Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)
GEM
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
1 other identifier
interventional
21
1 country
3
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Feb 2011
Longer than P75 for phase_1
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2011
CompletedFirst Submitted
Initial submission to the registry
February 21, 2011
CompletedFirst Posted
Study publicly available on registry
February 23, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2015
CompletedApril 5, 2017
April 1, 2017
3.6 years
February 21, 2011
April 3, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The incidence of adverse events
The number and percentage of patients with treatment emergent adverse events.
24 weeks
Secondary Outcomes (1)
Change from baseline in subretinal and intraretinal fluid as measured by OCT
24 weeks
Study Arms (1)
Subretinally Injected RetinoStat
EXPERIMENTALSubretinally injected RetinoStat
Interventions
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Oxford BioMedicalead
Study Sites (3)
The University of Iowa
Iowa City, Iowa, 52242-1098, United States
Johns Hopkins University Hospital
Baltimore, Maryland, 21287-9277, United States
Dr Andreas Lauer - Oregon Health & Science University
Portland, Oregon, 97239, United States
Related Publications (1)
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.
PMID: 27710144RESULT
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Peter A Campochiaro, MD
Johns Hopkins University Hospital
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 21, 2011
First Posted
February 23, 2011
Study Start
February 1, 2011
Primary Completion
September 1, 2014
Study Completion
May 1, 2015
Last Updated
April 5, 2017
Record last verified: 2017-04