Trial Outcomes & Findings for OT-551 Antioxidant Eye Drops to Treat Geographic Atrophy in Age-Related Macular Degeneration (NCT NCT00306488)

Last Updated: 2020-07-14

Results Overview

Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. This acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters the Snellen measurement is 20/20.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

2 years

Results posted on

2020-07-14

Participant Flow

Participant milestones

Participant milestones
Measure	OT-511 Antioxidant Eye Drop The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day. The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

OT-551 Antioxidant Eye Drops to Treat Geographic Atrophy in Age-Related Macular Degeneration

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Participant Information n=11 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	10 Participants n=5 Participants
Age, Continuous	76.91 years STANDARD_DEVIATION 7.85 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Data collected from the 10 participants that completed the 24-month follow-up visit were analyzed. Ten study eyes and 10 fellow eyes were analyzed.

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=10 Participants The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=10 Eyes The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
The Change in Best-corrected Visual Acuity (BCVA) From Baseline to Year 2 for All Participants.	0.2 ETDRS Letters Standard Deviation 13.3	-11.3 ETDRS Letters Standard Deviation 7.6

SECONDARY outcome

Timeframe: 2 years

Population: Data collected from the ten participants that completed the full twenty-four months of the study were analyzed. Ten study eyes and ten fellow eyes were analyzed.

Geographic Atrophy (GA), or the death of photoreceptors and surrounding cells in the retina, is a common condition in patients with Age-Related Macular Degeneration (AMD). The death of these photoreceptors results in lesions that cause vision loss. The amount of GA is measured from images produced via a non-invasive technique called Fundus Autofluorescence Imaging, which uses a Confocal Scanning Ophthalmoscope to detect the naturally-fluorescing lipofuscin (the waste that is left behind by dead photoreceptors and digested by surrounding cells) that is prevalent at the border of the lesion.

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=10 Eyes The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=10 Eyes The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
The Change in Geographic Atrophy (GA), as Measured on Fundus Autofluorescence Imaging Using a Confocal Scanning Ophthalmoscope (HRA FAF) From Baseline to Year 2.	2.17 mm^2 Standard Deviation 0.83	2.24 mm^2 Standard Deviation 0.91

SECONDARY outcome

Timeframe: 2 years

Population: Data collected from the ten participants that completed the full twenty-four months of the study were analyzed. Ten study eyes and ten fellow eyes were analyzed.

GA was also measured using Stereoscopic Color Fundus Photography (CFP), which produces color images of the inside of the eye.

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=10 Eyes The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=10 Eyes The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
The Change in GA, as Measured on Stereoscopic Color Fundus Photography (CFP) From Baseline to Year 2.	2.46 mm^2 Standard Deviation 1.25	2.47 mm^2 Standard Deviation 0.73

SECONDARY outcome

Timeframe: 2 years

Population: Data collected from the ten participants that completed the full twenty-four months of the study were analyzed. Ten study eyes and ten fellow eyes were analyzed.

The Pelli-Robson Chart is comprised of 10 groups of 3 large letters with levels of contrast ranging from 100% (black against white) to 1% (very light gray against white). Each eye is assigned a score based on the contrast of the last group in which two or three letters were correctly read. A score of 2 log units, which represents a normal sensitivity contrast, indicates that the eye was able to detect two of the three letters with a contrast of 1 percent (contrast sensitivity = 100 percent or log 2).

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=10 Eyes The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=10 Eyes The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
The Change in Contrast Sensitivity as Measured by the Pelli-Robson Chart From Baseline to Year 2.	0.075 Log Units Standard Deviation 0.33	-0.15 Log Units Standard Deviation 0.27

SECONDARY outcome

Timeframe: 2 years

Population: Data were only collected in 8 of the 10 participants that completed the full 24 months. Eight study eyes and eight fellow eyes were analyzed.

Scotomatous points are testing points on microperimetry examination that are centered on the macula and report a lack of retinal sensitivity within the range tested.

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=8 Participants The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=8 Participants The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
Number of Participants With an Increase in the Number of Scotomatous Points Between Study and Fellow Eyes From Baseline to Year 2.	7 participants	7 participants

SECONDARY outcome

Timeframe: Baseline, 2 years

Outcome measures

Outcome measures
Measure	Study Eye: OT-511 Antioxidant Eye Drop n=10 Participants The study eye was treated with one drop (40µL) of 0.45% OT-551 antioxidant eye drops three times a day.	Fellow Eye n=10 Participants The fellow eye was not treated with the study medication (OT-511 antioxidant eye drops).
The Change in Total Drusen Area From Baseline to Year 2.	-0.09 mm^2 Standard Error 0.22	-0.06 mm^2 Standard Error 0.14

Adverse Events

Participant Adverse Event Information

Serious events: 0 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Participant Adverse Event Information n=11 participants at risk
Eye disorders Retinal haemmorhage	36.4% 4/11 • Number of events 5 • Adverse event data were collected between December 2006 through May 2010.
Investigations Intraocular pressure increased	9.1% 1/11 • Number of events 2 • Adverse event data were collected between December 2006 through May 2010.
Injury, poisoning and procedural complications Joint injury	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Surgical and medical procedures Mole excision	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Investigations Blood cholesterol increased	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Cataract subcapsular	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Infections and infestations Upper respiratory tract infection	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Basal cell carcinoma	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Vision blurred	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Respiratory, thoracic and mediastinal disorders Dry throat	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Infections and infestations Candidiasis	9.1% 1/11 • Number of events 2 • Adverse event data were collected between December 2006 through May 2010.
Infections and infestations Tooth abscess	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamas cell carcinoma	18.2% 2/11 • Number of events 2 • Adverse event data were collected between December 2006 through May 2010.
Gastrointestinal disorders Oedema mouth	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Musculoskeletal and connective tissue disorders Muscle spasms	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Eye pain	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Visual acuity reduced	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Immune system disorders Allergy to arthropod sting	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Nervous system disorders Transient ischaemic attack	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Eye disorders Eyelid oedema	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Skin and subcutaneous tissue disorders Skin haemorrhage	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Infections and infestations Herpes zoster	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Surgical and medical procedures Knee arthoplasty	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Skin and subcutaneous tissue disorders Dry skin	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Endocrine disorders Hypothyroidism	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Metabolism and nutrition disorders Hypercholesterolaemia	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Injury, poisoning and procedural complications Nerve root injury cervical	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Metabolism and nutrition disorders Start of new cholesterol-lowering medication	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.
Surgical and medical procedures Spinal laminectomy	9.1% 1/11 • Number of events 1 • Adverse event data were collected between December 2006 through May 2010.

Additional Information

Wai Wong, MD, PhD

National Eye Institute

Phone: 301-496-7566

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place