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New hope for PNH patients: early access to danicopan combo therapy

NCT ID NCT05982938

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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, and have failed or cannot use other approved treatments. The goal is to control the disease by reducing red blood cell destruction outside blood vessels.

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.

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 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.

Key Inclusion Criteria * Male or female, aged 18 years and older * Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count ≥ 120 × 109/L. * Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment. * Platelet count ≥ 30,000/µL without the need for platelet transfusions. * Absolute neutrophil counts ≥ 500/µL. * Vaccinated against N meningitidis. Key Exclusion Criteria * Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan). * Laboratory abnormalities at screening, as follows: * Alanine aminotransferase (ALT) \> 2 × upper limit of normal (ULN) * Direct bilirubin \> 2 × ULN, with the exception of: * participants with increased bilirubin due to EVH in the opinion of the Treating Physician or * participants with documented Gilbert's syndrome * Active bacterial or viral infection, a body temperature \> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.

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Conditions

The condition(s) this trial relates to.

anemia Hemoglobinuria, Paroxysmal Hemolysis hemolytic anemia paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Extravascular hemolysis Paroxysmal nocturnal hemoglobinuria PNH

How to take part

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

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

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

  3. 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 ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Research Site

    AVAILABLE

    Bologna, 40138, Italy

  • Research Site

    AVAILABLE

    Cagliari, 09121, Italy

  • Research Site

    AVAILABLE

    Catania, 95123, Italy

  • Research Site

    AVAILABLE

    Civitanova Marche, 62012, Italy

  • Research Site

    AVAILABLE

    Cremona, 26100, Italy

  • Research Site

    AVAILABLE

    Cuneo, 12100, Italy

  • Research Site

    AVAILABLE

    Frosinone, 03100, Italy

  • Research Site

    AVAILABLE

    Novara, 28100, Italy

  • Research Site

    AVAILABLE

    Palermo, 90146, Italy

  • Research Site

    AVAILABLE

    Pescara, 65100, Italy

  • Research Site

    AVAILABLE

    Ravenna, 48121, Italy

  • Research Site

    AVAILABLE

    Reggio Calabria, 89100, Italy

  • Research Site

    AVAILABLE

    Roma, 00161, Italy

  • Research Site

    AVAILABLE

    Roma, 00168, Italy

  • Research Site

    AVAILABLE

    Torino, 10126, Italy

More trials for these conditions

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