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Eye drug trial halted: could danicopan still slow blind spots?

NCT ID NCT05019521

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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 26, 2026

Summary

This Phase 2 study tested an oral drug called danicopan in 365 people with geographic atrophy, an advanced form of dry age-related macular degeneration that causes blind spots. Participants took different doses of danicopan or a placebo for up to 104 weeks to see if it could slow the growth of damaged areas in the eye. The trial was terminated early, so the full results are not available, but the data may still offer clues for future treatments.

What this could mean

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

Active substance
Danicopan (ALXN2040) - an oral tablet
What this could lead to
If successful, danicopan could slow the growth of blind spots in the eye, potentially preserving vision longer for people with advanced dry age-related macular degeneration.
What could go wrong
This trial was terminated early, so results are limited. It is a Phase 2 study, meaning it is still early and may not show clear benefit. The drug also requires vaccination against meningitis due to infection risk.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

365 people

The number who actually took part.

Started

Aug 2021

Finished

Jan 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.

Ages

60 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: * Vaccination for Neisseria meningitidis. * Capable of giving signed informed consent. * Presentation of GA secondary to AMD in at least 1 eye * The entire GA lesion must be \> 1 μm outside of the foveal center Key Exclusion Criteria: * GA in the study eye due to cause other than AMD. * Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye for intraocular vascular disease. * Have previously received any stem cell/gene therapy for any ophthalmological condition in either eye. * Use of any investigational medicinal product (ie, participation in interventional clinical studies for any ophthalmic indications) or use of any regulatory approved treatment for GA in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the last dose of the investigational or commercial product (whichever is longer). * Presence of active ocular diseases in the study eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments. * Known or suspected complement deficiency. * History or presence of any medical or psychological condition that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study. * Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients. Note: Additional inclusion/exclusion criteria may apply, per protocol.

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Conditions

The condition(s) this trial relates to.

age-related macular degeneration dry age related macular degeneration Geographic Atrophy

