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New pill combo aims to keep PNH patients stable for years

NCT ID NCT05389449

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and helps maintain healthy red blood cell levels over the long term.

What this could mean

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

Active substance
danicopan (oral tablet)
What this could lead to
If successful, this could show that adding danicopan to existing PNH therapy helps maintain better blood health over the long term.
What could go wrong
This is an extension study with no placebo group, so results are limited. It may not prove danicopan works better than standard care alone, and side effects are still being tracked.

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

80 people

The number who actually took part.

Started

Oct 2022

Expected to finish

Aug 2026

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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.

Key Inclusion Criteria: * All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment. * Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol. * Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors. Key Exclusion Criteria: * Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant. * Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study. * Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal Hemolysis 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

  • Research Site

    Los Angeles, California, 90089, United States

  • Research Site

    Chicago, Illinois, 60612, United States

  • Research Site

    Baltimore, Maryland, 21231, United States

  • Research Site

    Kansas City, Missouri, 64111, United States

  • Research Site

    Long Island City, New York, 11101, United States

  • Research Site

    Cleveland, Ohio, 44195, United States

  • Research Site

    Dallas, Texas, 75390-9047, United States

  • Research Site

    Porto Alegre, 90110-270, Brazil

  • Research Site

    Rio de Janeiro, 20211-080, Brazil

  • Research Site

    Toronto, Ontario, M5G 2C4, Canada

  • Research Site

    Brno, 625 00, Czechia

  • Research Site

    Lille, 59037, France

  • Research Site

    Paris, 75010, France

  • Research Site

    Pessac, 33604, France

  • Research Site

    Pierre-Bénite, 69495, France

  • Research Site

    Athens, 11527, Greece

  • Research Site

    Thessaloniki, 57010, Greece

  • Research Site

    Haifa, 31048, Israel

  • Research Site

    Jerusalem, 91120, Israel

  • Research Site

    Avellino, 83100, Italy

  • Research Site

    Bassano del Grappa, 36061, Italy

  • Research Site

    Florence, 50134, Italy

  • Research Site

    Milan, 20122, Italy

  • Research Site

    Reggio Calabria, 89131, Italy

  • Research Site

    Roma, 161, Italy

  • Research Site

    Bunkyō City, 113 8603, Japan

  • Research Site

    Kyoto, 605-0981, Japan

  • Research Site

    Osaka, 530-8480, Japan

  • Research Site

    Shibuya-ku, 150-8935, Japan

  • Research Site

    Tsukuba, 305-8576, Japan

  • Research Site

    Kota Kinabalu, 88586, Malaysia

  • Research Site

    Kuching, 93200, Malaysia

  • Research Site

    Miri, 98000, Malaysia

  • Research Site

    Gdansk, 80-952, Poland

  • Research Site

    Seoul, 03722, South Korea

  • Research Site

    Seoul, 06591, South Korea

  • Research Site

    Suwon, 16247, South Korea

  • Research Site

    Badalona, 8916, Spain

  • Research Site

    Barcelona, 08036, Spain

  • Research Site

    Majadahonda, 28222, Spain

  • Research Site

    Seville, 41013, Spain

  • Research Site

    Bangkok, 10330, Thailand

  • Research Site

    Airdrie, ML6 0JS, United Kingdom

  • Research Site

    Leeds, LS9 7TF, United Kingdom

  • Research Site

    London, SE5 9RS, United Kingdom

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