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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Oxford BioMedica
ClinicalTrials.gov Identifier:
NCT01301443
First received: February 21, 2011
Last updated: May 28, 2014
Last verified: May 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 2015
Estimated Primary Completion Date: September 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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01301443

Locations
United States, Iowa
The University of Iowa
Iowa City, Iowa, United States
United States, Maryland
Johns Hopkins University Hospital
Baltimore, Maryland, United States, 21287-9277
United States, Oregon
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
  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: May 28, 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 July 22, 2014