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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Oxford BioMedica Identifier:
First received: February 21, 2011
Last updated: April 20, 2015
Last verified: April 2015
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.

Condition Intervention Phase
Age Related Macular Degeneration
Drug: 3 dose cohorts gene therapy
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:

Further study details as provided by Oxford BioMedica:

Primary Outcome Measures:
  • The incidence of adverse events [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
    The number and percentage of patients with treatment emergent adverse events.

Secondary Outcome Measures:
  • Change from baseline in subretinal and intraretinal fluid as measured by OCT [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography

Enrollment: 21
Study Start Date: February 2011
Estimated Study Completion Date: May 2015
Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Subretinally Injected RetinoStat
Dose escalation of subretinally injected RetinoStat
Drug: 3 dose cohorts gene therapy
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.

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.

Ages Eligible for Study:   50 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
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Please refer to this study by its identifier: NCT01301443

United States, Iowa
The University of Iowa
Iowa City, Iowa, United States
United States, Maryland
Johns Hopkins University Hospital
Baltimore, Maryland, United States, 21287-9277
United States, Oregon
Dr Andreas Lauer - Oregon Health & Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Oxford BioMedica
Principal Investigator: Peter A Campochiaro, MD Johns Hopkins University Hospital
  More Information

Responsible Party: Oxford BioMedica Identifier: NCT01301443     History of Changes
Other Study ID Numbers: RS1/001/10 
Study First Received: February 21, 2011
Last Updated: April 20, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Oxford BioMedica:
Age Related Macular Degeneration

Additional relevant MeSH terms:
Macular Degeneration
Eye Diseases
Retinal Degeneration
Retinal Diseases processed this record on May 23, 2016