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

NCT ID: NCT01301443

Last Updated: 2017-04-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-02-28
• Study Completion Date: 2015-05-31

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.

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.

Conditions

Age Related Macular Degeneration

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Subretinally Injected RetinoStat

Subretinally injected RetinoStat

Group Type: EXPERIMENTAL

Subretinally injected RetinoStat

Intervention Type: DRUG

Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.

Interventions

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.

Intervention Type: DRUG

Other Intervention Names

OXB-201

Eligibility 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.

• Minimum Eligible Age: 50 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Oxford BioMedica

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Peter A Campochiaro, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University Hospital

Locations

The University of Iowa

Iowa City, Iowa, United States

Johns Hopkins University Hospital

Baltimore, Maryland, United States

Dr Andreas Lauer - Oregon Health & Science University

Portland, Oregon, United States

Countries

United States

References

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.

Reference Type: RESULT

PMID: 27710144 (View on PubMed)

Other Identifiers

RS1/001/10

Identifier Type: -

Identifier Source: org_study_id