Proof of concept and dose-finding study of danicopan in patients with geographic atrophy secondary to AMD

JRCT ID: jRCT2051220074

Registered date:10/08/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
• Date of first enrollment: 13/06/2022
• Target sample size: 13
• Countries of recruitment: Australia,Japan,Canada,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Hungary,Japan,Italy,Japan,Republic of Korea,Japan,Latvia,Japan,Slovakia,Japan,Spain,Japan,UK,Japan,US,Japan
• Study type: Interventional

Intervention(s)

Drug: Danicopan/Placebo 100mg,200mg or 400mg oral tablet
Other Name: ALXN2040

Outcome(s)

• Primary Outcome: 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)

Secondary Outcome

1.Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF
2.Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF
3.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
4.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
5.Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye
6.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
7.Plasma Concentration Of Danicopan Over Time [ Time Frame: Up to 4 hours postdose ]
8.PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time
9.Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
10.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

Key inclusion & exclusion criteria

• Age minimum: >= 70age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Documentation of vaccination for Neisseria meningitidis.
2.Capable of giving signed informed consent.
3.Presentation of GA secondary to AMD in at least 1 eye with new inclusion criterion
4.The entire GA lesion must be extrafoveal without foveal involvement

Exclude criteria

1.GA in either eye due to cause other than AMD.
2.Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye.
3.Have previously received any complement/stem cell/gene therapy for any ophthalmological condition.
4.Previous participation in interventional clinical studies for treatment of drusen, nascent GA or GA (except vitamins or minerals) irrespective of route of administration (ocular or systemic) in either eye.
5.Presence of active ocular diseases in either eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments.
6.Known or suspected complement deficiency.
7.History or presence of any clinically relevant co-morbidities or any uncontrolled conditions.
8.Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.

Related Information

• Primary Sponsor: Kanemoto Koji
• Secondary ID(s): NCT05019521