Trial Outcomes & Findings for Sirolimus to Treat Diabetic Macular Edema (NCT NCT00711490)

Last Updated: 2024-01-05

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

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

6 months

Results posted on

2024-01-05

Participant Flow

Participant milestones

Participant milestones
Measure	Sirolimus This is a single-arm study with all participants receiving a minimum of two 20 μL (440 μg) sirolimus injections: one injection at baseline and one injection at Month 2. Patients for whom re-treatment criteria were satisfied received additional injections every 2 months thereafter.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Sirolimus to Treat Diabetic Macular Edema

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sirolimus n=5 Participants This is a single-arm study with all participants receiving a minimum of two 20 μL (440 μg) sirolimus injections: one injection at baseline and one injection at Month 2. Patients for whom re-treatment criteria were satisfied received additional injections every 2 months thereafter.
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	4 Participants n=5 Participants
Age, Continuous	67.2 years STANDARD_DEVIATION 4.76 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Study Eye n=5 Participants The study eye was treated with sirolimus.	Fellow Eye n=5 Participants The fellow eye was not treated with sirolimus.
Change in Visual Acuity From Baseline to 6 Months	7 ETDRS letters Interval -7.0 to 13.0	0 ETDRS letters Interval -4.0 to 2.0

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Study Eye n=5 Participants The study eye was treated with sirolimus.	Fellow Eye n=5 Participants The fellow eye was not treated with sirolimus.
Change in Visual Acuity From Baseline to 12 Months	1 ETDRS letters Interval -8.0 to 13.0	-1 ETDRS letters Interval -3.0 to 1.0

SECONDARY outcome

Timeframe: 6 months

Retinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.

Outcome measures

Outcome measures
Measure	Study Eye n=5 Participants The study eye was treated with sirolimus.	Fellow Eye n=5 Participants The fellow eye was not treated with sirolimus.
Change in Retinal Thickness From Baseline to 6 Months, as Measured by Optical Coherence Tomography (OCT)	-37 μm Interval -163.0 to 74.0	-10 μm Interval -63.0 to 0.0

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome measures
Measure	Study Eye n=5 Participants The study eye was treated with sirolimus.	Fellow Eye n=5 Participants The fellow eye was not treated with sirolimus.
Change in Retinal Thickness From Baseline to 12 Months, as Measured by Optical Coherence Tomography (OCT)	-55 μm Interval -321.0 to 144.0	-51 μm Interval -152.0 to -7.0

SECONDARY outcome

Timeframe: 6 months

Population: Due to lack of effect in other outcome measures, at investigator's discretion, data for this outcome measure was neither collected nor analyzed. As such, zero participants have data for this outcome.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Outcome data not reported

Adverse Events

Sirolimus

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Sirolimus n=5 participants at risk This is a single-arm study with all participants receiving a minimum of two 20 μL (440 μg) sirolimus injections: one injection at baseline and one injection at Month 2. Patients for whom re-treatment criteria were satisfied received additional injections every 2 months thereafter.
Investigations Blood urea increased	40.0% 2/5 • Number of events 2 • Duration of study
Metabolism and nutrition disorders Hyperglycaemia	60.0% 3/5 • Number of events 4 • Duration of study
Investigations White blood cell count decreased	20.0% 1/5 • Number of events 1 • Duration of study
Musculoskeletal and connective tissue disorders Muscle spasms	20.0% 1/5 • Number of events 1 • Duration of study
Skin and subcutaneous tissue disorders Ecchymosis	20.0% 1/5 • Number of events 1 • Duration of study
General disorders Oedema peripheral	80.0% 4/5 • Number of events 5 • Duration of study
Blood and lymphatic system disorders Anaemia	40.0% 2/5 • Number of events 3 • Duration of study
Investigations Blood cholesterol increased	40.0% 2/5 • Number of events 2 • Duration of study
Injury, poisoning and procedural complications Rib fracture	20.0% 1/5 • Number of events 1 • Duration of study
Eye disorders Vision blurred	20.0% 1/5 • Number of events 1 • Duration of study
Respiratory, thoracic and mediastinal disorders Cough	20.0% 1/5 • Number of events 1 • Duration of study
Musculoskeletal and connective tissue disorders Arthralgia	20.0% 1/5 • Number of events 1 • Duration of study
Eye disorders Visual acuity reduced	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Blood glucose increased	40.0% 2/5 • Number of events 2 • Duration of study
Investigations Blood uric acid increased	40.0% 2/5 • Number of events 2 • Duration of study
Investigations Glycosylated haemoglobin increased	20.0% 1/5 • Number of events 1 • Duration of study
Eye disorders Conjunctival haemorrhage	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Red cell distribution width increased	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Blood creatine phosphokinase increased	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Haematocrit decreased	20.0% 1/5 • Number of events 1 • Duration of study
Infections and infestations Sinusitis	20.0% 1/5 • Number of events 1 • Duration of study
Investigations White blood cell count increased	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Protein urine present	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Intraocular pressure increased	20.0% 1/5 • Number of events 4 • Duration of study
Investigations Blood trigylcerides increased	40.0% 2/5 • Number of events 2 • Duration of study
Eye disorders Conjunctival hyperaemia	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Blood creatinine increased	40.0% 2/5 • Number of events 2 • Duration of study
Investigations Red blood cell count decreased	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Blood potassium increased	20.0% 1/5 • Number of events 2 • Duration of study
Investigations Blood pressure increased	20.0% 1/5 • Number of events 2 • Duration of study
Investigations Haemoglobin decreased	20.0% 1/5 • Number of events 1 • Duration of study
Investigations Blood chloride increased	20.0% 1/5 • Number of events 1 • Duration of study

Additional Information

Catherine Meyerle, MD

National Eye Institute

Phone: 301-435-7821

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place