Trial Outcomes & Findings for Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral GA Associated With AMD (NCT NCT00766649)
NCT ID: NCT00766649
Last Updated: 2020-07-14
Results Overview
One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
COMPLETED
PHASE1/PHASE2
11 participants
Baseline and Month 24
2020-07-14
Participant Flow
Participant milestones
| Measure |
Sirolimus
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral GA Associated With AMD
Baseline characteristics by cohort
| Measure |
Sirolimus
n=11 Participants
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
|
Age, Continuous
|
78.4 years
STANDARD_DEVIATION 7.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Month 24One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Participants
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Rate of Change in Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
|
0.102 MPS DA/month
Standard Deviation 0.049
|
PRIMARY outcome
Timeframe: Baseline and Month 24One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Participants
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Rate of Change in Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
|
0.087 MPS DA/month
Standard Deviation 0.034
|
SECONDARY outcome
Timeframe: Baseline and Month 24Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. 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 equivalent Snellen measurement is 20/20.
Outcome measures
| Measure |
Sirolimus
n=8 Participants
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Number of Study Eyes With a 15 Letter Drop From Baseline at 24 Months
|
4 eyes
|
SECONDARY outcome
Timeframe: Baseline and Month 24Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). This outcome measure was calculated by subtracting the GA value for the study eye at baseline from the GA value for the study eye at Month 24. One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Absolute Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
|
2.46 MPS DA
Standard Deviation 1.18
|
SECONDARY outcome
Timeframe: Baseline and Month 24Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). This outcome measure was calculated by subtracting the GA value for the fellow eye at baseline from the GA value for the study eye at Month 24. One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Absolute Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
|
2.08 MPS DA
Standard Deviation 0.83
|
SECONDARY outcome
Timeframe: Baseline to Month 24Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). This outcome measure was calculated by dividing the absolute change in the total area of GA in the study eye at 24 months by the baseline value. One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina. As the unit of measure for both the absolute change in the total area of GA and the baseline value for the total area of GA is MPS DA, the unit of measure for the relative change in the total area of GA is "ratio."
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Relative Change in Total Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline
|
0.55 Ratio
Standard Deviation 0.62
|
SECONDARY outcome
Timeframe: Baseline and Month 24Geographic atrophy (GA) is the death of photoreceptors and surrounding cells in the retina. The death of these photoreceptors results in lesions that cause vision loss. The area of GA was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). This outcome measure was calculated by dividing the absolute change in the total area of GA in the fellow eye at 24 months by the baseline value. One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina. As the unit of measure for both the absolute change in the total area of GA and the baseline value for the total area of GA is MPS DA, the unit of measure for the relative change in the total area of GA is "ratio."
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Relative Change in Total Area of Geographic Atrophy (GA) in the Fellow Eye at 24 Months as Compared to Baseline
|
0.40 Ratio
Standard Deviation 0.25
|
SECONDARY outcome
Timeframe: Baseline and Month 24The total area occupied by drusen was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Change in Total Area of Drusen in the Study Eye at 24 Months as Compared to Baseline
|
0.04 MPS DA
Standard Deviation 0.58
|
SECONDARY outcome
Timeframe: Baseline and Month 24The total area occupied by drusen was determined using planimetry for color stereoscopic fundus images by masked graders at the Doheny Image Reading Center (University of Southern California, Los Angeles, CA). One Macular Photocoagulation Study Disc Area (MPS DA) is equivalent to 1.77 mm\^2 on the retina.
Outcome measures
| Measure |
Sirolimus
n=8 Eyes
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Change in Total Area of Drusen in the Fellow Eye at 24 Months as Compared to Baseline
|
0.08 MPS DA
Standard Deviation 0.36
|
SECONDARY outcome
Timeframe: Baseline and Month 24Outcome measures
| Measure |
Sirolimus
n=8 Participants
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Change in Drusen Volume as Measured by Optical Coherence Tomography (OCT) at 24 Months as Compared to Baseline
|
0.04 mm^3
Standard Deviation 0.58
|
SECONDARY outcome
Timeframe: Baseline and Month 24Population: The PROS thickness analysis was not performed. As a result, data were not collected for this outcome measure.
Outcome measures
Outcome data not reported
Adverse Events
Sirolimus
Serious adverse events
| Measure |
Sirolimus
n=11 participants at risk
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Gastrointestinal disorders
Intestinal perforation
|
9.1%
1/11 • Number of events 1 • 42 months
|
Other adverse events
| Measure |
Sirolimus
n=11 participants at risk
Participants receive a 20 μL (440 μg) subconjunctival injection of sirolimus in the study eye at baseline and every three months thereafter.
|
|---|---|
|
Eye disorders
Eye discharge
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Eye disorders
Eye haemorrhage
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Eye disorders
Eye irritation
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Eye disorders
Foreign body sensation in eyes
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
General disorders
Oedema peripheral
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Infections and infestations
Influenza
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Infections and infestations
Nasopharyngitis
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Infections and infestations
Otitis externa
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Injury, poisoning and procedural complications
Back injury
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Injury, poisoning and procedural complications
Periorbital haematoma
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Alanine aminotransferase increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood chloride increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood creatine phosphokinase decreased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood creatinine increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood glucose increased
|
27.3%
3/11 • Number of events 3 • 42 months
|
|
Investigations
Blood lactate dehydrogenase increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood magnesium increased
|
36.4%
4/11 • Number of events 4 • 42 months
|
|
Investigations
Blood phosphorus decreased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood potassium increased
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
Blood uric acid increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Blood urine
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Carbon dioxide increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Haemoglobin decreased
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
Haemoglobin increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Intraocular pressure increased
|
9.1%
1/11 • Number of events 2 • 42 months
|
|
Investigations
Lymphocyte count decreased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Mean cell volume increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Monocyte count increased
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
Platelet count decreased
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
Protein total decreased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Red blood cell count decreased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Red blood cells urine positive
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
Red cell distribution width increased
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Urine ketone body present
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
Urine leukocyte esterase
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Investigations
White blood cell count increased
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Investigations
White blood cells urine
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
18.2%
2/11 • Number of events 2 • 42 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
18.2%
2/11 • Number of events 4 • 42 months
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
9.1%
1/11 • Number of events 1 • 42 months
|
|
Surgical and medical procedures
Cataract operation
|
9.1%
1/11 • Number of events 2 • 42 months
|
|
Vascular disorders
Varicose vein
|
9.1%
1/11 • Number of events 1 • 42 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place