Phase 2
N=5
Sirolimus Injections for Autoimmune Scleritis
Scleritis
Bottom Line
View on ClinicalTrials.gov: NCT01517074 ↗Enrolled (actual)
5
Serious AEs
20.0%
Results posted
Jun 2014
Primary outcome: Primary: Number of Participants Who Experience at Least 2-step Reduction or Reduction to Grade 0 of Scleral Inflammation in the Study Eye According to the National Eye Institute (NEI) Photographic Scleritis Grading System Within 8 Weeks Post-injection. — 5 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sirolimus (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- National Eye Institute (NEI)
- Primary completion
- May 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Experience at Least 2-step Reduction or Reduction to Grade 0 of Scleral Inflammation in the Study Eye According to the National Eye Institute (NEI) Photographic Scleritis Grading System Within 8 Weeks Post-injection. |
5 | — |
| SECONDARY Number of Participants Who Experience a Disease Flare as Defined by a ≥ 1-step Increase in Scleral Inflammation |
3 | — |
| SECONDARY Mean Change in Visual Acuity Via the Early Treatment Diabetic Retinopathy Study (ETDRS) |
-0.25; -2.25 | — |
| SECONDARY Median Change in Visual Acuity Via the Early Treatment Diabetic Retinopathy Study (ETDRS) |
0.00; -2.50 | — |
| SECONDARY Number of Participants Needing a Second Injection |
3 | — |
| SECONDARY Mean Number of Days Between the First Injection to the Second Injection |
65.3 | — |
| SECONDARY Number of Participants Who Experienced Ocular Toxicities |
2 | — |
| SECONDARY Number of Participants Who Experienced Systemic Toxicities |
— | — |
| SECONDARY Number of Participants Who Tapered Off One or More Systemic Immunosuppressive Medications or Tapered Off Prednisone (≤10 mg) After Week 16 |
1 | — |
| SECONDARY Proportion of Participants With Loss of ≥ 15 Early Treatment Diabetic Retinopathy Study (ETDRS) Letters |
0; 1 | — |
| SECONDARY Number of Participants Who Experience a Substantial Rise in Elevated Intraocular Pressure (IOP) |
— | — |
| SECONDARY Step Changes in Scleral Inflammation According to the Standardized Photographic Grading System Developed at National Eye Institute (NEI) |
3; 0; -4; 1; 0; -2 | — |
Summary
Background:
Autoimmune scleritis is an inflammatory disease that affects the white outer part of the eye. It is associated with immune system disorders like rheumatoid arthritis. It can cause blindness in severe cases. Most treatments for scleritis involve steroid or immune-suppressing drugs, but these can cause side effects in the whole body.
Sirolimus is a drug used to help prevent transplant rejection. It helps prevent the immune system from attacking the body. Researchers want to try giving sirolimus injections into the eye to treat severe scleritis.
Objectives:
To see if sirolimus is a safe and effective treatment for autoimmune scleritis.
Eligibility:
Individuals at least 18 years of age with autoimmune scleritis in at least one eye that has not responded to standard treatments.
Design:
* Participants will be screened with a medical history, physical exam, and eye exam. Blood and urine samples will also be collected.
* One eye will be selected as the study eye to receive injections.
* Participants will have six study visits over 4 months (initial visit and weeks 2, 4, 8, 12, and 16). The injection will be given at the first visit. If the study eye responds to the treatment, participants may have injections in the other eye at the second visit.
* If there is still inflammation after the first injection, or if the scleritis improves but then returns, participants may have a second injection at Week 4.
* Injections will be monitored with blood tests and eye exams.
* Participants may have study visits and injections for up to 1 year if the injections seem to be working.
Eligibility Criteria
INCLUSION CRITERIA
- Participant has the ability to understand and sign the informed consent document.
- Participant is 18 years of age or older.
- Participant has a diagnosis of active, autoimmune, non-necrotizing, anterior scleritis.
- Participant, if currently taking immunosuppressive medications, is on a stable regimen of immunosuppressive medications (no increase and/or start of new immunosuppressive medications) over the last four weeks.
- Participant has tried therapy such as oral non-steroidal anti-inflammatory drugs (NSAIDs), or oral or topical corticosteroids or immunosuppressive medication at any time in the past to control scleritis flares, or has intolerance or contraindications to these medications.
- Participant is willing and able to comply with the study procedures.
- Female participants of childbearing potential must not be pregnant or breast-feeding, have a negative pregnancy test at screening and must be willing to undergo pregnancy tests throughout the study.
- Both female participants of childbearing potential and male participants able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse or must agree to practice two acceptable methods of contraception throughout the course of the study and for four months after the last investigational product injection. Acceptable methods of contraception include: hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, or surgical sterilization (tubal ligation).
EXCLUSION CRITERIA
- Participant has a significant active intraocular infection in either eye that requires antibiotic treatment.
- Participant has an active serious infection or a history of recurring serious infections such as human immunodeficiency virus (HIV) or syphilis that in the best medical judgment of the investigators would pose unnecessary risk to the participant.
- Participant has active joint or systemic inflammation requiring immediate addition or increase in systemic anti-inflammatory medications.
- Participant is taking systemic azole anti-fungal medication (e.g., ketoconazole, voriconazole, itraconazole).
STUDY EYE ELIGIBILITY CRITERIA:
The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below.
STUDY EYE INCLUSION CRITERIA:
- Participant has anterior scleritis with greater than or equal to 1 plus in at least one quadrant of the study eye.
- Participant has visual acuity in the study eye of 20/640 or better.
- Participant agrees not to undergo elective intraocular surgery in the study eye (e.g., cataract extraction) for three months after the last injection.
- Participant has not received a periocular or intravitreal injection in the study eye in the last six weeks.
STUDY EYE EXCLUSION CRITERIA:
- Participant has necrotizing scleritis in the study eye.
- Participant had intraocular surgery in the study eye in the last four weeks.
Data sourced from ClinicalTrials.gov (NCT01517074). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.