Danicopan for Age-Related Macular Degeneration

Recruiting at 269 trial locations

Overseen By Alexion Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexion Pharmaceuticals, Inc.

Must not be taking: Anti-VEGF, Stem cell therapy

Disqualifiers: Complement deficiency, Active ocular diseases, others

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 104 weeks

365 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial investigates the effectiveness of a new treatment, danicopan, for individuals with geographic atrophy (GA) due to age-related macular degeneration (AMD), a condition that can lead to vision loss. Participants will receive one of three doses of the medication or a placebo to determine the optimal dosage and assess its safety. This trial is suitable for individuals with GA linked to AMD in at least one eye who have not used other recent treatments for AMD. As a Phase 2 trial, it measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to important research.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that danicopan is being tested for safety in treating geographic atrophy (GA), a condition related to age-related macular degeneration (AMD). Studies have found that danicopan can reach the eye tissues needing treatment, indicating potential effectiveness. However, the focus here is on its safety.

Researchers have studied danicopan to assess its safety. It generally appears well-tolerated by patients, but, like many treatments, it carries some risks. Some research suggests that because danicopan affects the immune system, there might be a higher chance of infections or other immune-related side effects. More detailed long-term safety information is still being collected.

In summary, while early findings about danicopan's safety are promising, some risks exist. Anyone considering joining a trial should discuss these factors with a healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for age-related macular degeneration, which often involve injections into the eye to slow disease progression, danicopan offers a unique approach by targeting the complement pathway, which is a part of the immune system involved in inflammation and tissue damage. This oral medication could potentially streamline treatment by offering a non-invasive option that might reduce the frequency or need for eye injections. Researchers are excited about danicopan because it not only provides a different mechanism of action but may also improve patient compliance and comfort with its oral administration.

What evidence suggests that this trial's treatments could be effective for age-related macular degeneration?

Research has shown that danicopan can enter the eye and attach to a pigment called melanin. This attachment results in higher drug levels in the eye, which might help slow the progression of geographic atrophy (GA) in age-related macular degeneration (AMD). Although direct clinical proof for danicopan's effectiveness in AMD is not yet available, its mechanism suggests potential benefits in slowing the disease. Danicopan is already approved for other conditions, demonstrating its safety and some effectiveness in those cases. Participants in this trial will receive varying doses of danicopan or a placebo to evaluate its effectiveness for AMD. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

This trial is for individuals with a condition called Geographic Atrophy (GA) that's due to Age-Related Macular Degeneration (AMD). Participants must have received a specific vaccination, be able to consent, and have GA lesions outside the central part of their retina. People are excluded if they've had other treatments or studies for GA in the last 3 months, have complement deficiencies, certain medical conditions deemed risky by the investigator, previous stem cell/gene therapy for eye conditions, active ocular diseases affecting vision assessment or allergies to study drug components.

Inclusion Criteria

I have been vaccinated against meningitis.

The entire GA lesion must be greater than 1 μm outside of the foveal center

I have vision loss in one eye due to AMD.

Exclusion Criteria

I have received stem cell or gene therapy for an eye condition.

I am not allergic to the trial drug or its ingredients.

History or presence of any medical or psychological condition that would make the patient inappropriate for the study

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 6 weeks

Treatment

Participants receive danicopan or placebo during the 104-week masked Treatment Period. Participants may be switched to the optimal dose after 52 weeks.

104 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Danicopan

Trial Overview The study tests different doses of Danicopan (100 mg twice daily, 200 mg twice daily, and 400 mg once daily) against a placebo to see which is more effective and safe over two years. It aims to find out how well Danicopan works on GA secondary to AMD and monitors participants' reactions over time after taking it.

How Is the Trial Designed?

Group I: Danicopan: 400 mg Experimental Treatment 1 Intervention

Participants will receive danicopan 400 mg qd during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group II: Danicopan: 200 mg Experimental Treatment 1 Intervention

Participants will receive danicopan 200 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group III: Danicopan: 100 mg Experimental Treatment 1 Intervention

Participants will receive danicopan 100 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group IV: Placebo Placebo Group 2 Interventions

Participants will receive matching placebo and will be re-randomized to one of the active treatment groups at Week 52, or to the optimal dose, if already identified.

Who Is Running the Clinical Trial?

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials

267

Recruited

141,000+

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Alexion Pharmaceuticals

Lead Sponsor

Trials

231

Recruited

36,700+

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9624268/

Danicopan, an Oral Complement Factor D Inhibitor, Exhibits ... Danicopan crosses the blood–retina barrier and binds melanin reversibly, leading to a higher and more sustained exposure in melanin-containing ocular tissues.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05019521

NCT05019521 | A Study of Danicopan in Participants With ... This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD.

3. synapse.patsnap.com synapse.patsnap.com/article/how-does-danicopancompare-with-other-treatments-for-amd

How does Danicopancompare with other treatments for ... However, at present, there is no direct published clinical evidence or mature trial data available in AMD populations for danicopan, and its use ...

4. mayo.edu mayo.edu/research/clinical-trials/cls-20533353

A Study of Danicopan in Participants With Geographic ... The purposes of this study are to find out whether danicopan can slow the rate of geographic atrophy (GA) disease progression and slow the loss of visual ...

5. journals.lww.com journals.lww.com/ijsgh/fulltext/2025/03000/danicopan_s_fda_approval__a_breakthrough_in_the.5.aspx

Danicopan's FDA approval: a breakthrough in the treatment... The findings of clinical studies, especially phase 3 ALPHA trials, suggest that Danicopan is safe and effective in reducing hemolytic indicators ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2024/218037Orig1s000OtherR.pdf

center for drug evaluation and research - accessdata.fda.gov The purpose of this analysis is to characterize the safety of danicopan in treatment naïve patients and to study the long-term risks of serious ...