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Geographic AtrophyPhase 2SirolimusNCT01675947

Intravitreal Injections of Sirolimus in the Treatment of Geographic Atrophy

A Phase 2 study testing Sirolimus and Lidocaine in people with Geographic Atrophy, run by National Eye Institute (NEI) at 13 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study will seek to enroll 50 persons who have central or non-central geographic atrophy (GA) associated with age-related macular degeneration (AMD). GA in treated eye must be between 0.75 disk areas (DA) and 8 DA. Read more about this study

Who is it for?
Ages 55 years and older
Conditions the study is for
Geographic Atrophy
What is being tested?
Sirolimus and Lidocaine
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
52 participants
Where
13 sitesUnited States
Study dates
Feb 2012to Feb 2016
Phase
Phase 2
Are there results?
None posted in the registry

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 55 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age 55 years of age or older. Prior participation in the Age-Related Eye Disease Study 2 (AREDS2) is not required.
  • Participant must understand and sign the protocol's informed consent document.
  • Participant must have central or non-central geographic atrophy (GA) in at least one eye. GA should be at least 0.75 disk areas (DA) in size but no more than 8 disk areas (DA); approximately 2.54 mm2 is 1 DA. GA is defined as one or more well-defined, usually more or less circular patches of partial or complete de-pigmentation of the retinal pigment epithelium (RPE), typically with exposure of underlying choroidal blood vessels. Even if much of the RPE appears to be preserved and large choroidal vessels are not visible, a round patch of RPE partial de-pigmentation may still be classified as early GA.
  • Participant must have a steady fixation in the study eye in the foveal or parafoveal area and media clear enough for good quality photographs.
  • Participant must have visual acuity between 20/25 and 20/200 in the study eye.
  • Female participants must be post-menopausal.
  • Male participants with female partners capable of conceiving children will be required to use contraception during the study and for four months after their last sirolimus injection.

Who cannot join

  • Participant is in another investigational study other than AREDS2 and actively receiving study therapy for geographic atrophy or choroidal neovascularization (CNV).
  • Participant is unable to comply with study procedures or follow-up visits.
  • Participant has evidence of ocular disease other than AMD in either eye that may confound the outcome of the study (e.g., glaucoma, diabetic retinopathy with 10 or more hemorrhages or microaneurysms, uveitis, pseudovitelliform macular degeneration, moderate/severe myopia).
  • Participant has received treatment for AMD, such as macular laser, photodynamic therapy (PDT) or anti-vascular endothelial growth factor (anti-VEGF) therapy injection. Or the participant received an IVT injection of any agent (e.g., triamcinolone) other than an anti-VEGF agent within the last four months prior to study enrollment. Vitamin supplementation for AMD is not considered an exclusionary criterion.
  • Participant has had a vitrectomy.
  • Participant is expected to need ocular surgery during the course of the trial.
Show all 16Show fewer
  • Participant has undergone lens removal in the last three months or Yttrium Aluminium Garnet (YAG) laser capsulotomy within the last month.
  • Participant is on chemotherapy.
  • Participant is on immunosuppressive medication or is immunosuppressed.
  • Participant is on ocular or systemic medications known to be toxic to the lens, retina or optic nerve.
  • Participant with a history of malignancy that would compromise the 2-year study survival.
  • Participant with a history of ocular herpes simplex virus (HSV).
  • 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).
  • 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.)
  • Ocular or periocular inflammation or infection in either eye.
  • Presence of active or inactive toxoplasmosis in the study eye.

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

United States13 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Elman Retina Group

    Baltimore, Maryland, 21237

  • Charlotte Eye Ear Nose & Throat Associates

    Charlotte, North Carolina, 28210

  • University of Illinois

    Chicago, Illinois, 60612

  • Texas Retina Associates

    Dallas, Texas, 75231

  • Vision Research Foundation

    Grand Rapids, Michigan, 49546

  • University of Florida HSC

    Jacksonville, Florida, 32209

  • Southeastern Retina Associates, P.C.

    Knoxville, Tennessee, 37909

  • Loma Linda University

    Loma Linda, California, 92354

  • University of Wisconsin

    Madison, Wisconsin, 53705

  • Scheie Eye Institute, University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • University of California, Davis

    Sacramento, California, 95817

  • Palmetto Retina Center

    West Columbia, South Carolina, 29169

Other trials in United States

About this study

Official title: Multi-Center, Randomized, Single Masked Phase 2 Study of Intravitreal Injections of Sirolimus in the Treatment of Geographic Atrophy Associated With Age-Related Macular Degeneration

This study will seek to enroll 50 persons who have central or non-central geographic atrophy (GA) associated with age-related macular degeneration (AMD). GA in treated eye must be between 0.75 disk areas (DA) and 8 DA. Eligible participants will be randomly chosen to receive one of the following treatments in the study eye:

  1. A 20 μL (440 μg) intravitreal (IVT) injection of sirolimus, or
  2. A sham treatment (subconjunctival injection of lidocaine) Participants with two (2) eligible eyes will have one eye randomly assigned to receive intravitreal sirolimus and no sham in the fellow eye.
Read more

The first injection will begin at Day 0, which may occur on the date of screening/enrollment or up to two weeks following screening/enrollment visit, and every month thereafter. The visit schedule is as follows:

  1. A clinical evaluation, including safety measures, will occur monthly.
  2. Vision will be measured at the screening/enrollment visit and at 2, 3, 6, 9, 12, 18 and 24 months after the first injection has occurred.
  3. Fundus autofluorescence will occur at screening/enrollment and at 2, 6, 12, 18 and 24 months after the first injection has occurred.
  4. Fundus color photography and optical coherence tomography will occur at screening/enrollment and at 6, 12, 18 and 24 months after the first injection has occurred.

The primary goal is to evaluate whether the persons receiving the sirolimus injections show a slower worsening of geographic atrophy compared to the persons receiving the sham injections. A secondary goal is to evaluate the impact of sirolimus on vision compared to the sham.

NOTE: As of May 30, 2014, study injections were discontinued due to safety concerns. No further enrollments will occur and follow-up will continue on all active study participants on a quarterly basis. That is, on visits coinciding with 3 month intervals from date of enrollment.

Being tested
Sirolimus, Lidocaine
Medical subject headings
Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • SirolimusDrug

    Also called Rapamycin, Rapamune

    Immunosuppressive agent. Blocks the T-lymphocyte activation and smooth muscle and endothelial cell proliferation that occurs in response to antigenic and cytokine (interleukin IL-2, IL-4 and IL-15) stimulation through either Ca2+-dependent or Ca2+-independent pathways. Sirolimus arrests cell cycle progression by direct interaction with two intracellular proteins (immunophilin FK binding protein 12 (FKBP-12) and the mammalian target of rapamycin (mTOR), a multifunctional serine-threonine kinase). In cells, sirolimus binds to FKBP-12, and the resulting sirolimus-FKBP-12 complex then binds to and inhibits mTOR.

  • LidocaineDrug

    Also called Xylocaine, lignocaine

    Lidocaine 2%

Study groups in NCT01675947
Study groupRoleWhat they receive
SirolimusExperimentalMonthly 20 μL (440 μg) intravitreal injection of sirolimus
Receives: Drug: Sirolimus
LidocaineSham comparatorMonthly subconjunctival injection of 2% lidocaine
Receives: Drug: Lidocaine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Rate of change in area of geographic atrophy

    Measured: Every 6 months after enrollment for 2 years

Secondary outcomes (1)

  1. Change in best-corrected visual acuity

    Measured: Every 6 months after enrollment for 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Aug 2012 (estimated)
  2. Study startFeb 2012
  3. Primary completionJun 2015
  4. Study completionFeb 2016 (estimated)

Why it stopped: On May 30, 2014, study injections were discontinued at the request of the DSMC. No further recruitment will occur.

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Participant
Enrolment
52 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01675947
Sponsor's protocol ID
3368-12056
Acronym
Sirolimus
Status last verified
Dec 2015
First submitted
28 Aug 2012
Last updated
10 Dec 2015
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sirolimus and Lidocaine in people with Geographic Atrophy, run by National Eye Institute (NEI) at 13 sites 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?

The sponsor lists 7 inclusion and 16 exclusion criteria. 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: stopped early, as of the sponsor's last update on 10 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

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?

The study was stopped early (reason given: On May 30, 2014, study injections were discontinued at the request of the DSMC. No further recruitment will occur.). Results are not available in the source record.

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?

Rate of change in area of geographic atrophy (time frame: Every 6 months after enrollment for 2 years). 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 NCT01675947.

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 10 Dec 2015.

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