Diabetic RetinopathyPhase 1/2SDMENCT00711490
Sirolimus to Treat Diabetic Macular Edema
A Phase 1/2 study testing Sirolimus in people with Diabetic Retinopathy, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
Objective: Diabetic macular edema (DME) is a frequent manifestation of diabetic retinopathy, a leading cause of blindness in the United States. The only proven treatment for DME is laser photocoagulation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Diabetic Retinopathy
- What is being tested?
- Sirolimus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2008to Jan 2011
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Participant-Level Inclusion Criteria
- Participant is 18 years of age or older.
- Diagnosis of diabetic mellitus (type 1 or type 2). Any one of the following will be considered to be sufficient evidence that diabetes is present:
- Current regular use of insulin for the treatment of diabetes.
- Current regular use of oral anti-hyperglycemia agents for the treatment of diabetes.
- Documented diabetes by American Diabetes Association (ADA) and/or World Health Organization (WHO) criteria.
- Documented hemoglobin A1C 12% or less within one month of baseline.
- Able and willing to provide informed consent.
- Both female participants of childbearing potential and male participants able to father a child must agree to practice two* forms of adequate birth control throughout the course of the study and for three months following the completion of the study treatment. Acceptable methods of birth control include hormonal contraception (birth control pills, injected hormones or vaginal ring), intrauterine device, barrier methods with spermicide (diaphragm with spermicide, condom and spermicide) or surgical sterilization (hysterectomy, tubal ligation or vasectomy). *Participants with a hysterectomy or vasectomy (or who have a partner with a hysterectomy or vasectomy) are exempt from using two methods of contraception. However, female participants with a tubal ligation (or male participants who have a female partner with a tubal ligation) are not exempt, and are required to practice another acceptable method of birth control.
- Female participants of childbearing potential must be willing to undergo pregnancy testing for the duration of the study.
- At least one eye meets the study eye criteria listed in Section 4.2.
Participant-Level Exclusion Criteria
- History of chronic renal failure requiring dialysis or kidney transplant.
- Positive serum or urine pregnancy test or currently lactating for women of childbearing potential.
- A condition that, in the opinion of the investigator, would preclude participation in the study (e.g., unstable medical status including blood pressure and glycemic control).
- Participation in an investigational trial within 30 days of study entry that involved treatment with any drug that has not received regulatory approval at the time of study entry.
- History of cancer (other than a non-melanoma skin cancer) diagnosed within the past five years that could be worsened by immunosuppression.* *The risk of immunosuppression must be determined by an oncology consultation prior to enrollment.
- Laboratory values outside normal limits and considered clinically significant by the investigator.
- Blood pressure > 180/110 (systolic above 180 OR diastolic above 110).
- History of intravitreal anti-VEGF therapy or subtenon/intravitreal steroids in either eye within three months prior to study entry.
- History of treatment with systemic anti-VEGF agents or steroids within one year prior to study entry.
- Participant is currently taking one of the following drugs: amprenavir, atazanavir, clarithromycin, darunavir, delavirdine, erythromycin, fluconazole (at doses of 200mg or greater), fluvoxamine, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, quinupristin, ritonavir, saquinavir, telithromycin, troleandomycin, verapamil or voriconazole.
Study Eye Inclusion Criteria
- Best-corrected Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity score of ≤ 74 letters (i.e., 20/32 or worse).
- Definite retinal thickening due to diabetic macular edema based on clinical exam involving the center of the macula that is not refractory to further therapy as based on the investigator's clinical judgment.
- Retinal thickness on baseline OCT measurement > 260 microns in the central subfield.
- Media clarity, pupillary dilation and patient cooperation sufficient for adequate fundus photographs.
Study Eye Exclusion Criteria
- Macular edema is considered to be due to a cause other than diabetic macular edema.
- An ocular condition is present such that, in the opinion of the investigator, visual acuity would not improve from resolution of macular edema (e.g., foveal atrophy, pigmentary changes, dense subfoveal hard exudates, nonretinal condition, etc.).
- An ocular condition is present (other than diabetic retinopathy) that, in the opinion of the investigator, might affect macular edema or alter visual acuity during the course of the study (e.g., vein occlusion, uveitis or other ocular inflammatory disease, Irvine-Gass Syndrome, etc.).
- Substantial cataract that, in the opinion of the investigator, is likely to be decreasing visual acuity by three lines or more (i.e., cataract would be reducing acuity to 20/40 or worse if eye was otherwise normal).
- History of focal/grid macular photocoagulation within 12 weeks (three months) prior to study entry.
- History of panretinal scatter photocoagulation (PRP) within four months prior to study entry.
- Anticipated need for PRP in the four months following study entry.
- History of prior pars plana vitrectomy.
- History of major ocular surgery (including cataract extraction, scleral buckle, any intraocular surgery, etc.) within prior six months or anticipated within the next six months following study entry.
- History of (Yttrium Aluminium Garnet) YAG laser capsulotomy performed within two months prior to study entry.
- Exam evidence of external ocular infection, including conjunctivitis, chalazion, or significant blepharitis.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Pilot Study of the Evaluation of Sirolimus in the Treatment of Diabetic Macular Edema
Objective: Diabetic macular edema (DME) is a frequent manifestation of diabetic retinopathy, a leading cause of blindness in the United States. The only proven treatment for DME is laser photocoagulation. Sirolimus has been shown to inhibit the production, signaling and activity of many growth factors relevant to the development of diabetic retinopathy. Therefore, this study will investigate the safety and efficacy of multiple sirolimus injections in patients with DME.
Study Population: Eligibility criteria include central macular thickening > 260 microns and visual acuity 20/32 or worse in one or both eyes.
Read more
Design: Five participants will be enrolled into this open-label pilot study. After receiving a 20 μL (440 μg) subconjunctival injection in the study eye at baseline and Month 2, the participants will be re-evaluated every two months for at least one year for possible additional injections. During follow-up, participants will not undergo re-injection if they show significant clinical improvement or treatment success, defined as no intraretinal fluid or cysts present on optical coherence tomography (OCT) OR 100% reduction in excess retinal thickness over 260 microns on OCT OR no leakage on fluorescein angiography (FA). Beginning at Month 4, participants will be assessed for treatment failure, defined as loss of 15 or more letters of vision compared to baseline at two consecutive visits OR a 50% or greater increase in total retinal thickness as measured by OCT at two consecutive visits. Individual participants deemed treatment failures will continue receiving sirolimus injections, but will be allowed to receive focal laser therapy for any amenable leaking microaneurysms at Month 4. Beginning at Month 6, focal laser therapy will be permitted for both treatment failures and participants who do not meet the criteria of a treatment success. Participants will have the option of continuing treatment until a common termination date of one year.
Outcome Measures: The primary outcome is the change in visual acuity in the study eye at six months compared to baseline. Secondary outcomes include changes in visual acuity in the study eye at one year as compared with baseline, changes in retinal thickness as measured by OCT and changes in fluid leakage in the macula as demonstrated by FA at six months, one year and throughout the study period in the study and fellow eyes. Safety outcomes include number and severity of systemic and ocular toxicities, adverse events and infections, and the number of participants withdrawn from study therapy.
- Conditions
- Diabetic Retinopathy
- Being tested
- Sirolimus
- Keywords
- Diabetic Macular Edema, Diabetic Retinopathy, Rapamycin, Sirolimus, Diabetes
- Medical subject headings
- Diabetic Retinopathy, Diabetes Mellitus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune
20 μL (440 μg) of sirolimus were injected at baseline, Month 2, and every 2 months thereafter if re-treatment criteria were satisfied.
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus | Experimental | The study eye was treated with sirolimus. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Visual Acuity From Baseline to 6 Months
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.
