Trial Outcomes & Findings for Evaluation of Oral Minocycline in the Treatment of Geographic Atrophy Associated With Age-Related Macular Degeneration (NCT NCT02564978)
NCT ID: NCT02564978
Last Updated: 2025-11-18
Results Overview
The difference in the square-root transformed GA area expansion rates in the study eye based on fundus autofluorescence (FAF) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) was compared using a linear spline regression model with a fixed knot at Month 9, at a Type I error rate of 2.5%.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
37 participants
Primary outcome timeframe
Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)
Results posted on
2025-11-18
Participant Flow
Up to forty-five (45) participants with unilateral or bilateral geographic atrophy (GA) associated with age-related macular degeneration (AMD) will be enrolled. Up to an additional 15 participants may be enrolled to replace participants who withdraw from the study prior to reaching the Month 33 visit. Participants with GA in at least one eye that is associated with AMD were enrolled. Enrollment closed at the National Eye Institute and Bristol Eye Hospital on December 31, 2019.
Participants must have at least one eye meeting all inclusion criteria and none of the exclusion criteria as defined in the protocol. For participants with two qualifying eyes, the fellow (non-study) eye will be noted as a "qualifying fellow eye." All eligible participants undergo treatment starting at Month 9 (e.g., are a part of the "Minocycline" group). The IP used in the study is oral minocycline and is not anticipated to differentially treat participant eyes.
Participant milestones
| Measure |
Minocycline
Oral administration of minocycline.
Minocycline: Participants underwent a nine-month run-in phase without taking minocycline. At Month 9, they were instructed to begin taking 100 mg of oral minocycline twice daily until study termination, after a minimum of 36 months on IP. Starting at Month 9 and continuing at Month 12, a three-month supply was dispensed to the participant during the study visit or mailed to the participant. Starting at Month 15 participants received two bottles for a six-month supply. Participants were required to bring their bottles of IP to each appropriate visit for capsule counts for compliance monitoring. Participants could complete participation in the study and discontinue treatment as early as Month 45 (36 months of treatment phase). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol.
|
|---|---|
|
Overall Study
STARTED
|
37
|
|
Overall Study
Completed Run-in Phase
|
36
|
|
Overall Study
Completed Month 33 Visit
|
21
|
|
Overall Study
Completed Month 45 Visit
|
10
|
|
Overall Study
Completed Month 57 Visit
|
4
|
|
Overall Study
COMPLETED
|
5
|
|
Overall Study
NOT COMPLETED
|
32
|
Reasons for withdrawal
| Measure |
Minocycline
Oral administration of minocycline.
Minocycline: Participants underwent a nine-month run-in phase without taking minocycline. At Month 9, they were instructed to begin taking 100 mg of oral minocycline twice daily until study termination, after a minimum of 36 months on IP. Starting at Month 9 and continuing at Month 12, a three-month supply was dispensed to the participant during the study visit or mailed to the participant. Starting at Month 15 participants received two bottles for a six-month supply. Participants were required to bring their bottles of IP to each appropriate visit for capsule counts for compliance monitoring. Participants could complete participation in the study and discontinue treatment as early as Month 45 (36 months of treatment phase). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol.
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Death
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Withdrawal by Subject
|
15
|
|
Overall Study
Early Study Closure
|
2
|
|
Overall Study
Other Reason
|
5
|
Baseline Characteristics
For one participant, GA area could not be graded at baseline due to poor image quality.
Baseline characteristics by cohort
| Measure |
Minocycline
n=37 Participants
Oral administration of minocycline.
Minocycline: Participants underwent a nine-month run-in phase without taking minocycline. At Month 9, they were instructed to begin taking 100 mg of oral minocycline twice daily until study termination, after a minimum of 36 months on IP. Starting at Month 9 and continuing at Month 12, a three-month supply was dispensed to the participant during the study visit or mailed to the participant. Starting at Month 15 participants received two bottles for a six-month supply. Participants were required to bring their bottles of IP to each appropriate visit for capsule counts for compliance monitoring. Participants could complete participation in the study and discontinue treatment as early as Month 45 (36 months of treatment phase). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=37 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=37 Participants
|
|
Age, Categorical
>=65 years
|
34 Participants
n=37 Participants
|
|
Age, Continuous
|
74.3 years
STANDARD_DEVIATION 7.6 • n=37 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=37 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=37 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=37 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=37 Participants
|
|
Race (NIH/OMB)
White
|
34 Participants
n=37 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=37 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=37 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=37 Participants
|
|
Region of Enrollment
Europe
|
7 participants
n=37 Participants
|
|
Square Root of Total Area of Definite Decreased Autofluorescence
|
2.5 mm
STANDARD_DEVIATION 0.9 • n=36 Participants • For one participant, GA area could not be graded at baseline due to poor image quality.
|
|
Square Root of Total Area of Macular GA
|
2.5 mm
STANDARD_DEVIATION 1.0 • n=31 Participants • Summary based on participants who received at least one dose of the study drug. Due to a software issue, some CFP images could not be graded for quantitative measures.
|
|
Best-Corrected Visual Acuity Total Letters Read
|
64.4 letters read
STANDARD_DEVIATION 14.3 • n=35 Participants • Summary based on participants who received at least one dose of the study drug.
|
|
Low Luminance Visual Acuity Total Letters Read
|
51.5 letters read
STANDARD_DEVIATION 16.0 • n=35 Participants • Summary based on participants who received at least one dose of the study drug.
|
|
Central Retinal Thickness
|
123.1 um
STANDARD_DEVIATION 67.5 • n=34 Participants • Summary based on participants who received at least one dose of the study drug.
|
PRIMARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the enrolled population (all participants enrolled in the study, regardless of compliance, follow-up or treatment received) with at least one data point for the study eye.
The difference in the square-root transformed GA area expansion rates in the study eye based on fundus autofluorescence (FAF) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) was compared using a linear spline regression model with a fixed knot at Month 9, at a Type I error rate of 2.5%.
Outcome measures
| Measure |
Minocycline
n=36 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in Square-Root Transformed GA Area Expansion Rates in the Study Eye Between the Treatment Phase and Run-in Phase Based on Fundus Autofluorescence (FAF)
|
-0.03 mm/year
Standard Error 0.03
|
SECONDARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the enrolled population (all participants enrolled in the study, regardless of compliance, follow-up or treatment received) with data for the respective eyes at Baseline, Month 9 and Month 33 for the respective analysis.
The difference in the square-root transformed of GA area expansion rates based on fundus autofluorescence (FAF) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) in the study eyes alone, the qualifying fellow eyes alone, and the study eyes and qualifying fellow eyes together, analyzed via a two-sided Student's paired t-test at a Type I error rate of 5%.
Outcome measures
| Measure |
Minocycline
n=33 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Fundus Autofluorescence (FAF)
Study eye
|
-0.01 mm/year
Interval -0.06 to 0.03
|
|
Difference in Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Fundus Autofluorescence (FAF)
Qualifying fellow eye
|
-0.1 mm/year
Interval -0.24 to 0.04
|
|
Difference in Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Fundus Autofluorescence (FAF)
Avg. of Study eye and Qualifying fellow eye
|
-0.04 mm/year
Interval -0.09 to 0.01
|
SECONDARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the Per-protocol population 2 (Participants who completed at least three follow-up visits during the 24-month treatment phase and did not discontinue treatment prior to the completion of at least three follow-up visits) with data for the respective eyes, for the respective analysis.
The difference in the square-root transformed of GA area expansion rates based on color fundus photography (CFP) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) in the study eyes alone, qualifying fellow eyes alone, and study eyes and qualifying fellow eyes together was compared using a linear spline regression model with a fixed knot at Month 9 at a Type I error rate of 2.5%.
Outcome measures
| Measure |
Minocycline
n=41 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in the Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Color Fundus Photography (CFP)
Study eye
|
0.03 mm/year
Standard Error 0.05
|
|
Difference in the Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Color Fundus Photography (CFP)
Qualifying fellow eye
|
-0.06 mm/year
Standard Error 0.08
|
|
Difference in the Square-root Transformed GA Area Expansion Rates Between the Treatment Phase and Run-in Phase Based on Color Fundus Photography (CFP)
Study eye + Qualifying fellow eye
|
-0.01 mm/year
Standard Error 0.04
|
SECONDARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the Per-protocol population 2 (Participants who completed at least three follow-up visits during the 24-month treatment phase and did not discontinue treatment prior to the completion of at least three follow-up visits) with data for the respective eyes at Baseline, Month 9 and Month 33 for the respective analysis.
The difference in the mean rate of change in Best Corrected Visual Acuity (BCVA) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) for the study eyes alone, the qualifying fellow eyes alone, and the study eyes and qualifying fellow eyes together, analyzed via a two-sided Student's paired t-test at a Type I error rate of 5%.
