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

New PNH pill matches standard infusion therapy in trial

NCT ID NCT05630001

Disease control 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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested an oral medication called iptacopan in 52 adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were already stable on standard infusion treatments. Participants switched from their usual therapy to iptacopan pills taken twice daily. The main goal was to see if their hemoglobin levels stayed stable, and results showed iptacopan was non-inferior to standard care.

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.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

52 people

The number who actually took part.

Started

Apr 2023

Finished

Oct 2024

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.

Ages

18 to 100 years

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: * Signed informed consent must be obtained prior to participation in the study. * Male and female participants ≥ 18 years of age, at the time of ICF signatures and with a diagnosis of PNH confirmed by treating physician. * Stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months prior to screening * Mean hemoglobin level ≥10 g/dL * Vaccination against Neisseria meningitidis and S. pneumoniae infection are required prior to the start of iptacopan treatment. * If not received previously, vaccination against Haemophilus influenzae infections is recommended, if available and according to local regulations. * Ability to communicate well with the investigator, to understand and comply with the requirements of the study * Other protocol -defined inclusion criteria may apply at the end. Exclusion Criteria: * Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment * Patients requiring red blood cell transfusion in the 6 months prior to screening or during screening * History of stem cell transplantation or any solid organ transplantation * Active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to study drug administration * Presence of fever ≥ 38.0 °C (100.4 °F) within 7 days prior to study drug administration * Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody at Screening) * A history of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus * Unstable medical condition including, but not limited to, myocardial ischemia, active gastrointestinal bleeding, coexisting chronic anemia unrelated to PNH, or unstable thrombotic event not amenable to active treatment as judged by the investigator at Screening. * History of cancer of any part of the body within the past 5 years, * Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial. * Any medical condition deemed likely to interfere with the patient's participation in the study * Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study

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

  • City Of Hope National Med Center

    Duarte, California, 91010, United States

  • Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • Huntsman Cancer Institute Univ of Utah

    Salt Lake City, Utah, 84112 0550, United States

  • Lakes Research

    Miami Lakes, Florida, 33014, United States

  • Mass Gen Hosp Cancer Center

    Boston, Massachusetts, 02114, United States

  • Montefiore Medical Center

    The Bronx, New York, 10461, United States

  • Novartis Investigative Site

    Nantes, 44093, France

  • Novartis Investigative Site

    Nice, 06202, France

  • Novartis Investigative Site

    Paris, 75475, France

  • Novartis Investigative Site

    Dresden, Saxony, 01307, Germany

  • Novartis Investigative Site

    Aachen, 52074, Germany

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Ulm, 89081, Germany

  • Novartis Investigative Site

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    Bassano del Grappa, VI, 36061, Italy

  • Novartis Investigative Site

    Seoul, Seoul, 06351, South Korea

  • Novartis Investigative Site

    Barcelona, 08036, Spain

  • Novartis Investigative Site

    Istanbul, Fatih, 34093, Turkey (Türkiye)

  • Novartis Investigative Site

    Leeds, West Yorkshire, LS9 7TF, United Kingdom

  • Novartis Investigative Site

    London, SE5 9RS, United Kingdom

  • Prisma Health Upstate

    Greenville, South Carolina, 29615, United States

  • USC Norris Cancer Center

    Los Angeles, California, 90033, United States

  • University Of Minnesota

    Minneapolis, Minnesota, 55455, United States

More trials for these conditions

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