Measured: 6 months
Secondary outcomes (5)
Change in Visual Acuity From Baseline to 12 Months
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.
Measured: 12 months
Change in Retinal Thickness From Baseline to 6 Months, as Measured by Optical Coherence Tomography (OCT)
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.
Measured: 6 months
Change in Retinal Thickness From Baseline to 12 Months, as Measured by Optical Coherence Tomography (OCT)
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.
Measured: 12 months
Change in Fluid Leakage in the Macula of the Study Eye From Baseline to 6 Months, as Measured on Fluorescein Angiography (FA)
Measured: 6 months
Change in Fluid Leakage in the Macula of the Study Eye From Baseline to 12 Months, as Measured on Fluorescein Angiography (FA)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2008 (estimated)
- Study startJul 2008
- Primary completionJan 2011
- Study completionJan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Diabetic RetinopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Catherine Meyerle, MD, Principal investigator, National Eye Institute/ National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00711490
- Sponsor's protocol ID
- 080175
- Other IDs
- 08-EI-0175
- Acronym
- SDME
- Status last verified
- Sep 2016
- First submitted
- 8 Jul 2008
- Last updated
- 5 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Kempen JH, O'Colmain BJ, Leske MC, Haffner SM, Klein R, Moss SE, Taylor HR, Hamman RF; Eye Diseases Prevalence Research Group. The prevalence of diabetic retinopathy among adults in the United States. Arch Ophthalmol. 2004 Apr;122(4):552-63. doi: 10.1001/archopht.122.4.552.
PubMed 15078674Klein R, Klein BE, Moss SE, Davis MD, DeMets DL. The Wisconsin epidemiologic study of diabetic retinopathy. IV. Diabetic macular edema. Ophthalmology. 1984 Dec;91(12):1464-74. doi: 10.1016/s0161-6420(84)34102-1.
PubMed 6521986Photocoagulation for diabetic macular edema. Early Treatment Diabetic Retinopathy Study report number 1. Early Treatment Diabetic Retinopathy Study research group. Arch Ophthalmol. 1985 Dec;103(12):1796-806.
PubMed 2866759Krishnadev N, Forooghian F, Cukras C, Wong W, Saligan L, Chew EY, Nussenblatt R, Ferris F 3rd, Meyerle C. Subconjunctival sirolimus in the treatment of diabetic macular edema. Graefes Arch Clin Exp Ophthalmol. 2011 Nov;249(11):1627-33. doi: 10.1007/s00417-011-1694-9. Epub 2011 May 13.
PubMed 21567211
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of the Evaluation of Sirolimus in the Treatment of Diabetic Macular Edema
- ClinicalTrials.gov ID
- NCT00711490
- Acronym / sponsor ID
- SDME / 080175
- Conditions
- Diabetic Retinopathy
- Keywords
- Diabetic Macular Edema, Diabetic Retinopathy, Rapamycin, Sirolimus, Diabetes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- Jul 2008
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 9 Jul 2008 (estimated)
- Results
- Posted 3 Jun 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Objective: Diabetic macular edema (DME) is a frequent manifestation of diabetic retinopathy, a leading cause of blindness in the United States. The only proven treatment for DME is laser photocoagulation. Sirolimus has been shown to inhibit the production, signaling and activity of many growth factors relevant to the development of diabetic retinopathy. Therefore, this study will investigate the safety and efficacy of multiple sirolimus injections in patients with DME.
Study Population: Eligibility criteria include central macular thickening > 260 microns and visual acuity 20/32 or worse in one or both eyes.
