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

This study is currently recruiting participants.
Verified February 2014 by Oxford BioMedica
Sponsor:
Information provided by (Responsible Party):
Oxford BioMedica
ClinicalTrials.gov Identifier:
NCT01301443
First received: February 21, 2011
Last updated: February 22, 2014
Last verified: February 2014
  Purpose

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 Intervention Phase
Age Related Macular Degeneration
Drug: 3 dose cohorts gene therapy
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Oxford BioMedica:

Primary Outcome Measures:
  • The incidence of adverse events [ Time Frame: 24 weeks ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]
    The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography


Estimated Enrollment: 18
Study Start Date: February 2011
Estimated Study Completion Date: March 2014
Estimated Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Subretinally Injected RetinoStat
Dose escalation of subretinally injected RetinoStat
Drug: 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.

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.

  Eligibility

Ages Eligible for Study:   50 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01301443

Contacts
Contact: Peter A Campochiaro, MD pcampo@jhmi.edu

Locations
United States, Iowa
The University of Iowa Recruiting
Iowa City, Iowa, United States
Contact: Elliot Sohn, MD         
Principal Investigator: Elliott Sohn, MD         
United States, Maryland
Johns Hopkins University Hospital Recruiting
Baltimore, Maryland, United States, 21287-9277
Principal Investigator: Peter Campochiaro, MD         
United States, Oregon
Dr Andreas Lauer Recruiting
Portland, Oregon, United States, 97239
Contact: Andreas K Lauer, MD         
Principal Investigator: Andreas K Lauer, MD         
Sponsors and Collaborators
Oxford BioMedica
Investigators
Principal Investigator: Peter A Campochiaro, MD Johns Hopkins University Hospital
  More Information

No publications provided

Responsible Party: Oxford BioMedica
ClinicalTrials.gov Identifier: NCT01301443     History of Changes
Other Study ID Numbers: RS1/001/10
Study First Received: February 21, 2011
Last Updated: February 22, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Oxford BioMedica:
Age Related Macular Degeneration

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

ClinicalTrials.gov processed this record on April 16, 2014