This trial is active, not recruiting.

Condition age related macular degeneration
Treatment 3 dose cohorts gene therapy
Phase phase 1
Sponsor Oxford BioMedica
Start date February 2011
End date September 2014
Trial size 21 participants
Trial identifier NCT01301443, RS1/001/10


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Dose escalation of subretinally injected RetinoStat
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.

Primary Outcomes

The incidence of adverse events
time frame: 24 weeks

Secondary Outcomes

Change from baseline in subretinal and intraretinal fluid as measured by OCT
time frame: 24 weeks

Eligibility Criteria

Male or female participants at least 50 years old.

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.

Additional Information

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
Principal investigator Peter A Campochiaro, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2015.
Information provided to ClinicalTrials.gov by Oxford BioMedica.