GT005 for Dry Age-Related Macular Degeneration

Recruiting at 14 trial locations

Overseen By Gyroscope Therapeutics Limited

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Gyroscope Therapeutics Limited

Must not be taking: Investigational products, Gene therapy

Disqualifiers: Choroidal neovascularisation, Diabetic retinopathy, Glaucoma, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

56 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tested an eye injection treatment called GT005 for people with vision loss from Macular Atrophy due to AMD. The goal was to improve or maintain their vision. The trial was stopped because the treatment was not effective, but it was not due to safety issues.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What data supports the effectiveness of the treatment GT005 for dry age-related macular degeneration?

The research mentions that gene therapy is being explored as a novel treatment for dry age-related macular degeneration, with some promising results in early clinical trials. GT005, being a gene therapy, may benefit from these findings as it targets similar pathways to slow disease progression. ^{1 2 3 4 5}

How is the treatment GT005 different from other treatments for dry age-related macular degeneration?

GT005 is a gene therapy, which is a novel approach for treating dry age-related macular degeneration. Unlike traditional treatments, it aims to address the underlying genetic causes of the disease, potentially offering a more targeted and long-lasting effect. ^{1 3 5 6 7}

Research Team

Chief Medical Officer

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

This trial is for adults aged 55+ with Dry Age-Related Macular Degeneration (AMD) causing macular atrophy. Participants must have specific sizes of GA lesions in their eyes and meet certain vision acuity requirements. Women capable of childbearing need a negative pregnancy test, and all participants should be able to attend study visits.

Inclusion Criteria

I have been diagnosed with GA due to AMD in one eye and AMD in the other.

I am not pregnant and have taken a test in the last two weeks to confirm this, or I cannot become pregnant due to menopause or surgery.

My eye condition is fully visible in a specific eye test image.

Exclusion Criteria

I have not had eye surgery in the last 12 weeks, except for a specific laser procedure over 10 weeks ago.

Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant

Have a contraindication to the specified protocol corticosteroid regimen

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single subretinal injection of GT005

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

48 weeks

Regular visits (in-person)

Long-term follow-up

Participants have the option to be followed for a further 4 years to assess long-term safety and efficacy

192 weeks

Treatment Details

Interventions

• GT005

Trial Overview The FOCUS trial is testing GT005, delivered using the Orbit Subretinal Delivery System, on people with Dry AMD to see if it's safe and effective in treating macular atrophy. It's an open-label Phase I/II multicentre study where everyone receives the treatment.

Participant Groups

Group I: GT005 Dose 3 with Orbit Subretinal Delivery System Experimental Treatment 2 Interventions

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group II: GT005 Dose 3 with Orbit Subretinal Delivery Sysem Experimental Treatment 1 Intervention

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group III: GT005 Dose 3 Experimental Treatment 1 Intervention

A single dose of GT005 will be administered via subretinal injection

Group IV: GT005 Dose 2 with Orbit Subretinal Delivery System Experimental Treatment 2 Interventions

A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach

Group V: GT005 Dose 2 Experimental Treatment 1 Intervention

A single dose of GT005 will be administered via subretinal injection

Group VI: GT005 Dose 1, 2 or 3 Experimental Treatment 1 Intervention

A single dose of GT005 will be administered via subretinal injection. This dose will be determined by dose levels determined to be tolerable in Arms 1,2 and 3

Group VII: GT005 Dose 1 Experimental Treatment 1 Intervention

A single dose of GT005 will be administered via subretinal injection

Who Is Running the Clinical Trial?

Gyroscope Therapeutics Limited

Lead Sponsor

Trials

5

Recruited

720+

Novartis Pharmaceuticals

Industry Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Findings from Research

In a study of 593,277 patients with late dry age-related macular degeneration (dAMD), it was found that the majority had mild disease (64.4%) or intermediate disease (29.4%), with visual acuity (VA) decreasing as the disease progressed.

The annual probability of stable dAMD was high (82.1% for GA without subfoveal involvement to 92.3% for GA with subfoveal involvement), but the risk of progression to geographic atrophy or neovascular AMD increased significantly with disease severity, highlighting the need for effective treatments as patients advance in disease stage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dry Age-Related Macular Degeneration: Distribution of Visual Acuity and Progression Risk in a Large Registry. Leng, T., Schwartz, J., Nimke, D., et al.[2023]

In a study of 130 eyes from 115 patients with age-related macular degeneration, those who received annual intravitreal anti-vascular endothelial growth factor injections maintained stable visual acuity over an average follow-up of 11.1 years.

Patients who did not receive at least one injection each year experienced a significant decline in visual acuity, highlighting the importance of continuous treatment for preserving vision in this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

TEN-YEAR FOLLOW-UP OF PATIENTS WITH EXUDATIVE AGE-RELATED MACULAR DEGENERATION TREATED WITH INTRAVITREAL ANTI-VASCULAR ENDOTHELIAL GROWTH FACTOR INJECTIONS. Starr, MR., Kung, FF., Mejia, CA., et al.[2021]

Only about 0.1% of the Polish population requires treatment for neovascular age-related macular degeneration (nAMD) when considering patients without severe irreversible eye damage, indicating a relatively low prevalence of this condition needing intervention.

The COVID-19 pandemic significantly impacted the enrollment in the national AMD therapeutic program, with a notable decrease in monthly enrollments, highlighting potential barriers to treatment access during health crises.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Need for Treatment of Neovascular Age-Related Macular Degeneration: A Study Based on the Polish National Registry. Teper, SJ., Nowińska, A., Figurska, M., et al.[2022]

Antibody-based therapies have significantly improved the treatment of wet age-related macular degeneration (AMD), with innovative approaches like port delivery systems and bispecific antibodies showing promise in clinical trials.

While stem cell and gene therapies are still under investigation for both wet and dry AMD, they present potential benefits and risks that need further evaluation before clinical use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gene, Cell and Antibody-Based Therapies for the Treatment of Age-Related Macular Degeneration. Akyol, E., Lotery, A.[2020]

References

1. England pubmed.ncbi.nlm.nih.gov

Dry Age-Related Macular Degeneration: Distribution of Visual Acuity and Progression Risk in a Large Registry. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

TEN-YEAR FOLLOW-UP OF PATIENTS WITH EXUDATIVE AGE-RELATED MACULAR DEGENERATION TREATED WITH INTRAVITREAL ANTI-VASCULAR ENDOTHELIAL GROWTH FACTOR INJECTIONS. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Treatments for dry age-related macular degeneration: therapeutic avenues, clinical trials and future directions. [2022]

4. England pubmed.ncbi.nlm.nih.gov

The Need for Treatment of Neovascular Age-Related Macular Degeneration: A Study Based on the Polish National Registry. [2022]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Gene, Cell and Antibody-Based Therapies for the Treatment of Age-Related Macular Degeneration. [2020]

6. England pubmed.ncbi.nlm.nih.gov

Long-Term Outcomes in Patients with Neovascular Age-Related Macular Degeneration Who Maintain Dry Macula after Three Monthly Ranibizumab Injections. [2018]

7. United States pubmed.ncbi.nlm.nih.gov

Four-year incidence and progression of age-related macular degeneration: the Los Angeles Latino Eye Study. [2021]