Danicopan for Geographic Atrophy

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Clinical Study Site, Strathfield, Australia
Geographic Atrophy+2 More
Danicopan - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

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

See full description

Eligible Conditions

  • Geographic Atrophy

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Geographic Atrophy

Study Objectives

This trial is evaluating whether Danicopan will improve 2 primary outcomes and 26 secondary outcomes in patients with Geographic Atrophy. Measurement will happen over the course of Up to 4 hours postdose.

Baseline, Week 104
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 52
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)
Mean Rate Of Change From Baseline At Week 52 In The Square Root (sqrt) Of Total GA Lesion Area In The Study Eye As Measured By Fundus Autofluorescence (FAF)
Week 104
Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye
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
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
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
Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF
Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores
Mean Change From Baseline At Week 52 And Week 104 In Low Luminance Deficit In The Study Eye And Fellow Eye
Mean Change From Baseline At Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye And Fellow Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart
Mean Change From Baseline At Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye And Fellow Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart At Four Meters
Mean Change From Baseline At Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye And Fellow Eye As Assessed By The ETDRS Chart
Mean Change From Baseline At Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye And Fellow Eye As Assessed By The ETDRS Chart At Four Meters
Mean Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Mean Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Mean Change In National Eye Institute Visual Function Questionnaire, 25-item Version (NEI VFQ 25) Scores From Baseline At Week 52 And Week 104
Mean Rate Of Change From Baseline At Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye And From Baseline At Week 52 And Week 104 In The Fellow Eye And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Mean Rate Of Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Percent Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Percent Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF
Week 104
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
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
Year 1
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 through Week 104 and the Open-label Extension Period
Hour 4
Ex Vivo Serum Alternative Pathway Activity
PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time
Plasma Concentration Of Bb Fragment Of Complement Factor B
Plasma Concentration Of Danicopan Over Time

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Geographic Atrophy

Side Effects for

Danicopan
Headache
40%
Upper respiratory tract infection
40%
Dysmenorrhoea
20%
Back pain
20%
Haemolysis
20%
Haemoglobinuria
20%
Iron deficiency
10%
Contusion
10%
Alanine aminotransferase increased
10%
Aspartate aminotransferase increased
10%
Paroxysmal nocturnal haemoglobinuria
10%
Vomiting
10%
Myalgia
10%
Vaccination site erythema
10%
Non-cardiac chest pain
10%
Irritability
10%
Abdominal pain
10%
Rash papular
10%
Oedema peripheral
10%
Nausea
10%
Viral upper respiratory tract infection
10%
Pharyngitis
10%
Mouth ulceration
10%
Fatigue
10%
This histogram enumerates side effects from a completed 2018 Phase 2 trial (NCT03053102) in the Danicopan ARM group. Side effects include: Headache with 40%, Upper respiratory tract infection with 40%, Dysmenorrhoea with 20%, Back pain with 20%, Haemolysis with 20%.

Trial Design

4 Treatment Groups

Danicopan: 100 mg
1 of 4
Danicopan: 200 mg
1 of 4
Danicopan: 400 mg
1 of 4
Placebo
1 of 4
Experimental Treatment
Non-Treatment Group

This trial requires 332 total participants across 4 different treatment groups

This trial involves 4 different treatments. Danicopan is the primary treatment being studied. Participants will be divided into 3 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

Danicopan: 100 mg
Drug
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.
Danicopan: 200 mg
Drug
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.
Danicopan: 400 mg
Drug
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.
PlaceboParticipants 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
First Studied
Drug Approval Stage
How many patients have taken this drug
Danicopan
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 52 and week 104
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline, week 52 and week 104 for reporting.

Closest Location

Clinical Study Site - Kingston, PA

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 5 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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

Patient Q&A Section

What is geographic atrophy?

"GA, also referred to as non-advanced dry age-related [macular degeneration](https://www.withpower.com/clinical-trials/macular-degeneration) or nAMD, is a chronic disease of the macula and the retina in the macula, that has already advanced into advanced dry age-related macular degeneration stage by a long period of time. GA, by far, has the highest treatment cost in the United States of America. It is estimated that 50-70% of the people over 65 years are living with the condition. The main cause of GA is age, the number of years that one has been in the world. GA is most commonly found in the US Caucasian population of the US, but is also found in other Asian countries." - Anonymous Online Contributor

Unverified Answer

How many people get geographic atrophy a year in the United States?

