A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
Primary Purpose
Geographic Atrophy
Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RNA-144101
Sponsored by
About this trial
This is an interventional treatment trial for Geographic Atrophy
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
Sites / Locations
- University of Kentucky / Dept of Ophthalmology
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RNA-144101
Arm Description
Outcomes
Primary Outcome Measures
Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.
Secondary Outcome Measures
Pharmacokinetic studies measuring drug concentrations in serum over study period.
Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01093170
Brief Title
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
2. Study Status
Record Verification Date
January 2018
Overall Recruitment Status
Withdrawn
Why Stopped
Study was not conducted, no subjects recruited.
Study Start Date
March 2011 (undefined)
Primary Completion Date
September 2012 (Actual)
Study Completion Date
September 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Mark Kleinman
4. Oversight
Data Monitoring Committee
No
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Geographic Atrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RNA-144101
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
RNA-144101
Intervention Description
Intravitreous administration of RNA-144101
Primary Outcome Measure Information:
Title
Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.
Time Frame
Through study completion or discontinuation
Secondary Outcome Measure Information:
Title
Pharmacokinetic studies measuring drug concentrations in serum over study period.
Time Frame
Through study completion or discontinuation
Title
Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)
Time Frame
Through duration of study or discontinuation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jayakrishna Ambati, MD
Organizational Affiliation
University of Kentucky
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Kentucky / Dept of Ophthalmology
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
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