A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Geographic Atrophy Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05019521
• Other study ID #: ALXN2040-GA-201
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: August 23, 2021
• Est. completion date: August 14, 2025

Study information

• Verified date: June 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 365
• Est. completion date: August 14, 2025
• Est. primary completion date: July 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 60 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Geographic Atrophy
• Macular Degeneration

Intervention

Drug:
• Danicopan
Oral tablet.
• Placebo
Oral tablet.

Locations

Country	Name	City	State
Australia	Research Site	Adelaide
Australia	Research Site	Albury
Australia	Research Site	East Melbourne
Australia	Research Site	Hurstville
Australia	Research Site	Parramatta
Australia	Research Site	Strathfield
Australia	Research Site	Sydney
Canada	Research Site	Ottawa	Ontario
Czechia	Research Site	Pardubice
Czechia	Research Site	Praha 5
France	Research Site	Creteil
France	Research Site	Écully
France	Research Site	La Tronche
France	Research Site	Lyon Cedex 04
France	Research Site	Marseille
France	Research Site	Nantes
France	Research Site	Nice
France	Research Site	Paris
France	Research Site	Paris
France	Research Site	Paris
France	Research Site	Poitiers
France	Research Site	Strasbourg Cedex
Germany	Research Site	Berlin
Germany	Research Site	Bonn
Germany	Research Site	Düsseldorf
Germany	Research Site	Freiburg
Germany	Research Site	Göttingen
Germany	Research Site	Hannover
Germany	Research Site	Heidelberg
Germany	Research Site	Mainz A. Rhein
Germany	Research Site	Marburg
Germany	Research Site	Muenchen
Germany	Research Site	Münster
Germany	Research Site	Münster
Germany	Research Site	Neubrandenburg
Germany	Research Site	Regensburg
Germany	Research Site	Tübingen
Germany	Research Site	Ulm
Germany	Research Site	Würzburg
Hungary	Research Site	Budapest
Hungary	Research Site	Pécs
Italy	Research Site	Firenze
Italy	Research Site	Genova
Italy	Research Site	Milano
Italy	Research Site	Milano
Italy	Research Site	Milano
Italy	Research Site	Naples
Italy	Research Site	Roma
Italy	Research Site	Roma
Japan	Research Site	Fukushima-shi
Japan	Research Site	Kita-gun
Japan	Research Site	Osaka-shi
Korea, Republic of	Research Site	Busan
Korea, Republic of	Research Site	Daegu
Korea, Republic of	Research Site	Incheon
Korea, Republic of	Research Site	Jinju-si
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Latvia	Research Site	Riga
Latvia	Research Site	Riga
Slovakia	Research Site	Bratislava
Slovakia	Research Site	Zilina
Spain	Research Site	Barcelona
Spain	Research Site	Barcelona
Spain	Research Site	Barcelona
Spain	Research Site	Bilbao
Spain	Research Site	Cordoba
Spain	Research Site	Majadahonda
Spain	Research Site	Pamplona
Spain	Research Site	Sevilla
Spain	Research Site	Valencia
Spain	Research Site	Valladolid
Spain	Research Site	Zaragoza
United Kingdom	Research Site	Bristol
United Kingdom	Research Site	Liverpool
United Kingdom	Research Site	London
United Kingdom	Research Site	London
United Kingdom	Research Site	London
United Kingdom	Research Site	London
United Kingdom	Research Site	Newcastle Upon Tyne
United Kingdom	Research Site	Southampton
United States	Research Site	Augusta	Georgia
United States	Research Site	Austin	Texas
United States	Research Site	Austin	Texas
United States	Research Site	Bellaire	Texas
United States	Research Site	Beverly Hills	California
United States	Research Site	Cincinnati	Ohio
United States	Research Site	Clearwater	Florida
United States	Research Site	Cleveland	Ohio
United States	Research Site	Colorado Springs	Colorado
United States	Research Site	Conroe	Texas
United States	Research Site	Coral Springs	Florida
United States	Research Site	Dallas	Texas
United States	Research Site	Deerfield Beach	Florida
United States	Research Site	Durham	North Carolina
United States	Research Site	Edmond	Oklahoma
United States	Research Site	Encino	California
United States	Research Site	Eugene	Oregon
United States	Research Site	Fairfax	Virginia
United States	Research Site	Fresno	California
United States	Research Site	Fullerton	California
United States	Research Site	Great Neck	New York
United States	Research Site	Greenville	South Carolina
United States	Research Site	Hagerstown	Maryland
United States	Research Site	Jacksonville	Florida
United States	Research Site	Kingston	Pennsylvania
United States	Research Site	La Jolla	California
United States	Research Site	Ladson	South Carolina
United States	Research Site	Longmont	Colorado
United States	Research Site	Los Angeles	California
United States	Research Site	Lynchburg	Virginia
United States	Research Site	Miami	Florida
United States	Research Site	Miami	Florida
United States	Research Site	Monroeville	Pennsylvania
United States	Research Site	Naples	Florida
United States	Research Site	New Albany	Indiana
United States	Research Site	Palm Beach Gardens	Florida
United States	Research Site	Pasadena	California
United States	Research Site	Philadelphia	Pennsylvania
United States	Research Site	Philadelphia	Pennsylvania
United States	Research Site	Phoenix	Arizona
United States	Research Site	Portland	Oregon
United States	Research Site	Rapid City	South Dakota
United States	Research Site	Reno	Nevada
United States	Research Site	Rosedale	Maryland
United States	Research Site	Sacramento	California
United States	Research Site	Saint Petersburg	Florida
United States	Research Site	San Antonio	Texas
United States	Research Site	San Mateo	California
United States	Research Site	Southaven	Mississippi
United States	Research Site	Southlake	Texas
United States	Research Site	Springfield	Oregon
United States	Research Site	Springfield	Massachusetts
United States	Research Site	Teaneck	New Jersey
United States	Research Site	Towson	Maryland
United States	Research Site	Walnut Creek	California
United States	Research Site	Waterford	Connecticut
United States	Research Site	West Des Moines	Iowa

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Czechia,  France,  Germany,  Hungary,  Italy,  Japan,  Korea, Republic of,  Latvia,  Slovakia,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)		Baseline, Week 52
Secondary	Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF		Baseline, Week 104
Secondary	Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF		Baseline, Week 52 and Week 104
Secondary	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		Baseline, Week 52 and Week 104
Secondary	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		Baseline, Week 52 and Week 104
Secondary	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		Baseline, Week 52 and Week 104
Secondary	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		Baseline, Week 52 and Week 104
Secondary	Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye		Baseline, Week 52 and Week 104
Secondary	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		Baseline, Week 52 and Week 104
Secondary	Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores		Baseline, Week 52 and Week 104
Secondary	Plasma Concentration Of Danicopan Over Time		Up to 4 hours postdose
Secondary	PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time		Up to 4 hours postdose
Secondary	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		Day 1 through Week 104