Trial · NCT05019521
A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
NCT05019521, Phase 2, Terminated, Alexion Pharmaceuticals, Inc.; results first posted 2025-09-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration. Trials, record NCT05019521. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05019521
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration
- NCT number
- NCT05019521
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 365 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Geographic Atrophy
- Interventions
- Danicopan, Placebo
- First posted
- 2021-08-25
- Last updated
- 2025-09-05
- Other registries' IDs
- eudract:2021-001198-22
- Results first posted
- 2025-09-05
- Limitations and caveats
- The study was terminated early because the primary efficacy endpoint was not met.
Source ClinicalTrials.gov
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Baseline, Week 52 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (square millimeter \[mm\^2\]) was transformed into sqrt (millimeter \[mm\]). Greater area affected means a worse outcome than smaller area affected. The rate of change from Baseline (mm/year) for each treatment group was estimated based on the coefficients for time and the interaction of time and treatment from the mixed models for repeated measures (MMRM). MMRM analysis included the change from Baseline at post-baseline visit in the sqrt GA lesion area as dependent variable. |
| 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 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area measurement. The total GA lesion area (mm\^2) was transformed into sqrt (mm). Greater area affected means a worse outcome than smaller area affected. |
| 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 | FAF provides high-contrast retinal images particularly valuable for the detection of atrophic areas. FAF images of the study eye were taken and sent to the reading center for total GA lesion area (mm\^2) measurement. Greater area affected means a worse outcome than smaller area affected. |
| Secondary | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Baseline, Week 52 and Week 104 | Photoreceptor loss was assessed by measuring the percent of macular EZ total attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome. |
| Secondary | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | Photoreceptor degradation was assessed by measuring the percent of macular EZ partial attenuation using SD-OCT. The EZ was considered to be formed mainly by mitochondria within the ellipsoid layer of the outer portion of the inner segments of the photoreceptors. In SD-OCT, decreases in the integrity and intensity of the EZ have been correlated with the reduction in cone photoreceptors and retinal sensitivity in retinal degenerative diseases. Lower increase in percentage means a better outcome. Higher increase in percentage means a worse outcome. |
| Secondary | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The ONL - RPE mean central 1 mm subfield thickness refers to the average thickness of the retinal layers between the ONL and RPE measured within a 1 mm radius from the foveal center (the central point of the retina). |
| Secondary | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The ONL - RPE mean central macular 2 mm subfield thickness refers to the thickness of the retina in the central part of the macula, specifically within a 2 mm radius. |
| Secondary | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE macular volume refers to the volume of the retinal tissue between the EZ and the RPE, specifically within the macular region. Changes in EZ-RPE volume indicate the presence or progression of diseases affecting the outer retina. |
| Secondary | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE mean central 1 mm subfield thickness is a measurement of the thickness of the ellipsoid zone, a layer within the retina, in the central 1 mm region. Changes in EZ-RPE thickness indicate damage or dysfunction within the photoreceptors, which is associated with various eye diseases like AMD. |
| Secondary | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | The EZ - RPE mean central macular 2 mm thickness measurement reflects the distance between the outer border of the EZ and the inner border of the RPE layer, typically within a central 2 mm area. Decreased EZ-RPE thickness is an early indicator of disease, such as in dry AMD, where a thinning or loss of the EZ is a hallmark. Changes in EZ-RPE thickness is used to monitor treatment response in conditions like AMD. |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 registered outcomesOutcomes29
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Baseline, Week 52 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) who had any post-baseline FAF GA lesion area data at the Week 52 analysis. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. | mm/year | LEAST_SQUARES_MEAN |
| 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 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline visit and post-baseline (Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for the participants. Therefore, the 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. | mm | MEAN |
| 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 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit FAF GA lesion area data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable at specified timepoints. | mm^2 | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in Percent Macular Ellipsoid Zone (EZ) Total Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by Spectral-domain Optical Coherence Tomography (SD-OCT) | Baseline, Week 52 and Week 104 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | percentage of EZ total attenuation | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in Percent Macular EZ Partial Attenuation in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit SD-OCT data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | percentage of EZ partial attenuation | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in Outer Nuclear Layer (ONL) - Retinal Pigment Epithelium (RPE) Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Hence, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | micrometers (µm) | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in ONL-RPE Mean Central Macular 2 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit ONL-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | µm | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in Ellipsoid Zone (EZ) - (RPE) Macular Volume in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in the FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment group) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE macular volume data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below = number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | cubic millimeters (mm^3) | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central 1 mm Subfield Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central 1 mm subfield thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | µm | MEAN |
