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

Study Overview
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Brief Summary
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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).

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

Conditions
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Geographic Atrophy

Intervention / Treatment
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* Drug: RNA-144101


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RNA-144101<br> | Drug: RNA-144101<br>* Intravitreous administration of RNA-144101<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 Outcome Measures

| Outcome Measure | Measure Description | 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 |