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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Withdrawn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

About this trial

This is an interventional treatment trial for Geographic Atrophy

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

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

NCT ID

NCT01093170

First Posted

March 22, 2010

Last Updated

January 15, 2018

Sponsor

Mark Kleinman

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

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

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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