REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorNovartis Pharmaceuticals
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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,
Disqualifiers
None
Trial population
Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
50 Participants
are grouped into 1 trial groupGroup A: Iptacopan
Trial outcomes
Primary outcomes
1
absolute change in Hb level post iptacopan initiation
12-month absolute change in hemoglobin (Hb) level post iptacopan initiation
Time frame
12 months2
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.
Time frame
12 monthsSecondary outcomes
1
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.
Time frame
date of iptacopan start, day 02
Absolute change in Hb level post iptacopan initiation
24-month absolute change in Hb level post iptacopan initiation
Time frame
date of iptacopan start (day 0), 24 months3
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
Time frame
24 months4
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
Time frame
12 month, 24 monthSponsors and contacts
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Source ClinicalTrials.gov