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New drug iptacopan tested in Real-Life PNH patients

NCT ID NCT07229235

Recruiting now Knowledge-focused Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looks at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The goal is to understand the drug's real-world benefits and safety outside of controlled clinical trials.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
iptacopan
What this could lead to
If successful, this study could confirm that iptacopan works well in everyday medical practice for PNH patients, potentially improving anemia and reducing the need for blood transfusions.
What could go wrong
This is an observational study, not a controlled trial, so results may be influenced by factors like patient selection or concurrent treatments. It is also small (50 patients) and limited to Italy, so findings may not apply broadly.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA, PNH

Contacts and locations

Locations

  • Novartis Investigative Site

    RECRUITING

    Alessandria, AL, 15121, Italy

  • Novartis Investigative Site

    RECRUITING

    Avellino, AV, 83100, Italy

  • Novartis Investigative Site

    RECRUITING

    Brescia, BS, 25123, Italy

  • Novartis Investigative Site

    RECRUITING

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    RECRUITING

    Genova, GE, 16132, Italy

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20122, Italy

  • Novartis Investigative Site

    RECRUITING

    Milan, MI, 20132, Italy

  • Novartis Investigative Site

    RECRUITING

    Palermo, PA, 90146, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00161, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00168, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Bassano del Grappa, VI, 36061, Italy

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