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New drug iptacopan tested in Real-Life PNH patients

NCT ID NCT07229235

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2026

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Italian population of adult patients with PNH either naïve to iptacopan or transitioning from the Managed Access Program (MAP) starting from January 2023

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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).

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    12 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.