HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005

NCT04566445 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: GT005-03CPPY988A122012020-002431-30
• Study Type: interventional
• Target: 255
• Locations: 7 countries
• Sites: 62

Brief Summary

The purpose of this clinical study was to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with geographic atrophy secondary to age-related macular degeneration (AMD).

Conditions

Dry Age-related Macular Degeneration

Keywords

Dry age-related macular degeneration; Geographic atrophy; Retinal disease; Eye disease; Retinal degeneration; Macular atrophy

Outcome Measures

Primary Outcomes (1)

• The Change From Baseline to Week 72 in Geographic Atrophy (GA)

  GA area as measured by fundus autofluorescence (FAF)

  Baseline, Weeks 12, 24, 36, 48 and 72

Secondary Outcomes (10)

• The Change From Baseline at Week 96 in Geographic Atrophy (GA)

  Baseline, Week 96

• Summary of Adverse Events

  Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.

• Ocular AEs Occurring in ≥2% of Subjects by Primary System Organ Class and Preferred Term for the Study Eye

  Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.

• Non-ocular AEs Occurring in ≥2% of Subjects

  Adverse events are reported from randomization to the end of study, at Week 96, up to a maximum timeframe of approximately 96 weeks.

• Change in GA Morphology From Baseline to Week 96 on Multimodal Imaging - Number of Participants With Increase in Fundus Autofluorescence

  Baseline, Weeks 5, 12, 24, 36, 48, 72 and 96

Study Arms (3)

GT005 Medium dose [5E10 vg]

EXPERIMENTAL

GT005 Medium dose \[5E10 vg\]

Drug: GT005

GT005 High dose [2E11 vg]

EXPERIMENTAL

GT005 High dose \[2E11 vg\]

Drug: GT005

Untreated control

NO INTERVENTION

Untreated control

Interventions

GT005 DRUG

GT005 is a recombinant, non-replicating AAV2 expressing human complement factor I (CFI). GT005 was administered as a single time subretinal injection into the study eye of subjects allocated to one of the two GT005 doses.

GT005 High dose [2E11 vg]; GT005 Medium dose [5E10 vg]

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able and willing to give written informed consent
• Age ≥55 years
• GA lesion(s) within an acceptable size on FAF, in the study eye
• The GA lesion in the study eye must reside completely within the FAF image
• Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye
• Have a BCVA of ≥24 letters (6/95 or 20/320 Snellen acuity equivalent), using ETDRS charts, in the study eye
• a. In Stage 1: Meet one of the pre-specified AMD genetic subgroup criteria; b. In Stage 2: Genotyping is not required for study eligibility
• Able to attend all study visits and complete the study procedures
• Women of child-bearing potential must have a negative pregnancy test within 2 weeks prior to randomisation (not required for postmenopausal women) or provide documentation of being surgically sterilised

You may not qualify if:

• a. In Stage 1: Carriers of excluded genetic variants; b. In Stage 2: Subjects are excluded if they have a clinical diagnosis of Stargardt Disease or other retinal dystrophies
• Have a history, or evidence, of CNV in the study eye
• Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in the study eye
• Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye
• History of intraocular surgery in the study eye within 12 weeks prior to Visit 1
• Have clinically significant cataract that may require surgery during the study period in the study eye
• Presence of moderate to severe glaucomatous optic neuropathy, uncontrolled intraocular pressure (IOP), despite use of two or more topical agents; or a history of glaucoma-filtering or valve surgery
• Axial myopia of greater than -8 diopters in the study eye
• Have received any investigational product for the treatment of GA within the past 6 months or 5 half-lives (whichever is longer), other than nutritional supplements such as the age-related eye disease study (AREDS) formula
• Have received a gene or cell therapy at any time.
• Have a contraindication to the protocol specified corticosteroid regimen
• Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
• Active malignancy within the past 12 months, except for appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with a stable prostate-specific antigen (PSA) ≥ 12 months
• Have any other significant ocular or non-ocular medical or psychiatric condition which, in the opinion of the Investigator, may either put the subject at risk or may influence the results of the study

Sponsors & Collaborators

• Gyroscope Therapeutics Limited: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (62)

Retinal Research Institute (retina consultants of AZ)

Phoenix, Arizona, 85053, United States

Location

Retina Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

Retina Consultants of Orange County

Fullerton, California, 92835, United States

Location

Northern California Retina Vitreous Associates

Mountain View, California, 94040, United States

Location

Byers Eye Institute at Stanford

Palo Alto, California, 94303, United States

Location

Retina Consultants of San Diego

Poway, California, 92064, United States

Location

University of California (UC) Davis Medical Group Eye Center

Sacramento, California, 95817, United States

Location

Southwest Retina Research Center

Durango, Colorado, 81303, United States

Location

Rand Eye Institute

Deerfield Beach, Florida, 33064, United States

Location

VitreoRetinal Associates, P.A.

