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Clinical Trials in Italy / NCT07229235
Recruiting Observational

REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT07229235 · tracked via the Priya Life Science Italy tracker
Sponsor
Novartis Pharmaceuticals
Phase
Observational
Started
2026-01-13
Last updated
2026-06-25

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Study 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. 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

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion criteria: 1. 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 2. Male and female, 3. ≥ 18 years of age, 4. documented diagnosis of PNH, 5. followed in Italian clinical sites, 6. 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)) Exclusion criteria: 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).

Primary outcome measure(s)

  • absolute change in Hb level post iptacopan initiation — 12 months
    12-month absolute change in hemoglobin (Hb) level post iptacopan initiation
  • Proportion of patients with no RBC transfusions — 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.

Trial sites (12)

FacilityCityRegionStatus
Novartis Investigative Site Alessandria AL Recruiting
Novartis Investigative Site Avellino AV Recruiting
Novartis Investigative Site Brescia BS Recruiting
Novartis Investigative Site Florence FI Recruiting
Novartis Investigative Site Genova GE Recruiting
Novartis Investigative Site Milan MI Recruiting
Novartis Investigative Site Milan MI Recruiting
Novartis Investigative Site Palermo PA Recruiting
Novartis Investigative Site Roma RM Recruiting
Novartis Investigative Site Roma RM Recruiting
Novartis Investigative Site Torino TO Recruiting
Novartis Investigative Site Bassano del Grappa VI Recruiting

More Novartis Pharmaceuticals trials in Italy

Phase 3 Study of Pelabresib (CPI-0610) in Myelofibrosis (MF) (MANIFEST-2) Active, not recruiting
Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study Recruiting
Study of PIT565 in Relapsed and/or Refractory B-cell Malignancies Active, not recruiting
A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy Recruiting
A Phase Ib/II Open-label Study of AMO959 With Lutetium (177Lu) Vipivotide Tetraxetan (AAA617) in Combination With ARPI in Adult Participants With PSMA-positive mCRPC Recruiting

Other trials for the same condition

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients Recruiting
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07229235 on ClinicalTrials.gov ↗ ← All trials in Italy