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Completed

NCT Number: NCT01301443

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

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.

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Key information

Conditions

Age Related Macular Degeneration Eye Diseases Macular Degeneration Retinal Degeneration Retinal Diseases

Age range

50 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

The University of Iowa, Iowa City, Iowa, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Subretinally injected RetinoStat

Drug

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

Other names: OXB-201

Primary outcomes

  1. The incidence of adverse events

    Time frame: 24 weeks

    The number and percentage of patients with treatment emergent adverse events.

Secondary outcomes

  1. Change from baseline in subretinal and intraretinal fluid as measured by OCT

    Time frame: 24 weeks

    The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography

Sponsors and collaborators

Lead sponsor

Oxford BioMedica

Industry

Registry information

Official study title

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

Acronym: GEM

Important dates

Study start
2011
Primary completion
2014
Study completion
2015
First posted
Feb 23, 2011
Registry last updated
Apr 5, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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