Sirolimus Injections for Autoimmune Scleritis (ISAS)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) )
ClinicalTrials.gov Identifier:
NCT01517074
First received: January 24, 2012
Last updated: June 11, 2014
Last verified: June 2014
Results First Received: May 12, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Scleritis
Intervention: Drug: Sirolimus

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Sirolimus Participants will receive a15 μL (660 μg) subconjunctival injection of sirolimus in the study eye at baseline if a single quadrant or two adjacent quadrants are involved. If greater than two quadrants are involved (i.e., 3 or 4 quadrant involvement) or two non-adjacent quadrants are involved, two 15 μL (660 μg) injections will be given in two quadrants 180 degrees apart (total dose of 30 μL or 1,320 μg). Participants that still demonstrate active inflammation (incomplete or no response to initial injection) or experience a flare-up (as defined by a ≥1-step increase in scleral inflammation) after the initial injection will be eligible for a re-injection in the study eye at or after Week 4 (not to exceed a dose of 1,320 μg per eye within an eight-week period).

Participant Flow:   Overall Study
    Sirolimus  
STARTED     5  
COMPLETED     5  
NOT COMPLETED     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Sirolimus Participants will receive a15 μL (660 μg) subconjunctival injection of sirolimus in the study eye at baseline if a single quadrant or two adjacent quadrants are involved. If greater than two quadrants are involved (i.e., 3 or 4 quadrant involvement) or two non-adjacent quadrants are involved, two 15 μL (660 μg) injections will be given in two quadrants 180 degrees apart (total dose of 30 μL or 1,320 μg). Participants that still demonstrate active inflammation (incomplete or no response to initial injection) or experience a flare-up (as defined by a ≥1-step increase in scleral inflammation) after the initial injection will be eligible for a re-injection in the study eye at or after Week 4 (not to exceed a dose of 1,320 μg per eye within an eight-week period).

Baseline Measures
    Sirolimus  
Number of Participants  
[units: participants]
  5  
Age  
[units: years]
Mean ± Standard Deviation
  50.6  ± 15.9  
Age  
[units: participants]
 
<=18 years     0  
Between 18 and 65 years     5  
>=65 years     0  
Gender  
[units: participants]
 
Female     3  
Male     2  
Region of Enrollment  
[units: participants]
 
United States     5  



  Outcome Measures
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1.  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.   [ Time Frame: Baseline and Week 8 ]

2.  Secondary:   Number of Participants Who Experience a Disease Flare as Defined by a ≥ 1-step Increase in Scleral Inflammation   [ Time Frame: Baseline and Week 52 ]

3.  Secondary:   Number of Participants Needing a Second Injection   [ Time Frame: Baseline and Week 52 ]

4.  Secondary:   Mean Number of Days Between the First Injection to the Second Injection   [ Time Frame: Baseline and Week 52 ]

5.  Secondary:   Number of Participants Who Experienced Ocular Toxicities   [ Time Frame: Baseline and Week 52 ]

6.  Secondary:   Number of Participants Who Experienced Systemic Toxicities   [ Time Frame: Baseline and Week 52 ]

7.  Secondary:   Number of Participants Who Tapered Off One or More Systemic Immunosuppressive Medications or Tapered Off Prednisone (≤10 mg) After Week 16   [ Time Frame: Week 16 and Week 52 ]

8.  Secondary:   Number of Participants Who Experience a Substantial Rise in Elevated Intraocular Pressure (IOP)   [ Time Frame: Baseline and Week 52 ]

9.  Secondary:   Mean Change in Visual Acuity Via the Early Treatment Diabetic Retinopathy Study (ETDRS)   [ Time Frame: Baseline and Week 52 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

10.  Secondary:   Median Change in Visual Acuity Via the Early Treatment Diabetic Retinopathy Study (ETDRS)   [ Time Frame: Baseline and Week 52 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No

11.  Secondary:   Proportion of Participants With Loss of ≥ 15 Early Treatment Diabetic Retinopathy Study (ETDRS) Letters   [ Time Frame: Baseline and Week 52 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   Yes

12.  Secondary:   Step Changes in Scleral Inflammation According to the Standardized Photographic Grading System Developed at National Eye Institute (NEI)   [ Time Frame: Baseline and Week 52 ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: H. Nida Sen, MD, MHSc, Principal Investigator, NEI
Organization: National Institutes of Health
phone: 301-402-3254
e-mail: senh@nei.nih.gov


Publications:

Responsible Party: National Institutes of Health Clinical Center (CC) ( National Eye Institute (NEI) )
ClinicalTrials.gov Identifier: NCT01517074     History of Changes
Other Study ID Numbers: 120057, 12-EI-0057
Study First Received: January 24, 2012
Results First Received: May 12, 2014
Last Updated: June 11, 2014
Health Authority: United States: Federal Government
United States: Food and Drug Administration