Project 1

Clinical Study

Heads of  Project: Ingrid Scott, M.D., M.P.H. (POC1 Studies) & Greg Jackson, Ph.D. (ENDDR Studies)

Specific Aims:
The overall goal of the therapeutic module is to establish the infrastructure for proof-of-concept clinical testing of therapeutic agents for diabetic retinopathy, and the specific objectives are to investigate whether doxycycline can 1) slow the deterioration or improve retinal function and 2) induce regression, or slow progression, of diabetic retinopathy. Two 24 month proof-of-concept clinical studies will be conducted. 



The Diagnostic Module was rebranded as the “ENDpoints for Diabetic Retinopathy” (END DR) project which better reflects the ultimate goal of identifying better clinical trial endpoints. The data set was finalized in February 2009.  Preliminary study results were presented to the Penn State Hershey Eye Center and our external advisory committee.  The first public presentation of the data will occur at the Association for Vision Research and Ophthalmology meeting in Fort Lauderdale, FL in May 2009.  Manuscript preparation is underway.      


The POC 1 study was initiated during the project period.  Recruitment began in September 2008 and the first participant was enrolled in October 2008.  Currently, 6 patients have been enrolled and randomized.  Furthermore, we have reached out to ophthalmology practices in the south central Pennsylvania region to assist with indentifying potential participants.  We expect our enhanced recruitment strategies to be fruitful in the upcoming project period. 


We have developed the protocol for POC 2, and received approval for the study from our external advisory committee.  The protocol and informed consent form for POC 2 were approved by the Data Safety and Monitoring Committee (DSMC).


Who benefits from this research?:

Ultimately, the patient benefits from this research because this project lays the basic groundwork for the acceptance of new diagnostic tests for diabetic retinopathy, and the testing of future therapeutic agents for diabetic retinopathy.


Future Plans:

Patient recruitment will continue for POC 1, and patient recruitment for POC 2 is poised to begin pending final approval from JDRF.  Based upon feedback from industry, we plan to expand the patient population enrolled in the diagnostic module to include a cohort of patients with proliferative diabetic retinopathy and a cohort of patients with macular edema.


To best leverage the END DR study, we plan to follow the study population for 36 months of follow-up.  One of the tests, frequency doubling perimetry, appears to be a prime candidate for a useful functional endpoint.             


The next major initiatives are to conduct the POC 2 Study, to conduct additional proof-of-concept studies, and to conduct a detailed electrophysiological study of patients with diabetes both with and without retinopathy.