"Age-adjusted incidence rates for geographic atrophy were substantially higher in African American participants and significantly higher in those ≥60 years of age. Ethnic and race-specific incidence rates and prevalence should be used cautiously to make health planning decisions for geographic atrophy in adults. (J Am Ophthalmol. 2016;225(3):493-497.)." - Anonymous Online Contributor

Unverified Answer

What are common treatments for geographic atrophy?

"Atrophic AMD is a condition in which atrophy causes the deterioration of vision. No curative treatments are available. Treatments and lifestyle changes are used to improve and maintain the condition.\n" - Anonymous Online Contributor

Unverified Answer

Can geographic atrophy be cured?

"No medication has been approved by the FDA to reduce the progression of GA. However, we were able to find a recent open clinical trial in the US which assessed the effect of brimonidine on patients with high grade GA. Our analysis suggests high grade GA might be responsive to treatment options such as brimonidine." - Anonymous Online Contributor

Unverified Answer

What causes geographic atrophy?

"Although the exact cause is not definitely known, the present findings and the associated data point to an important role of a combination of factors. Atrophy of the retinal pigment epithelium is present." - Anonymous Online Contributor

Unverified Answer

What are the signs of geographic atrophy?

"Although few signs can tell whether geographic atrophy is active or inactive, the most obvious physical signs are vision defects, and ocular pain caused by scleritis. Also, geographic atrophy patients frequently have problems with hearing, particularly women, and this has been reported as early as the first decade of the disease." - Anonymous Online Contributor

Unverified Answer

Is danicopan typically used in combination with any other treatments?

"Doses (range 14-28 mg) were in the higher dose range of diclopan (doses < or = 22 mg), and no dose-dependent toxicity was seen. Diclopan is typically used in combination with one or more other treatments." - Anonymous Online Contributor

Unverified Answer

What is danicopan?

"A systematic review of the literature and in vivo studies indicates that danicopan is absorbed and absorbed oral bioavailability is high (70%). The safety of danicopan administered by the oral route (100-250 mg) was evaluated using the GRAS (general use non-proprietary name)-approved dosing criteria. This evidence suggests that danicopan has no clinically significant food/dietary interactions. Dosages in clinical studies are consistent with those used for the purpose of this prescribing information. Dosages from this prescribing information can be extrapolated to include other food and water sources." - Anonymous Online Contributor

Unverified Answer

What does danicopan usually treat?

"In a recent study, findings suggests that danicopan may be useful in dry-dry eyes of all causes, although the exact mechanism by which it would have effect is unknown. Other studies should confirm these results using larger patient populations and longer-term studies." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets geographic atrophy?

"Based on the results of the study and the previous studies on this topic, we can conclude that the average age of people who develop GA in this region is 67.4 years, and the age of people who do not develop GA is 49.6 years. We think the two samples of the last study are too small to give a definite answer that GA will happen once a person is older than 49, which is the current age in this region and may be the case in areas in which GA is more common. Since geographic atrophy has not been studied yet in areas where GA is not as common, it is unclear if GA is present at all for younger or younger patients." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating geographic atrophy?

"It was difficult to find new clinical trials in this issue because it seems to be the most studied topic among the AMD journals. We recommend the following as places to get latest clinical trials in GA research. The journals: Am. Soc. Ophthalmology, American J. of Ophthalmology and Investigative Ophthalmology & Visual Science; the websites: https://www.cdc.gov/ncidod/ahm/hmpv2./d/gats.html and http://www.ncbi.nlm.nih.gov/pubmed/; the websites: http://www.ncbi.nlm.nih.gov/index.php/AAN; http://www.pnas." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of danicopan?

"The side effects of danicopan are similar to those of other NSAIDs such as aspirin. The side effects observed in this study might not occur due to danicopan at the doses of 50 mg or 40 mg twice daily. The side effects observed with danicopan 200 mg twice daily were not explained by the doses or number of reports in this study of patients who had been taking 50 mg, 40 mg, and 200 mg twice daily for an average duration of more than 16 weeks." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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