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

January 15, 2018 updated by: Mark Kleinman
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).

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky / Dept of Ophthalmology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

50 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RNA-144101
Drug: RNA-144101
Intravitreous administration of RNA-144101

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Through study completion or discontinuation

Secondary Outcome Measures

Outcome Measure
Time Frame
Pharmacokinetic studies measuring drug concentrations in serum over study period.
Time Frame: Through study completion or discontinuation
Through study completion or discontinuation
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
Through duration of study or discontinuation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mark Kleinman

Investigators

  • Principal Investigator: Jayakrishna Ambati, MD, University of Kentucky

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2011

Primary Completion (Actual)

September 1, 2012

Study Completion (Actual)

September 1, 2012

Study Registration Dates

First Submitted

March 22, 2010

First Submitted That Met QC Criteria

March 24, 2010

First Posted (Estimate)

March 25, 2010

Study Record Updates

Last Update Posted (Actual)

January 17, 2018

Last Update Submitted That Met QC Criteria

January 15, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 144101a

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Geographic Atrophy

Clinical Trials on RNA-144101

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in South Africa

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe