Phase I/IIa Study of StarGen in Patients With Stargardt Macular Degeneration
The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, StarGen, designed to treat StarGardt Macular Degeneration.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen Administered to Patients With Stargardt Macular Degeneration|
- The incidence of adverse events [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]The number and percentage of patients with treatment emergent adverse events.
- Delay in retinal degeneration [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]Changes from baseline function relative to the contralateral eye utilising retinal analytical techniques.
|Study Start Date:||June 2011|
|Estimated Study Completion Date:||February 2017|
|Estimated Primary Completion Date:||February 2017 (Final data collection date for primary outcome measure)|
Experimental: Subretinal injection StarGen
Dose escalation of subretinally injected StarGen
3 dose cohorts, gene therapy
There are two parts to the study. A dose-escalation phase looking at three doses of StarGen, eight patients will be recruited at the first dose level, and four each at the next two dose levels. This will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in an additional twelve patients.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01367444
|Contact: David Wilson, M.D||503 494 3795|
|United States, Oregon|
|Oregon Health & Science University||Recruiting|
|Portland, Oregon, United States, 97239-3098|
|Principal Investigator: David Wilson, MD|
|Centre Hospitalier Nationale d'Ophthalmologie des Quinze-Vingts||Recruiting|
|Paris, France, 75571|
|Contact: Jose Sahel, MD. Ph.D|
|Principal Investigator: Jose Sahel, MD.Ph.D|
|Principal Investigator:||David Wilson, MD||Oregon Health & Science University, Portland, Oregon|
|Principal Investigator:||Jose-Alain Sahel, MD. Ph.D||Hopital Nationale des Quinze-Vingt, Paris France|