Design: Five participants will be enrolled into this open-label pilot study. After receiving a 20 μL (440 μg) subconjunctival injection in the study eye at baseline and Month 2, the participants will be re-evaluated every two months for at least one year for possible additional injections. During follow-up, participants will not undergo re-injection if they show significant clinical improvement or treatment success, defined as no intraretinal fluid or cysts present on optical coherence tomography (OCT) OR 100% reduction in excess retinal thickness over 260 microns on OCT OR no leakage on fluorescein angiography (FA). Beginning at Month 4, participants will be assessed for treatment failure, defined as loss of 15 or more letters of vision compared to baseline at two consecutive visits OR a 50% or greater increase in total retinal thickness as measured by OCT at two consecutive visits. Individual participants deemed treatment failures will continue receiving sirolimus injections, but will be allowed to receive focal laser therapy for any amenable leaking microaneurysms at Month 4. Beginning at Month 6, focal laser therapy will be permitted for both treatment failures and participants who do not meet the criteria of a treatment success. Participants will have the option of continuing treatment until a common termination date of one year.
Outcome Measures: The primary outcome is the change in visual acuity in the study eye at six months compared to baseline. Secondary outcomes include changes in visual acuity in the study eye at one year as compared with baseline, changes in retinal thickness as measured by OCT and changes in fluid leakage in the macula as demonstrated by FA at six months, one year and throughout the study period in the study and fellow eyes. Safety outcomes include number and severity of systemic and ocular toxicities, adverse events and infections, and the number of participants withdrawn from study therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus | Experimental | Drug: SirolimusThe study eye was treated with sirolimus. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SirolimusAlso: Rapamune20 μL (440 μg) of sirolimus were injected at baseline, Month 2, and every 2 months thereafter if re-treatment criteria were satisfied. | Drug | Sirolimus |
Primary outcome
Change in Visual Acuity From Baseline to 6 Months
Time frame: 6 months
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.
5 secondary outcomes
Change in Visual Acuity From Baseline to 12 Months
Time frame: 12 months
Change in Retinal Thickness From Baseline to 6 Months, as Measured by Optical Coherence Tomography (OCT)
Time frame: 6 months
Change in Retinal Thickness From Baseline to 12 Months, as Measured by Optical Coherence Tomography (OCT)
Time frame: 12 months
Change in Fluid Leakage in the Macula of the Study Eye From Baseline to 6 Months, as Measured on Fluorescein Angiography (FA)
Time frame: 6 months
Change in Fluid Leakage in the Macula of the Study Eye From Baseline to 12 Months, as Measured on Fluorescein Angiography (FA)
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 15078674Kempen JH, O'Colmain BJ, Leske MC, Haffner SM, Klein R, Moss SE, Taylor HR, Hamman RF; Eye Diseases Prevalence Research Group. The prevalence of diabetic retinopathy among adults in the United States. Arch Ophthalmol. 2004 Apr;122(4):552-63. doi: 10.1001/archopht.122.4.552.
- Background: PubMed 6521986Klein R, Klein BE, Moss SE, Davis MD, DeMets DL. The Wisconsin epidemiologic study of diabetic retinopathy. IV. Diabetic macular edema. Ophthalmology. 1984 Dec;91(12):1464-74. doi: 10.1016/s0161-6420(84)34102-1.
- Background: PubMed 2866759Photocoagulation for diabetic macular edema. Early Treatment Diabetic Retinopathy Study report number 1. Early Treatment Diabetic Retinopathy Study research group. Arch Ophthalmol. 1985 Dec;103(12):1796-806.
- Results publication: PubMed 21567211Krishnadev N, Forooghian F, Cukras C, Wong W, Saligan L, Chew EY, Nussenblatt R, Ferris F 3rd, Meyerle C. Subconjunctival sirolimus in the treatment of diabetic macular edema. Graefes Arch Clin Exp Ophthalmol. 2011 Nov;249(11):1627-33. doi: 10.1007/s00417-011-1694-9. Epub 2011 May 13.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Catherine Meyerle, MD principal investigator
National Eye Institute/ National Institutes of Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sirolimus in people with Diabetic Retinopathy, run by National Eye Institute (NEI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jun 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Visual Acuity From Baseline to 6 Months (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00711490, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2024.