Gainesville, Florida, 32607, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Retina Vitreous Associates of Florida

St. Petersburg, Florida, 33711, United States

Location

Southeast Retina Center

Augusta, Georgia, 30909, United States

Location

Georgia Retina PC

Marietta, Georgia, 30060, United States

Location

Illinois Retina Associates

Chicago, Illinois, 60657, United States

Location

University Retina Macula Associates PC

Lemont, Illinois, 60452, United States

Location

Midwest Eye Institute Northside

Indianapolis, Indiana, 46290, United States

Location

Wolfe Eye Clinic

West Des Moines, Iowa, 50266, United States

Location

Retina Associates, LLC

Shawnee Mission, Kansas, 66204, United States

Location

The Retina Care Center

Baltimore, Maryland, 21209, United States

Location

Ophthalamic Consultants of Boston (OCB)

Boston, Massachusetts, 02114, United States

Location

Retina Associates of Michigan

Grand Blanc, Michigan, 48439, United States

Location

Associated Retinal Consultants PC

Royal Oak, Michigan, 48073, United States

Location

Sierra Eye Associates

Reno, Nevada, 89502, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Retina Associates of Western New York

Rochester, New York, 14620, United States

Location

Duke Eye Center

Durham, North Carolina, 27705, United States

Location

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Retina

Eugene, Oregon, 97401, United States

Location

Casey Eye Institute - OHSU

Portland, Oregon, 97239, United States

Location

Erie Retinal Surgery ,INC

Erie, Pennsylvania, 16507, United States

Location

Mid Atlantic Retina - Wills Eye Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Palmetto Retina Center

West Columbia, South Carolina, 29169, United States

Location

Charles Retina Institute

Germantown, Tennessee, 38138, United States

Location

Southeastern Retina Associates, PC

Knoxville, Tennessee, 37922, United States

Location

Austin Research Center for Retina, PLLC

Austin, Texas, 78705, United States

Location

Retina Consultants of Houston-TMC

Bellaire, Texas, 77401, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Texas Retina Associates (Dallas)

Dallas, Texas, 75231, United States

Location

Retinal Consultants of San Antonio

San Antonio, Texas, 78240, United States

Location

Retina Consultants of Houston

The Woodlands, Texas, 77384, United States

Location

Rocky Mountain Retina Consultants

Salt Lake City, Utah, 84107, United States

Location

Department of Ophthalmology UW Medicine

Seattle, Washington, 98104-2499, United States

Location

Retina Center Northwest

Silverdale, Washington, 98383, United States

Location

Sydney Hospital and Sydney Eye Hospital

Sydney, New South Wales, 2000, Australia

Location

The University of Melbourne - The Centre for Eye Research Australia (CERA)

Melbourne E., Victoria, 3002, Australia

Location

CHU Dijon - Hopital Mitterrand

Dijon, 21000, France

Location

Centre Paradis Monticelli

Marseille, 13008, France

Location

CHU de Nantes - Hôtel-Dieu

Nantes, 44093, France

Location

Universitätsklinikum Bonn

Bonn, 53127, Germany

Location

Internationale Innovative Ophthalmochirurgie

Düsseldorf, 40549, Germany

Location

Universitaetsklinikum Schleswig-Holstein - Campus Lübeck

Lübeck, 23562, Germany

Location

Universitaetsklinikum Tuebingen

Tübingen, 72076, Germany

Location

Oftalmika Spolka z ograniczona odpowiedzialnoscia

Bydgoszcz, 85-631, Poland

Location

Instituto de microcirugía ocular

Barcelona, 08035, Spain

Location

Clinica Baviera

Madrid, 28046, Spain

Location

Clinica Universidad de Navarra - Pamplona

Pamplona, 31008, Spain

Location

Moorfields Eye Hospital - NHS Foundation Trust

London, EC1V 2PD, United Kingdom

Location

Retina Clinic London

London, W1G 7LB, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Sunderland Eye Infirmary

Sunderland, SR2 9HP, United Kingdom

Location

Related Publications (1)

• Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.

  PMID: 37314061 DERIVED

Related Links

• Novartis Clinical Trial Results

MeSH Terms

Conditions

Geographic Atrophy; Retinal Diseases; Eye Diseases; Retinal Degeneration; Anetoderma

Condition Hierarchy (Ancestors)

Macular Degeneration; Eye Diseases, Hereditary; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Abnormalities; Skin Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@Novartis.com

+ 1 862 778 8300

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Quadruple (Participant, Care Provider, Investigator, Outcome Assessor). The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a Phase II, open-label, outcomes-assessor masked, multicentre, randomised, controlled study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal injection in subjects with GA secondary to AMD.

Study Record Dates

• First Submitted: September 9, 2020
• First Posted: September 28, 2020
• Study Start: September 28, 2020
• Primary Completion: June 10, 2024
• Study Completion: June 10, 2024
• Last Updated: January 28, 2026
• Results First Posted: July 10, 2025

Record last verified: 2026-01

Locations

DE (4); ES (3); GB (4); PL (1); AU (2); US (45); FR (3)