Geographic Atrophy
Conditions
Brief 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).
Interventions
DRUGRNA-144101
Intravitreous administration of RNA-144101
Sponsors
Mark Kleinman
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs. | Through study completion or discontinuation |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic studies measuring drug concentrations in serum over study period. | Through study completion or discontinuation |
| Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging) | Through duration of study or discontinuation |
Countries
United States
Outcome results
None listed