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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01301443
Recruitment Status : Completed
First Posted : February 23, 2011
Last Update Posted : April 5, 2017
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Age Related Macular Degeneration Drug: Subretinally injected RetinoStat Phase 1

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : February 2011
Primary Completion Date : September 2014
Study Completion Date : May 2015

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Subretinally Injected RetinoStat
Subretinally injected RetinoStat
Drug: Subretinally injected RetinoStat
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.
Other Name: OXB-201

Primary Outcome Measures :
  1. The incidence of adverse events [ Time Frame: 24 weeks ]
    The number and percentage of patients with treatment emergent adverse events.

Secondary Outcome Measures :
  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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Ages Eligible for Study:   50 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01301443

United States, Iowa
The University of Iowa
Iowa City, Iowa, United States, 52242-1098
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

Publications of Results:
Responsible Party: Oxford BioMedica Identifier: NCT01301443     History of Changes
Other Study ID Numbers: RS1/001/10
First Posted: February 23, 2011    Key Record Dates
Last Update Posted: April 5, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Oxford BioMedica:
Age Related Macular Degeneration

Additional relevant MeSH terms:
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases