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Real-World ravulizumab data: hope for PNH patients in poland

NCT ID NCT06633536

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 64 Polish adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking ravulizumab as part of their routine care. Researchers will track blood markers like LDH and how many patients need blood transfusions over time. The goal is to see if the drug works as well in everyday life as it did in 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
ravulizumab
What this could lead to
If successful, this study could confirm that ravulizumab works well in everyday practice for managing PNH, supporting its continued use.
What could go wrong
This is an observational study, not a controlled trial, so results may be less reliable. It only includes 64 patients in Poland, 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

64 people

The number who actually took part.

Started

Feb 2025

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

Adult (aged ≥18) patients with PNH

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. Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland. 2. Patients willing to participate in the study and signed ICF. Exclusion Criteria: 1. Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP. 2. Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

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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.

Contacts and locations

Locations

  • Research Site

    Bialystok, Poland

  • Research Site

    Brzozów, Poland

  • Research Site

    Bydgoszcz, Poland

  • Research Site

    Gdansk, Poland

  • Research Site

    Krakow, Poland

  • Research Site

    Lodz, Poland

  • Research Site

    Lublin, Poland

  • Research Site

    Opole, Poland

  • Research Site

    Szczecin, Poland

  • Research Site

    Warsaw, Poland

  • Research Site

    Wałbrzych, Poland