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

Sponsor
Oxford BioMedica (Industry)
Overall Status
Completed
CT.gov ID
NCT01301443
Collaborator
(none)
21
Enrollment
3
Locations
1
Arm
50.9
Duration (Months)
7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

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
  • 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 Design

Study Type:
Interventional
Actual Enrollment :
21 participants
Allocation:
N/A
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 :
Feb 1, 2011
Actual Primary Completion Date :
Sep 1, 2014
Actual Study Completion Date :
May 1, 2015

Arms and Interventions

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 Names:
  • OXB-201

    		•

    Outcome Measures

    Primary Outcome Measures

    1. The incidence of adverse events [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 [24 weeks]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    50 Years and Older
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 The University of Iowa Iowa City Iowa United States 52242-1098
    2 Johns Hopkins University Hospital Baltimore Maryland United States 21287-9277
    3 Dr Andreas Lauer - Oregon Health & Science University Portland Oregon United States 97239

    Sponsors and Collaborators

    • Oxford BioMedica

    Investigators

    • Principal Investigator: Peter A Campochiaro, MD, Johns Hopkins University Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Oxford BioMedica
    ClinicalTrials.gov Identifier:
    NCT01301443
    Other Study ID Numbers:
    • RS1/001/10
    First Posted:
    Feb 23, 2011
    Last Update Posted:
    Apr 5, 2017
    Last Verified:
    Apr 1, 2017
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    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

    Study Results

    No Results Posted as of Apr 5, 2017