imposed. Questionnaires should be collected at the indicated timepoints whenever possible, but, since the administration of the questionnaires represents an additional procedure not included in routine clinical care, these timepoints are considered indicative only, and patients will not be recalled specifically to complete questionnaires if a routine clinical visit is not scheduled.
- Keywords
- REAL-CARE, ptacopan, paroxysmal nocturnal hemoglobinuria, PNH, NIS
- 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 | Treatment with iptacopan |
What is being measured
The primary outcome is the main question the study is designed to answer.
absolute change in Hb level post iptacopan initiation
12-month absolute change in hemoglobin (Hb) level post iptacopan initiation
Measured: 12 months
Proportion of patients with no RBC transfusions
12-month proportion of patients with no Red Blood Cells (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, after Day 14.
Measured: 12 months
Secondary outcomes (19)
Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population
Descriptive measures of the socio-demographic variables, proportion of PNH cells, clinical symptoms, laboratory parameters, concomitant diseases and treatments and outcomes of PNH patients at iptacopan initiation.
Measured: date of iptacopan start, day 0
Absolute change in Hb level post iptacopan initiation
24-month absolute change in Hb level post iptacopan initiation
Measured: date of iptacopan start (day 0), 24 months
Proportion of patients who didn't need RBC transfusion
24-month proportion of patients who didn't need RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, post iptacopan initiation
Measured: 24 months
Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion
12- and 24-month proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, between Day 14 and month 12 or month 24
Measured: 12 month, 24 month
Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion
12- and 24-month proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, between Day 14 and month 12 or month 24
Measured: 12 month, 24 month
Average number of RBC transfusion
Average number of RBC transfusion, prescribed as per local requirement and based on Investigator's judgment, per patient during 12- and 24-months post iptacopan initiation
Measured: 12 months and 24 months
Percentage change in LDH level post iptacopan initiation
12- and 24-month percentage change in LDH level post iptacopan initiation.
Measured: date of iptacopan start (day 0), month 12, month 24
Change in reticulocyte count post iptacopan initiation.
12- and 24-month change in reticulocyte count post iptacopan initiation.
Measured: date of iptacopan start (day 0), month 12, month 24
Absolute change in bilirubin level post iptacopan initiation.
12- and 24-month absolute change in bilirubin level post iptacopan initiation.
Measured: date of iptacopan start (day 0), month 12, month 24
Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up
Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Proportion of BTH occurrence
Proportion of Breakthrough Hemolysis (BTH) occurrence over 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Type and proportion of Infections occurrence over 12 and 24 months of follow-up
Type and proportion (count and percentage) of Infections occurrence over 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Type and proportion of major adverse vascular events (MAVEs) occurrence
Type and proportion of major adverse vascular events (MAVEs) occurrence over 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Type and proportion of new comorbidities
Type and proportion of new comorbidities occurred within 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Type and proportion of concomitant medications newly prescribed
Type and proportion of concomitant medications newly prescribed during 12 and 24 months of follow-up post iptacopan initiation
Measured: 12 months and 24 months
Absolute change in Medication Adherence Report Scale - 5 item (MARS-5) scores
The MARS-5 questionnaire is a validated self-report questionnaire developed to assess patient adherence to prescribed medication, focusing on both intentional and unintentional non-adherence.
It consists of five items, each rated on a 5-point Likert Scale, from 0 ("Always") to 5 ("Never").
Items evaluate behaviors such as forgetting to take medications, altering the dose or stopping the treatment. The total score ranges from 5 to 25, with higher scores indicating better adherence to treatment.
Measured: date of iptacopan start (day 0), month 12 and month 24
Absolute change from iptacopan initiation in PROMIS-29+2 score.
The Patient-Reported Outcomes Measurement Information System (PROMIS) Profile-29+2 is a validated standardized patient reported outcome measure to assess generic health-related quality of life. The questionnaire covers the following nine health domains: Physical function, Anxiety, Depression, Fatigue, Sleep disturbance, Ability to participate in social roles and activities, Pain interference, Cognitive function and Pain intensity.
The recall period for all items is the past 7 days. Each of the first eight domains is assessed through 4 items using a 5-point Likert scale. Pain intensity is measured with a single item on an 11-point numeric rating scale from 0 ("no pain") to 10 ("worst imaginable pain").
Each domain is scored separately, with T-scores used to represent the severity or level of functioning in each area. Higher scores can indicate either better health (e.g., physical function) or greater severity of symptoms (e.g., anxiety, depression).
Measured: date of iptacopan start (day 0), month 12 and month 24
Absolute change from iptacopan initiation in WPAI:SHP score.
Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP) is a validated patient-reported outcome measure designed to assess the impact of a specific health problem on work and daily activities. In this study, the questionnaire has been customized for PNH. It includes six questions grouped into four key domains: Absenteeism (work time missed due to PNH), Presenteeism (impairment while working due to PNH), Overall work productivity loss (combined absenteeism and presenteeism), Activity impairment (impact of PNH on regular daily activities outside of work).
The recall period is the past 7 days. Responses are used to calculate the percentage of work time missed and the level of impairment experienced by the patient. Higher scores represent greater impairment and reduced productivity.
Measured: date of iptacopan start (day 0), month 12 and month 24
Number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of AEs related to the study treatment
Healthcare resources utilization:
The number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of adverse events (AEs) related to the study treatment will be documented.
