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

NCT01093170 | Withdrawn Phase 1

• 1 other identifier: 144101a
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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).

Conditions

Geographic Atrophy

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.

  Through study completion or discontinuation

Secondary Outcomes (2)

• Pharmacokinetic studies measuring drug concentrations in serum over study period.

  Through study completion or discontinuation

• Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)

  Through duration of study or discontinuation

Study Arms (1)

RNA-144101

EXPERIMENTAL

Drug: RNA-144101

Interventions

RNA-144101 DRUG

Intravitreous administration of RNA-144101

RNA-144101

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:

You may not qualify if:

• 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
• 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
• 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

Sponsors & Collaborators

• Mark Kleinman: lead

Study Sites (1)

University of Kentucky / Dept of Ophthalmology

Lexington, Kentucky, 40536, United States

Location

MeSH Terms

Conditions

Geographic Atrophy

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Jayakrishna Ambati, MD

  University of Kentucky

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor/PI

Study Record Dates

• First Submitted: March 22, 2010
• First Posted: March 25, 2010
• Study Start: March 1, 2011
• Primary Completion: September 1, 2012
• Study Completion: September 1, 2012
• Last Updated: January 17, 2018

Record last verified: 2018-01

Locations

US (1)