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

Age Related Macular Degeneration Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01301443
• Other study ID #: RS1/001/10
• Status: Completed
• Phase: Phase 1
• First received: February 21, 2011
• Last updated: April 3, 2017
• Start date: February 2011
• Est. completion date: May 2015

Study information

• Verified date: April 2017
• Source: Oxford BioMedica
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: May 2015
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Age Related Macular Degeneration
• Macular Degeneration

Intervention

Drug:
• Subretinally injected RetinoStat
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.

Locations

Country	Name	City	State
United States	Johns Hopkins University Hospital	Baltimore	Maryland
United States	The University of Iowa	Iowa City	Iowa
United States	Dr Andreas Lauer - Oregon Health & Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Oxford BioMedica

Country where clinical trial is conducted

United States, 

References & 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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The incidence of adverse events	The number and percentage of patients with treatment emergent adverse events.	24 weeks
Secondary	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	24 weeks