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2025
- Study start13 Jan 2026
- Primary completion31 Aug 2028 (estimated)
- Study completion31 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal Hemoglobinuria (PNH)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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 50 participants (estimated)
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
- NCT07229235
- Sponsor's protocol ID
- CLNP023C1IT01
- Acronym
- REAL-CARE
- Status last verified
- Jun 2026
- First submitted
- 30 Oct 2025
- Last updated
- 25 Jun 2026
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study
- ClinicalTrials.gov ID
- NCT07229235
- Acronym / sponsor ID
- REAL-CARE / CLNP023C1IT01
- Keywords
- REAL-CARE, ptacopan, paroxysmal nocturnal hemoglobinuria, PNH, NIS
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 13 Jan 2026 (actual)
- Primary completion
- 31 Aug 2028 (estimated)
- Study completion
- 31 Aug 2028 (estimated)
- First posted
- 14 Nov 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 study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.
The primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 months post-initiation, and the proportion of patients who remain free from red blood cell (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, from Day 14 through Month 12 after starting iptacopan.
Detailed description
This is a local, multicenter, observational study with retrospective, retro-prospective and prospective cohorts. Adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023 will be enrolled. Primary data will be collected prospectively; secondary data will be retrieved retrospectively from patients previously treated under MAP. A mix of primary and secondary data will be collected in retro-prospective patients. Patients will be indexed on the date of iptacopan initiation and will be followed up for 24 months or until iptacopan discontinuation (due to occurrence of an adverse event, lack of efficacy etc), clinical decision, death, administrative issues, consent withdrawal or loss to follow-up, whichever comes first. The enrollment period is planned to last 15 months, with potential adjustments based on enrollment progress. No treatment decision will be influenced by study participation.
No extra visits, examinations, or procedures are imposed. Questionnaires should be collected at the indicated timepoints whenever possible, but, since the administration of the questionnaires represents an additional procedure not included in routine clinical care, these timepoints are considered indicative only, and patients will not be recalled specifically to complete questionnaires if a routine clinical visit is not scheduled.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Iptacopan | – | –Treatment with iptacopan |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023
Key inclusion criteria (6 listed)
- signed informed consent: patient must provide written informed before any study assessment is collected; in case of deceased patients, informed consent is not required as per Art. 110, comma 1 of Legislative Decree n° 196, dated 30 June 2003 as amended by D.lgs. 101/2018 and the "Autorizzazione generale al trattamento dei dati personali effettuato per scopi di ricerca scientifica" n° 9/2016
- Male and female,
- ≥ 18 years of age,
- documented diagnosis of PNH,
- followed in Italian clinical sites,
Key exclusion criteria (2 listed)
- 1.Concomitant participation in an interventional clinical study related to PNH or its treatment.
- No other inclusion/exclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).
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
absolute change in Hb level post iptacopan initiation
Time frame: 12 months
12-month absolute change in hemoglobin (Hb) level post iptacopan initiation
Proportion of patients with no RBC transfusions
Time frame: 12 months
12-month proportion of patients with no Red Blood Cells (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, after Day 14.
19 secondary outcomes
Socio-demographic and clinical variables of Paroxysmal Nocturnal Hemoglobinuria population
Time frame: date of iptacopan start, day 0
Absolute change in Hb level post iptacopan initiation
Time frame: date of iptacopan start (day 0), 24 months
Proportion of patients who didn't need RBC transfusion
Time frame: 24 months
Proportion of patients with Hb levels ≥ 12 g/dL AND no RBC transfusion
Time frame: 12 month, 24 month
Proportion of patients with change in Hb levels ≥ 2 g/dL post iptacopan initiation AND no RBC transfusion
Time frame: 12 month, 24 month
Average number of RBC transfusion
Time frame: 12 months and 24 months
Percentage change in LDH level post iptacopan initiation
Time frame: date of iptacopan start (day 0), month 12, month 24
Change in reticulocyte count post iptacopan initiation.
Time frame: date of iptacopan start (day 0), month 12, month 24
Absolute change in bilirubin level post iptacopan initiation.
Time frame: date of iptacopan start (day 0), month 12, month 24
Frequency of adverse events (AE) and serious adverse events (SAE) over 12 and 24 months of follow-up
Time frame: 12 months and 24 months
Proportion of BTH occurrence
Time frame: 12 months and 24 months
Type and proportion of Infections occurrence over 12 and 24 months of follow-up
Time frame: 12 months and 24 months
Type and proportion of major adverse vascular events (MAVEs) occurrence
Time frame: 12 months and 24 months
Type and proportion of new comorbidities
Time frame: 12 months and 24 months
Type and proportion of concomitant medications newly prescribed
Time frame: 12 months and 24 months
Absolute change in Medication Adherence Report Scale - 5 item (MARS-5) scores
Time frame: date of iptacopan start (day 0), month 12 and month 24
Absolute change from iptacopan initiation in PROMIS-29+2 score.
Time frame: date of iptacopan start (day 0), month 12 and month 24
Absolute change from iptacopan initiation in WPAI:SHP score.
Time frame: date of iptacopan start (day 0), month 12 and month 24
Number of hospitalizations, emergency room visits, and hospital-based outpatient visits attributable to the management of AEs related to the study treatment
Time frame: Up to 24 months
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 25 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.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
12 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 (PNH), run by Novartis Pharmaceuticals at 12 sites in Italy.
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 6 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 25 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 12 sites in Italy. 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?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
absolute change in Hb level post iptacopan initiation (time frame: 12 months). 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?
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 25 Jun 2026.