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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05019521
Registration number
NCT05019521
Ethics application status
Date submitted
19/08/2021
Date registered
25/08/2021
Date last updated
5/06/2024
Titles & IDs
Public title
A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
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Scientific 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)
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Secondary ID [1]
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ALXN2040-GA-201
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy
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Condition category
Condition code
Eye
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Diseases / disorders of the eye
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Danicopan
Treatment: Drugs - Placebo
Experimental: Danicopan: 100 mg - 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.
Experimental: Danicopan: 200 mg - 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.
Experimental: Danicopan: 400 mg - 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.
Placebo Comparator: Placebo - 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.
Treatment: Drugs: Danicopan
Oral tablet.
Treatment: Drugs: Placebo
Oral tablet.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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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)
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Assessment method [1]
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Timepoint [1]
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Baseline, Week 52
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Secondary outcome [1]
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Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF
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Assessment method [1]
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Timepoint [1]
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Baseline, Week 104
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Secondary outcome [2]
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Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF
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Assessment method [2]
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Timepoint [2]
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Baseline, Week 52 and Week 104
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Secondary outcome [3]
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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
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Assessment method [3]
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Timepoint [3]
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Baseline, Week 52 and Week 104
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Secondary outcome [4]
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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
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Assessment method [4]
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Timepoint [4]
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Baseline, Week 52 and Week 104
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Secondary outcome [5]
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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
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Assessment method [5]
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Timepoint [5]
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Baseline, Week 52 and Week 104
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Secondary outcome [6]
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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
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Assessment method [6]
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Timepoint [6]
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Baseline, Week 52 and Week 104
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Secondary outcome [7]
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Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye
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Assessment method [7]
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Timepoint [7]
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Baseline, Week 52 and Week 104
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Secondary outcome [8]
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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
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Assessment method [8]
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Timepoint [8]
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Baseline, Week 52 and Week 104
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Secondary outcome [9]
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Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
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Assessment method [9]
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Timepoint [9]
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Baseline, Week 52 and Week 104
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Secondary outcome [10]
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Plasma Concentration Of Danicopan Over Time
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Assessment method [10]
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Timepoint [10]
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Up to 4 hours postdose
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Secondary outcome [11]
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PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time
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Assessment method [11]
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Timepoint [11]
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Up to 4 hours postdose
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Secondary outcome [12]
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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
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Assessment method [12]
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Timepoint [12]
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Day 1 through Week 104
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Eligibility
Key inclusion criteria
Key
- 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
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Minimum age
60
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/08/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
14/08/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
365
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Research Site - Adelaide
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5000 - Adelaide
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2640 - Albury
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3002 - East Melbourne
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2220 - Hurstville
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2150 - Parramatta
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2135 - Strathfield
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Recruitment postcode(s) [7]
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2000 - Sydney
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Recruitment outside Australia
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Alexion Pharmaceuticals, Inc.
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Ethics approval
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05019521
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05019521
Download to PDF