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

Mark Kleinman

Status and phase

Withdrawn

Phase 1

Conditions

Geographic Atrophy

Treatments

Drug: RNA-144101

Study type

Interventional

Funder types

Other

Identifiers

NCT01093170

144101a

Details and patient eligibility

About

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).

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

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