| SECONDARY | Change From Baseline to Week 52 and Week 104 in EZ-RPE Mean Central Macular 2 mm Thickness in the Study Eye, the Fellow Eye, and Both Eyes Combined as Measured by SD-OCT | Baseline, Week 52 and Week 104 | All participants in FAS (all randomized participants who received at least 1 dose \[full or partial\] of study drug grouped by randomized treatment) with both baseline and post-baseline (Week 52 and Week 104) visit EZ-RPE mean central macular 2 mm thickness data. As prespecified, data were collected and reported for participants. Therefore, 'Ns' reported below= number of participants analyzed regardless of which eye was evaluated. Number analyzed = participants evaluable for specified categories. | µm | MEAN |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 outcomesResults by group805
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Danicopan 100 mg BID | 0.465 | Standard Error 0.0289 | mm/year | |
| Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Danicopan 200 mg BID | 0.391 | Standard Error 0.0306 | mm/year | |
| Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Danicopan 400 mg QD | 0.443 | Standard Error 0.0301 | mm/year | |
| Rate of Change From Baseline to Week 52 in the Square Root (Sqrt) of Total Geographic Atrophy (GA) Lesion Area in the Study Eye as Measured by Fundus Autofluorescence (FAF) | Placebo | 0.406 | Standard Error 0.0304 | mm/year | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Danicopan 100 mg BID | 0.892 | Standard Deviation 0.4545 | mm | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Danicopan 200 mg BID | 0.705 | Standard Deviation 0.3831 | mm | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Danicopan 400 mg QD | 0.859 | Standard Deviation 0.3468 | mm | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Placebo - Danicopan 100 mg BID | 0.668 | Standard Deviation 0.4866 | mm | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Placebo - Danicopan 200 mg BID | 0.585 | Standard Deviation 0.1396 | mm | |
| Change From Baseline to Week 104 in the Sqrt of the Total GA Lesion Area in the Study Eye as Measured by FAF | Placebo - Danicopan 400 mg QD | 0.787 | Standard Deviation 0.1858 | mm |
1 to 10 of 805Source ClinicalTrials.gov
See all 805 results by groupParticipant flow56
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Primary Evaluation Period (52 Weeks) | STARTED | Danicopan 100 mg BID | 93 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Danicopan 200 mg BID | 91 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Danicopan 400 mg QD | 91 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Placebo | 90 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Placebo - Danicopan 100 mg BID | 0 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Placebo - Danicopan 200 mg BID | 0 | |
| Primary Evaluation Period (52 Weeks) | STARTED | Placebo - Danicopan 400 mg QD | 0 | |
| Primary Evaluation Period (52 Weeks) | Received at Least 1 Dose of Study Drug | Danicopan 100 mg BID | 93 | |
| Primary Evaluation Period (52 Weeks) | Received at Least 1 Dose of Study Drug | Danicopan 200 mg BID | 91 | |
| Primary Evaluation Period (52 Weeks) | Received at Least 1 Dose of Study Drug | Danicopan 400 mg QD | 91 |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 participant flowAdverse events1,250
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neovascular age-related macular degeneration | Eye disorders | Primary Evaluation Period: Danicopan 100 mg BID | 5 | 93 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Primary Evaluation Period: Danicopan 200 mg BID | 5 | 91 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Primary Evaluation Period: Danicopan 400 mg QD | 5 | 91 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Primary Evaluation Period: Placebo | 3 | 90 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Danicopan 100 mg BID | 3 | 73 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Danicopan 200 mg BID | 2 | 67 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Danicopan 400 mg QD | 1 | 73 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Placebo - Danicopan 100 mg BID | 1 | 24 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Placebo - Danicopan 200 mg BID | 1 | 23 | |
| other | Neovascular age-related macular degeneration | Eye disorders | Secondary Evaluation Period: Placebo - Danicopan 400 mg QD | 1 | 23 |
1 to 10 of 1,250Source ClinicalTrials.gov
See all 1,250 adverse eventsAdverse event totals30
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Primary Evaluation Period: Danicopan 100 mg BID | 0 | 93 |
| Total, all-cause mortality | Primary Evaluation Period: Danicopan 200 mg BID | 1 | 91 |
| Total, all-cause mortality | Primary Evaluation Period: Danicopan 400 mg QD | 0 | 91 |
| Total, all-cause mortality | Primary Evaluation Period: Placebo | 3 | 90 |
| Total, all-cause mortality | Secondary Evaluation Period: Danicopan 100 mg BID | 1 | 73 |
| Total, all-cause mortality | Secondary Evaluation Period: Danicopan 200 mg BID | 1 | 67 |
| Total, all-cause mortality | Secondary Evaluation Period: Danicopan 400 mg QD | 1 | 73 |
| Total, all-cause mortality | Secondary Evaluation Period: Placebo - Danicopan 100 mg BID | 1 | 24 |
| Total, all-cause mortality | Secondary Evaluation Period: Placebo - Danicopan 200 mg BID | 0 | 23 |
| Total, all-cause mortality | Secondary Evaluation Period: Placebo - Danicopan 400 mg QD | 0 | 23 |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37314061 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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GET https://conductscience.org/api/trials/v1/NCT05019521
GET https://conductscience.org/api/trials/v1/NCT05019521/registered?page=2