As listed by the trial registrant

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

Geographic atrophy

Contacts and locations

Locations

  • Research Site

    Phoenix, Arizona, 85020, United States

  • Research Site

    Beverly Hills, California, 90211, United States

  • Research Site

    Encino, California, 91436, United States

  • Research Site

    Fresno, California, 93720, United States

  • Research Site

    Fullerton, California, 92835, United States

  • Research Site

    La Jolla, California, 92093, United States

  • Research Site

    Pasadena, California, 91105, United States

  • Research Site

    Sacramento, California, 95841, United States

  • Research Site

    San Mateo, California, 94401, United States

  • Research Site

    Walnut Creek, California, 94598, United States

  • Research Site

    Colorado Springs, Colorado, 80909, United States

  • Research Site

    Longmont, Colorado, 80503, United States

  • Research Site

    Waterford, Connecticut, 06385, United States

  • Research Site

    Clearwater, Florida, 33761, United States

  • Research Site

    Coral Springs, Florida, 33067, United States

  • Research Site

    Deerfield Beach, Florida, 33064, United States

  • Research Site

    Naples, Florida, 34103, United States

  • Research Site

    Palm Beach Gardens, Florida, 33418, United States

  • Research Site

    St. Petersburg, Florida, 33711, United States

  • Research Site

    Augusta, Georgia, 30909, United States

  • Research Site

    New Albany, Indiana, 47150, United States

  • Research Site

    West Des Moines, Iowa, 50266, United States

  • Research Site

    Hagerstown, Maryland, 21740, United States

  • Research Site

    Rosedale, Maryland, 21237, United States

  • Research Site

    Towson, Maryland, 21204, United States

  • Research Site

    Springfield, Massachusetts, 01107, United States

  • Research Site

    Southaven, Mississippi, 38671, United States

  • Research Site

    Reno, Nevada, 89502, United States

  • Research Site

    Teaneck, New Jersey, 07666, United States

  • Research Site

    Great Neck, New York, 11021, United States

  • Research Site

    Durham, North Carolina, 27705, United States

  • Research Site

    Cincinnati, Ohio, 45219, United States

  • Research Site

    Portland, Oregon, 97225, United States

  • Research Site

    Springfield, Oregon, 97477, United States

  • Research Site

    Kingston, Pennsylvania, 18704, United States

  • Research Site

    Monroeville, Pennsylvania, 15146, United States

  • Research Site

    Philadelphia, Pennsylvania, 19104-2640, United States

  • Research Site

    Philadelphia, Pennsylvania, 19107, United States

  • Research Site

    Greenville, South Carolina, 29605, United States

  • Research Site

    Ladson, South Carolina, 29456, United States

  • Research Site

    Rapid City, South Dakota, 57701, United States

  • Research Site

    Austin, Texas, 78750, United States

  • Research Site

    Bellaire, Texas, 77401, United States

  • Research Site

    Conroe, Texas, 77384, United States

  • Research Site

    Dallas, Texas, 75231, United States

  • Research Site

    San Antonio, Texas, 78240, United States

  • Research Site

    Southlake, Texas, 76092, United States

  • Research Site

    Fairfax, Virginia, 22031, United States

  • Research Site

    Lynchburg, Virginia, 24502, United States

  • Research Site

    Adelaide, 5000, Australia

  • Research Site

    Albury, 2640, Australia

  • Research Site

    East Melbourne, 3002, Australia

  • Research Site

    Hurstville, 2220, Australia

  • Research Site

    Parramatta, 2150, Australia

  • Research Site

    Strathfield, 2135, Australia

  • Research Site

    Sydney, 2000, Australia

  • Research Site

    Pardubice, 530 02, Czechia

  • Research Site

    Prague, 150 00, Czechia

  • Research Site

    Créteil, 94010, France

  • Research Site

    Écully, 69130, France

  • Research Site

    Lyon, 69004, France

  • Research Site

    Marseille, 13008, France

  • Research Site

    Nantes, 44000, France

  • Research Site

    Nice, 06000, France

  • Research Site

    Paris, 75010, France

  • Research Site

    Paris, 75019, France

  • Research Site

    Paris, 75571, France

  • Research Site

    Poitiers, 86021, France

  • Research Site

    Strasbourg, 67091, France

  • Research Site

    Berlin, 12203, Germany

  • Research Site

    Bonn, 53127, Germany

  • Research Site

    Düsseldorf, 40225, Germany

  • Research Site

    Freiburg im Breisgau, 79106, Germany

  • Research Site

    Göttingen, 37075, Germany

  • Research Site

    Hanover, 30625, Germany

  • Research Site

    Heidelberg, 69120, Germany

  • Research Site

    Mainz A. Rhein, 55131, Germany

  • Research Site

    Marburg, 35043, Germany

  • Research Site

    München, 81675, Germany

  • Research Site

    Münster, 48145, Germany

  • Research Site

    Münster, 48149, Germany

  • Research Site

    Neubrandenburg, 17036, Germany

  • Research Site

    Regensburg, 93053, Germany

  • Research Site

    Tübingen, 72076, Germany

  • Research Site

    Ulm, 89081, Germany

  • Research Site

    Würzburg, 97080, Germany

  • Research Site

    Budapest, 1133, Hungary

  • Research Site

    Pécs, 7621, Hungary

  • Research Site

    Florence, 50134, Italy

  • Research Site

    Genova, 16132, Italy

  • Research Site

    Milan, 20122, Italy

  • Research Site

    Milan, 20132, Italy

  • Research Site

    Milan, 20157, Italy

  • Research Site

    Naples, 80131, Italy

  • Research Site

    Roma, 00133, Italy

  • Research Site

    Roma, 00168, Italy

  • Research Site

    Fukushima, 960-1295, Japan

  • Research Site

    Kita-gun, 761-0793, Japan

  • Research Site

    Osaka, 5458586, Japan

  • Research Site

    Riga, LV-1009, Latvia

  • Research Site

    Riga, LV1002, Latvia

  • Research Site

    Bratislava, 811 08, Slovakia

  • Research Site

    Žilina, 012 07, Slovakia

  • Research Site

    Busan, 49241, South Korea

  • Research Site

    Daegu, 42415, South Korea

  • Research Site

    Incheon, 22332, South Korea

  • Research Site

    Jinju, 52727, South Korea

  • Research Site

    Seoul, 03082, South Korea

  • Research Site

    Seoul, 06351, South Korea

  • Research Site

    Barcelona, 08022, Spain

  • Research Site

    Barcelona, 08035, Spain

  • Research Site

    Barcelona, 8025, Spain

  • Research Site

    Bilbao, 48006, Spain

  • Research Site

    Córdoba, 14012, Spain

  • Research Site

    Majadahonda, 28222, Spain

  • Research Site

    Pamplona, 31005, Spain

  • Research Site

    Seville, 41071, Spain

  • Research Site

    Valencia, 46014, Spain

  • Research Site

    Valladolid, 46012, Spain

  • Research Site

    Zaragoza, 50009, Spain

  • Research Site

    Bristol, BS1 2LX, United Kingdom

  • Research Site

    Liverpool, L69 3BX, United Kingdom

  • Research Site

    London, EC1V 2PD, United Kingdom

  • Research Site

    London, GU16 7UJ, United Kingdom

  • Research Site

    London, NW1 5QH, United Kingdom

  • Research Site

    London, SE5 9RS, United Kingdom

  • Research Site

    Newcastle upon Tyne, NE7 7DN, United Kingdom

  • Research Site

    Southampton, S016 6YD, United Kingdom

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