Real-world use of iptacopan for PNH in Italy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter observational program enrolling adults with PNH who are starting iptacopan or who were treated under the Managed Access Program, using retrospective, retro-prospective, and prospective cohorts. Primary data will be collected prospectively while secondary data will be retrieved from medical records for patients previously treated under the MAP. Patients are indexed at the date of iptacopan initiation and followed for up to 24 months, with the primary outcome being change in hemoglobin at 12 months and the proportion free from red blood cell transfusion from Day 14 through Month 12. No extra visits or procedures are mandated beyond routine care, and patient questionnaires are collected when possible but are not required.

Why it matters

Who can join: the full rules

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).

Where it's running

• Novartis Investigative Site
  Alessandria, Al, Italy Recruiting
• Novartis Investigative Site
  Avellino, Av, Italy Recruiting
• Novartis Investigative Site
  Brescia, Bs, Italy Recruiting
• Novartis Investigative Site
  Florence, Fi, Italy Recruiting
• Novartis Investigative Site
  Genova, Ge, Italy Recruiting
• Novartis Investigative Site
  Milan, Mi, Italy Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: +41613241111

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.