Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)

Phase 1

Completed

• Conditions: Age Related Macular Degeneration
• Interventions: Drug: Subretinally injected RetinoStat

• Registration Number: NCT01301443
• Lead Sponsor: Oxford BioMedica

Brief Summary

The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related macular degeneration.

Detailed Description

There are two parts to the study. A dose-escalation phase looking at three doses of RetinoStat starting with the lowest dose, three patients will be recruited at each dose level. The escalation phase will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in 9 patients.

Study Timeline

• Study Start: 2011-02
• Study First Submitted: 2011-02-21
• Study First Submitted QC: 2011-02-22
• Study First Posted: 2011-02-23
• Primary Completion: 2014-09
• Study Completion: 2015-05
• Status Verified: 2017-04
• Last Update Submitted: 2017-04-03
• Last Update Posted: 2017-04-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

• Clinical diagnosis of AMD with active CNV that shows evidence of leakage.
• BCVA less than or equal to 20/200 in the study eye for dose escalation phase.
• BCVA less than or equal to 20/80 in the study eye for maximum tolerated dose phase.

Exclusion Criteria

• Significant ocular abnormalities that prevent retinal assessment.
• Treatment with steroids within three months of screening.
• Treatment with anti-VEGF therapy to either eye within one month of screening.
• Clinically significant intercurrent illnesses, laboratory, ECG or chest XRay abnormalities.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Subretinally Injected RetinoStat	Subretinally injected RetinoStat	Subretinally injected RetinoStat

Primary Outcome Measures

Name	Time	Method
The incidence of adverse events	24 weeks	The number and percentage of patients with treatment emergent adverse events.

Secondary Outcome Measures

Name	Time	Method
Change from baseline in subretinal and intraretinal fluid as measured by OCT	24 weeks	The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography

Trial Locations

Locations (3)

Dr Andreas Lauer - Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

The University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Johns Hopkins University Hospital; 🇺🇸 Baltimore, Maryland, United States