Clinical trial NCT01093170: A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
Official title: A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
Conditions: Geographic Atrophy
Lead sponsor: University of Kentucky
Sponsor class: OTHER
First posted: 2010-03-25
Enrollment: 0
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility 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

Primary outcomes:
measure: Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.
timeFrame: Through study completion or discontinuation
Secondary outcomes:
measure: Pharmacokinetic studies measuring drug concentrations in serum over study period.
timeFrame: Through study completion or discontinuation
measure: Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)
timeFrame: Through duration of study or discontinuation
Other outcomes:
Locations:
city: Lexington
country: United States
facility: University of Kentucky / Dept of Ophthalmology
state: Kentucky
Study officials:
affiliation: University of Kentucky
name: Jayakrishna Ambati, MD
role: PRINCIPAL_INVESTIGATOR
References: