Danicopan for Age-Related Macular Degeneration

Recruiting at 250 trial locations

Overseen By Alexion Pharmaceuticals, Inc.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alexion Pharmaceuticals, Inc.

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

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called danicopan to see if it can help people with a specific type of eye disease called Geographic Atrophy, which is related to Age-related Macular Degeneration. The study will check how well the medication works, how safe it is, and how the body handles it.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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