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

A Phase I Dose Escalation Safety Study of Subretinally Injected RetinoStat, a Lentiviral Vector Expressing Endostatin and Angiostatin, in Patients With Advanced Neovascular Age-Related Macular Degeneration

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01301443

Acronym

GEM

Enrollment

Registered

2011-02-23

Start date

2011-02-28

Completion date

2015-05-31

Last updated

2017-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Age Related Macular Degeneration

Keywords

Age Related Macular Degeneration

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.

Interventions

DRUGSubretinally injected RetinoStat

Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

50 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
The incidence of adverse events	24 weeks	The number and percentage of patients with treatment emergent adverse events.

Secondary

Measure	Time frame	Description
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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026