Back to resultsPhase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD) (GEM)
Primary Purpose
Age Related Macular Degeneration
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Subretinally injected RetinoStat
Sponsored by
About this trial
This is an interventional treatment trial for Age Related Macular Degeneration focused on measuring Age Related Macular Degeneration
Eligibility Criteria
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.
Sites / Locations
- The University of Iowa
- Johns Hopkins University Hospital
- Dr Andreas Lauer - Oregon Health & Science University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Subretinally Injected RetinoStat
Arm Description
Subretinally injected RetinoStat
Outcomes
Primary Outcome Measures
The incidence of adverse events
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
The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01301443
Brief Title
Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)
Acronym
GEM
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 Type
Interventional
2. Study Status
Record Verification Date
April 2017
Overall Recruitment Status
Completed
Study Start Date
February 2011 (undefined)
Primary Completion Date
September 2014 (Actual)
Study Completion Date
May 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Oxford BioMedica
4. Oversight
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Age Related Macular Degeneration
Keywords
Age Related Macular Degeneration
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Subretinally Injected RetinoStat
Arm Type
Experimental
Arm Description
Subretinally injected RetinoStat
Intervention Type
Drug
Intervention Name(s)
Subretinally injected RetinoStat
Other Intervention Name(s)
OXB-201
Intervention Description
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.
Primary Outcome Measure Information:
Title
The incidence of adverse events
Description
The number and percentage of patients with treatment emergent adverse events.
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Change from baseline in subretinal and intraretinal fluid as measured by OCT
Description
The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography
Time Frame
24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter A Campochiaro, MD
Organizational Affiliation
Johns Hopkins University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
The University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242-1098
Country
United States
Facility Name
Johns Hopkins University Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287-9277
Country
United States
Facility Name
Dr Andreas Lauer - Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Citations:
PubMed Identifier
27710144
Citation
Campochiaro PA, Lauer AK, Sohn EH, Mir TA, Naylor S, Anderton MC, Kelleher M, Harrop R, Ellis S, Mitrophanous KA. Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study. Hum Gene Ther. 2017 Jan;28(1):99-111. doi: 10.1089/hum.2016.117. Epub 2016 Sep 26.
Results Reference
result
Learn more about this trial
Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)
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