Outcome measures
| Measure |
Minocycline
n=27 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in the Mean Rate of Change in Best Corrected Visual Acuity (BCVA) Between the Treatment Phase and Run-in Phase
Study eye
|
0.2 letters read/month
Interval -0.4 to 0.9
|
|
Difference in the Mean Rate of Change in Best Corrected Visual Acuity (BCVA) Between the Treatment Phase and Run-in Phase
Qualifying fellow eye
|
-0.3 letters read/month
Interval -0.8 to 0.2
|
|
Difference in the Mean Rate of Change in Best Corrected Visual Acuity (BCVA) Between the Treatment Phase and Run-in Phase
Avg. of Study eye and Qualifying fellow eye
|
0.2 letters read/month
Interval -0.4 to 0.8
|
SECONDARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the Per-protocol population 2 (Participants who completed at least three follow-up visits during the 24-month treatment phase and did not discontinue treatment prior to the completion of at least three follow-up visits) with data for the respective eyes at Baseline, Month 9 and Month 33 for the respective analysis.
The difference in the mean rate of change in Low-Luminance Visual Acuity (LLVA) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) for the study eyes alone, the qualifying fellow eyes alone, and the study eyes and qualifying fellow eyes together, analyzed via a two-sided Student's paired t-test at a Type I error rate of 5%.
Outcome measures
| Measure |
Minocycline
n=26 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in the Mean Rate of Change in Low-Luminance Visual Acuity (LLVA) Between the Treatment Phase and Run-in Phase
Study eye
|
0.2 letters read/month
Interval -0.4 to 0.9
|
|
Difference in the Mean Rate of Change in Low-Luminance Visual Acuity (LLVA) Between the Treatment Phase and Run-in Phase
Qualifying fellow eye
|
-0.4 letters read/month
Interval -0.9 to 0.1
|
|
Difference in the Mean Rate of Change in Low-Luminance Visual Acuity (LLVA) Between the Treatment Phase and Run-in Phase
Avg. of Study eye and Qualifying fellow eye
|
0.2 letters read/month
Interval -0.4 to 0.7
|
SECONDARY outcome
Timeframe: Run-in phase (baseline to Month 9) before initiation of IP and 24 months treatment phase after initiation of IP (Month 9 to Month 33)Population: Participants in the Per-protocol population 2 (Participants who completed at least three follow-up visits during the 24-month treatment phase and did not discontinue treatment prior to the completion of at least three follow-up visits) with data for the respective eyes at Baseline, Month 9 and Month 33 for the respective analysis. Participants may have data from the qualifying fellow eye only.
The difference in the mean rate of change of central retinal thickness as measured on Optical Coherence Tomography (OCT) between the treatment phase (Month 9 to Month 33) and the run-in phase (baseline to Month 9) for the study eyes alone, the qualifying fellow eyes alone, and the study eyes and qualifying fellow eyes together, analyzed via a two-sided Student's paired t-test at a Type I error rate of 5%.
Outcome measures
| Measure |
Minocycline
n=26 Eyes
Participants, ages 55 years and older, with unilateral or bilateral Geographic Atrophy (GA) associated with atrophic Age-Related Macular Degeneration (AMD) underwent a nine-month run-in period prior to receiving IP. Starting at Month 9 (IP start visit), participants received 100 mg minocycline orally twice a day for a total of at least 36 months (i.e., through Month 45).
|
|---|---|
|
Difference in the Mean Rate of Change of Central Retinal Thickness as Measured on Optical Coherence Tomography (OCT) Between the Treatment Phase and Run-in Phase
Study eye
|
0.7 μm/month
Interval -0.4 to 1.8
|
|
Difference in the Mean Rate of Change of Central Retinal Thickness as Measured on Optical Coherence Tomography (OCT) Between the Treatment Phase and Run-in Phase
Qualifying fellow eye
|
1.8 μm/month
Interval 0.4 to 3.1
|
|
Difference in the Mean Rate of Change of Central Retinal Thickness as Measured on Optical Coherence Tomography (OCT) Between the Treatment Phase and Run-in Phase
Avg. of Study eye and Qualifying fellow eye
|
1.0 μm/month
Interval 0.0 to 2.0
|
Adverse Events
Minocycline
Serious events: 8 serious events
Other events: 32 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Minocycline
n=37 participants at risk
Oral administration of minocycline.
