A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy

• Status: Withdrawn
• Trial end date: 2012-09-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a non-randomized, open-label, dose-escalating Phase Ia study performed at a single center designed to evaluate the safety, pharmacokinetics, and preliminary efficacy of intravitreous administration of RNA-144101 in patients with geographic atrophy (GA).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Mark Kleinman; University of Kentucky

Criteria

Inclusion Criteria:

• Males and females of all races and ethnicities between the ages of 50-99

• Female subjects must be 1-year postmenopausal or surgically sterilized and must have negative serum pregnancy test

• Subjects must have GA from AMD in one or both eyes:

• The study eye will be the eye that meets all inclusion/exclusion criteria

• If both eyes meet criteria, then the eye with the smaller GA lesion will be studied

• If the GA lesions are equal in size, then the eye with the best-corrected visual acuity (BCVA) will chosen as the study eye

• If both eyes have the same lesion size and same BCVA, the right eye will be chosen

• Subjects must have the following GA criteria for inclusion:

• GA lesions with an area > 2.5mm2 and ≤ 20mm2 as determine by fluorescein angiography and fundus autofluorescence

• A clear view to the fundus must be present in order to easily examine the study eye at baseline

• BCVA In the study eye between 20/20 and 20/100 as measured on Snellen chart or 85-50 letters by ETDRS

• Willing and able to provide signed informed consent prior to any study participation

Exclusion Criteria:

• GA due to a disease other than AMD

• Pregnancy or lactation

• Treatment of any systemic infection

• Autofluorescence pattern marked at none, focal or patchy

• Ocular surgery in the study eye in the previous 6 months.

• Presence or history of choroidal neovascularization (wet AMD) in the study eye

• Any inflammatory (autoimmune, uveitis) or neovascular (diabetic retinopathy) disease of the retina

• Any history of glaucoma or disc cupping in the study eye

• Any history of severe dry eye disease

• High myopia > - 8D or high hyperopia > +8D in the study eye

• Presence of life-threatening disease

• Abnormal basal metabolic panel or liver function tests

• Current alcohol or other substance abuse

• Unwilling or unable to provide signed informed consent for any study procedures