Clinical trial NCT05019521: A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
Official title: A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Geographic Atrophy
Keywords: Geographic Atrophy
GA
Danicopan
ALXN2040
Complement Inhibition
AMD
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-08-25
Enrollment: 365
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up to 6 weeks, a 104-week masked Treatment Period, followed by a 30-day Follow-up after the last dose. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo.


Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline To Week 52 In The Square Root (sqrt) Of Total GA Lesion Area In The Study Eye As Measured By Fundus Autofluorescence (FAF)
timeFrame: Baseline, Week 52
Secondary outcomes:
measure: Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF
timeFrame: Baseline, Week 104
measure: Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF
timeFrame: Baseline, Week 52 and Week 104
measure: Change from Baseline to Week 52 and Week 104 in macular ellipsoid zone (EZ) and outer retinal integrity in the study eye, the fellow eye, and both eyes combined as measured by SD-OCT
timeFrame: Baseline, Week 52 and Week 104
measure: Change from Baseline to Week 52 and Week 104 in subretinal pigment epithelium (sub-RPE) compartment/drusen/RPE complex in the study eye, fellow eye and both eyes combined as measured by SD OCT
timeFrame: Baseline, Week 52 and Week 104
measure: Change From Baseline To Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart
timeFrame: Baseline, Week 52 and Week 104
measure: Change From Baseline To Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye As Assessed By The ETDRS Chart
timeFrame: Baseline, Week 52 and Week 104
measure: Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye
timeFrame: Baseline, Week 52 and Week 104
measure: Change From Baseline To Week 52 And Week 104 In Monocular Reading Speeds In The Study Eye As Assessed By Minnesota Low Vision Reading Test (MNRead) Acuity Charts Or Radner Reading Charts
timeFrame: Baseline, Week 52 and Week 104
measure: Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
timeFrame: Baseline, Week 52 and Week 104
measure: Plasma Concentration Of Danicopan Over Time
timeFrame: Up to 4 hours postdose
measure: PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time
timeFrame: Up to 4 hours postdose
measure: Incidence Of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), And Ocular TEAEs, SAEs, And Clinical Laboratory Abnormalities, And Events Leading To Discontinuation Of Study Drug Throughout The Study
timeFrame: Day 1 through Week 104
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Beverly Hills
country: United States
facility: Research Site
state: California
city: Encino
country: United States
facility: Research Site
state: California
city: Fresno
country: United States
facility: Research Site
state: California
city: Fullerton
country: United States
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state: California
city: La Jolla
country: United States
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city: Pasadena
country: United States
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state: California
city: Sacramento
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country: United States
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country: United States
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city: Colorado Springs
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city: Longmont
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city: Waterford
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state: Connecticut
city: Clearwater
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city: Coral Springs
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city: Deerfield Beach
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city: Naples
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city: Palm Beach Gardens
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city: Saint Petersburg
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city: Augusta
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state: Georgia
city: New Albany
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state: Indiana
city: West Des Moines
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state: Iowa
city: Hagerstown
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state: Maryland
city: Rosedale
country: United States
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city: Towson
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city: Springfield
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state: Massachusetts
city: Southaven
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state: Mississippi
city: Reno
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state: Nevada
city: Teaneck
country: United States
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state: New Jersey
city: Great Neck
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city: Durham
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city: Cincinnati
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city: Portland
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city: Springfield
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city: Monroeville
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city: Philadelphia
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city: Philadelphia
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city: Greenville
country: United States
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state: South Carolina
city: Ladson
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city: Rapid City
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state: South Dakota
city: Austin
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city: Bellaire
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city: Lynchburg
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country: Australia
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country: Australia
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country: Australia
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country: Australia
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city: Sydney
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city: Pardubice
country: Czechia
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city: Praha 5
country: Czechia
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city: Creteil
country: France
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city: La Tronche
country: France
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city: Lyon
country: France
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country: France
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country: France
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country: France
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country: France
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country: France
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country: France
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country: France
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country: France
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country: France
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city: Berlin
country: Germany
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country: Germany
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country: Germany
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country: Germany
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country: Germany
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city: Mainz A. Rhein
country: Germany
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city: Marburg
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city: Muenchen
country: Germany
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city: Münster
country: Germany
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city: Münster
country: Germany
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city: Neubrandenburg
country: Germany
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city: Regensburg
country: Germany
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city: Tübingen
country: Germany
facility: Research Site
city: Ulm
country: Germany
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city: Würzburg
country: Germany
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Pécs
country: Hungary
facility: Research Site
city: Firenze
country: Italy
facility: Research Site
city: Genova
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Naples
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Fukushima-shi
country: Japan
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city: Kita-gun
country: Japan
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city: Osaka-shi
country: Japan
facility: Research Site
city: Busan
country: Korea, Republic of
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country: Korea, Republic of
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country: Korea, Republic of
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city: Jinju-si
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
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city: Seoul
country: Korea, Republic of
facility: Research Site
city: Riga
country: Latvia
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country: Latvia
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city: Bratislava
country: Slovakia
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country: Slovakia
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country: Spain
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country: Spain
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city: Barcelona
country: Spain
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country: Spain
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city: Cordoba
country: Spain
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city: Majadahonda
country: Spain
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city: Pamplona
country: Spain
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city: Sevilla
country: Spain
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city: Valencia
country: Spain
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city: Valladolid
country: Spain
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city: Zaragoza
country: Spain
facility: Research Site
city: Bristol
country: United Kingdom
facility: Research Site
city: Liverpool
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
city: Newcastle Upon Tyne
country: United Kingdom
facility: Research Site
city: Southampton
country: United Kingdom
facility: Research Site
Study officials:
References: