Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Real-World check: newer PNH drug holds up in routine care

NCT ID NCT05274633

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 120 adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers measured changes in a blood marker called LDH to see if the new drug controlled red blood cell breakdown just as well or better. The goal was to understand how ravulizumab performs outside of strict clinical trials, in real-life medical settings.

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

120 people

The number who actually took part.

Started

Jun 2022

Finished

Mar 2025

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

Participants with documented diagnosis of PNH that were already treated with eculizumab and started treatment with ravulizumab and met the inclusion and exclusion criteria.

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: * Body weight of 10 kilogram or above * Hemolysis with clinical symptom(s) indicative of high disease activity * Documented diagnoses of PNH confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells with granulocyte or monocyte clone size of ≥ 5% * Clinically stable after having been treated with eculizumab for at least the past 6 months * Participant already assigned to ravulizumab treatment as a specific therapeutic strategy within the current routine clinical practice (this decision has to be made independently and before the enrolment of the participant in the study) * Vaccinated against Neisseria meningitidis (according to Summary of Product Characteristics) \< 3 years before dosing or at least 2 weeks prior to initiating ravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection * Signed written informed and privacy consent prior to study participation Exclusion Criteria: * History of hematopoietic stem cell transplantation (evaluated at baseline) * Known pregnant or breastfeeding participant (evaluated at baseline) * Participant unable to read and write in Italian language and to autonomously fill in questionnaires and scales (evaluated at enrolment) * Participants enrolled in any clinical study receiving experimental treatments for PNH (evaluated at baseline) * Hypersensitivity to the active substance or to any of the excipient of the study drug. * Participants with unresolved N. meningitidis infection at treatment initiation * Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Paroxysmal nocturnal hemoglobinuria are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

Contacts and locations

Locations

  • Clinical Trial Site

    Brescia, Italy

  • Clinical Trial Site

    Catania, Italy

  • Clinical Trial Site

    Lecce, Italy

  • Clinical Trial Site

    Ragusa, Italy

  • Clinical Trial Site

    Roma, Italy

  • Clinical Trial Site

    Salerno, Italy

More trials for these conditions

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