Minocycline: Participants underwent a nine-month run-in phase without taking minocycline. At Month 9, they were instructed to begin taking 100 mg of oral minocycline twice daily until study termination, after a minimum of 36 months on IP. Starting at Month 9 and continuing at Month 12, a three-month supply was dispensed to the participant during the study visit or mailed to the participant. Starting at Month 15 participants received two bottles for a six-month supply. Participants were required to bring their bottles of IP to each appropriate visit for capsule counts for compliance monitoring. Participants could complete participation in the study and discontinue treatment as early as Month 45 (36 months of treatment phase). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol.
|
|---|---|
|
Cardiac disorders
Tachycardia
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Cardiac disorders
Atrial fibrillation
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Cardiac disorders
Myocardial infarction
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Papilloedema
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukaemia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Renal cell carcinoma
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Obstructive Pancreatitis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Pneumonia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Injury, poisoning and procedural complications
Fall
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Surgical and medical procedures
Knee arthroplasty
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
Other adverse events
| Measure |
Minocycline
n=37 participants at risk
Oral administration of minocycline.
Minocycline: Participants underwent a nine-month run-in phase without taking minocycline. At Month 9, they were instructed to begin taking 100 mg of oral minocycline twice daily until study termination, after a minimum of 36 months on IP. Starting at Month 9 and continuing at Month 12, a three-month supply was dispensed to the participant during the study visit or mailed to the participant. Starting at Month 15 participants received two bottles for a six-month supply. Participants were required to bring their bottles of IP to each appropriate visit for capsule counts for compliance monitoring. Participants could complete participation in the study and discontinue treatment as early as Month 45 (36 months of treatment phase). At the conclusion of the study, participants were no longer eligible to receive IP under this protocol.
|
|---|---|
|
Investigations
Blood thyroid stimulating hormone increased
|
40.5%
15/37 • Number of events 18 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Blood thyroid stimulating hormone decreased
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Blood cholesterol increased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Blood urea increased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Haemoglobin decreased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Hepatic enzyme increased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Lymphocyte count decreased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
SARS-CoV 2 test positive
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
Weight decreased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Investigations
White blood cell count decreased
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Diarrhoea
|
10.8%
4/37 • Number of events 4 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Constipation
|
8.1%
3/37 • Number of events 3 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Abdominal pain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Dry mouth
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Dyspepsia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Hiatus hernia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Nausea
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Oesophagitis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Portal hypertensive gastropathy
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Gastrointestinal disorders
Umbilical hernia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
16.2%
6/37 • Number of events 7 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Skin discolouration
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Conjunctival haemorrhage
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Photosensitivity reaction
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Rash
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Sebaceous glands overactivity
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Skin and subcutaneous tissue disorders
Telangiectasia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Conjunctivitis viral
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Diverticulitis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Gastroenteritis viral
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Gastrointestinal viral infection
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Infected dermal cyst
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Lower respiratory tract infection
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Oral candidiasis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Sinusitis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Subcutaneous abscess
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Upper respiratory tract infection
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Infections and infestations
Urinary tract infection
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Dry eye
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Meibomian gland dysfunction
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Eye pruritus
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Keratopathy
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Punctate keratitis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Retinal vein occlusion
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Eye disorders
Vitreous detachment
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Metabolism and nutrition disorders
Abnormal loss of weight
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Metabolism and nutrition disorders
Abnormal weight gain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Metabolism and nutrition disorders
Gout
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Injury, poisoning and procedural complications
Joint injury
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Nervous system disorders
Dizziness
|
2.7%
1/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Nervous system disorders
Dementia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Nervous system disorders
Paraesthesia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Cardiac disorders
Palpitations
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Cardiac disorders
Atrial fibrillation
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papilloma
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Seborrhoeic keratosis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Blood and lymphatic system disorders
Anaemia
|
5.4%
2/37 • Number of events 2 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Endocrine disorders
Autoimmune thyroiditis
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Endocrine disorders
Hypothyroidism
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
General disorders
Pain
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
2.7%
1/37 • Number of events 1 • Participants enrolled at the United States site are assessed for AEs starting at Month 9, after IP initiation, for up to 48 months. Participants enrolled at the European site are assessed for AEs starting at the baseline visit for up to 57 months.
|
Additional Information
Tiarnan Keenan, MD, PhD, Principal Investigator, NEI
National Institutes of Health
Phone: 301-451-6330
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place