Phase 2 Macular Degeneration Trial, Terminated NCT05019521 Sponsor: Alexion Pharmaceuticals, Inc. Condition: Macular Degeneration
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial (NCT05019521) studied a medicine called danicopan in people with geographic atrophy — a form of age-related macular degeneration where patches of the retina gradually break down, causing vision loss. Participants were randomly assigned to receive one of three doses of danicopan (100 mg twice daily, 200 mg twice daily, or 400 mg once daily) or a placebo (a dummy pill with no active medicine). In the first 52-week period, 93, 91, 91, and 90 people started in each of those groups respectively. After that, participants who had been on placebo were switched to one of the danicopan doses for a further 52 weeks. The trial's main goal was to measure how quickly the damaged area on the retina grew over the first year. The reported data shows that over the first 52 weeks, the rate at which the damaged retinal area grew was 0.465 mm/year in the 100 mg twice-daily group, 0.391 mm/year in the 200 mg twice-daily group, 0.443 mm/year in the 400 mg once-daily group, and 0.406 mm/year in the placebo group. (These numbers represent a mathematical transformation of the lesion size used to make the measurements more reliable — a smaller number means a slower rate of growth, which is considered a better result.) Several secondary measures were also reported, including the total size of the damaged area, the degree of damage to light-sensing cells in the retina, and the thickness of retinal layers, tracked at both 52 and 104 weeks. The reported data shows these measurements generally increased (indicating more damage) across all groups over time, with some variation between the danicopan doses and the placebo group; however, the figures for some sub-groups at Week 104 were not reported in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 2 Macular Degeneration Trial, Terminated

NCT05019521
Terminated Phase 2 🇦🇺 Australian site

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You must have received a vaccination against a bacterial infection called Neisseria meningitidis before joining.
  • You must be able to understand and sign a consent form agreeing to take part.
  • You must have a condition called geographic atrophy (a type of damage to the back of the eye) caused by age-related macular degeneration (AMD) in at least one eye.
  • The area of eye damage must be located a certain distance away from the very centre of your eye (confirm with trial site for exact measurement details).

Who may not be able to join:

  • Your eye damage is caused by something other than age-related macular degeneration.
  • You have previously had injections into your eye to treat a blood vessel condition in the eye being studied.
  • You have ever had stem cell or gene therapy for any eye condition in either eye.
  • You have taken part in another clinical trial for an eye condition, or used an approved treatment for your eye condition, within the last 3 months (or longer, depending on the treatment).
  • You have another active eye disease in the eye being studied that could affect your vision or interfere with the study results, in the opinion of the doctor running the trial.
  • You have a known or suspected problem with a part of your immune system called the complement system.
  • You have any medical or mental health condition that the lead doctor feels would make it unsuitable for you to take part.
  • You have a known allergy or sensitivity to a dye used in eye examinations (fluorescein), or to the study drug (danicopan) or any of its ingredients.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 13 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Australian sites

Research Site, Adelaide,
Research Site, Albury,
Research Site, East Melbourne,
Research Site, Hurstville,
Research Site, Parramatta,
Research Site, Strathfield,
Research Site, Sydney,

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

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Trial details

Status
Terminated
Phase
Phase 2
Sponsor
Alexion Pharmaceuticals, Inc.
Registry
ClinicalTrials.gov
Start date
23 August 2021
Est. completion
5 July 2024

Where this trial is recruiting

🇦🇺 Australia 🇨🇿 Czechia 🇫🇷 France 🇩🇪 Germany 🇭🇺 Hungary 🇮🇹 Italy 🇯🇵 Japan 🇱🇻 Latvia 🇸🇰 Slovakia 🇰🇷 South Korea 🇪🇸 Spain 🇬🇧 United Kingdom 🇺🇸 United States

7 site(s) in Australia. Confirm current status and contact details directly with the trial site.

Primary endpoints

Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF)

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 13 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov