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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05019521




Registration number
NCT05019521
Ethics application status
Date submitted
19/08/2021
Date registered
25/08/2021
Date last updated
14/11/2024

Titles & IDs
Public title
A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
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)
Secondary ID [1] 0 0
2021-001198-22
Secondary ID [2] 0 0
ALXN2040-GA-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF
Timepoint [1] 0 0
Baseline, Week 104
Secondary outcome [2] 0 0
Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF
Timepoint [2] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [5] 0 0
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
Timepoint [5] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [7] 0 0
Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye
Timepoint [7] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [8] 0 0
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
Timepoint [8] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [9] 0 0
Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
Timepoint [9] 0 0
Baseline, Week 52 and Week 104
Secondary outcome [10] 0 0
Plasma Concentration Of Danicopan Over Time
Timepoint [10] 0 0
Up to 4 hours postdose
Secondary outcome [11] 0 0
PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time
Timepoint [11] 0 0
Up to 4 hours postdose
Secondary outcome [12] 0 0
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
Timepoint [12] 0 0
Day 1 through Week 104

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

Key
Minimum age
60 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/08/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
14/08/2025
Actual
Sample size
Target
Accrual to date
Final
365
Recruitment in Australia
Recruitment state(s)
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Research Site - Adelaide
Recruitment hospital [2] 0 0
Research Site - Albury
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Research Site - East Melbourne
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Research Site - Hurstville
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Research Site - Parramatta
Recruitment hospital [6] 0 0
Research Site - Strathfield
Recruitment hospital [7] 0 0
Research Site - Sydney
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5000 - Adelaide
Recruitment postcode(s) [2] 0 0
2640 - Albury
Recruitment postcode(s) [3] 0 0
3002 - East Melbourne
Recruitment postcode(s) [4] 0 0
2220 - Hurstville
Recruitment postcode(s) [5] 0 0
2150 - Parramatta
Recruitment postcode(s) [6] 0 0
2135 - Strathfield
Recruitment postcode(s) [7] 0 0
2000 - Sydney
Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT05019521
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05019521