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Dry Age-related Macular DegenerationPhase 2EXPLORENCT04437368

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

A Phase 2 study testing GT005 in people with Dry Age-related Macular Degeneration, run by Gyroscope Therapeutics Limited at 55 sites in 8 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 55 years and older
Conditions the study is for
Dry Age-related Macular Degeneration
What is being tested?
GT005
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
98 participants
Where
55 sitesin 8 countries
Study dates
26 Apr 2019to 5 Apr 2024
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Able and willing to give written informed consent
  • Age ≥55 years
  • Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, and a diagnosis of AMD in the contralateral eye (except if the subject is monocular)
  • Have GA lesion(s) total size between or equal to 1.25mm2 to 17.5mm2 in the study eye
  • The GA lesion(s) 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, defined as either:
Show all 12Show fewer
  • Non-exudative/sub-clinical fellow eye CNV identified at Screening, or
  • Known history of fellow eye CNV with either ≥2 years since diagnosis or with no active treatment required in 6 months prior to Screening
  • Have a BCVA of 24 letters (6/95 and 20/320 Snellen acuity equivalent) or better, using ETDRS charts, in the study eye
  • Part 1 Only: Subjects carrying a CFI rare variant genotype (minor allele frequency of ≤1%) previously associated with low serum CFI or subjects carrying an unreported CFI rare variant genotype that have tested to have a low serum CFI
  • 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. A pregnancy test is not required for postmenopausal women (defined as being at least 12 consecutive months without menses) or those surgically sterilised (those having a bilateral tubal ligation/bilateral salpingectomy, bilateral tubal occlusive procedure, hysterectomy, or bilateral oophorectomy)

Who cannot join

  • Subjects who have a clinical diagnosis of Stargardt Disease or other retinal dystrophies, confirmed by the central reading centre
  • 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 Screening (Visit 1). Yttrium aluminium garnet capsulotomy is permitted if performed >10 weeks prior to Visit 1
  • Have clinically significant cataract that may require surgery during the study period in the study eye
Show all 14Show fewer
  • Presence of moderate to severe glaucomatous optic neuropathy in the study eye; uncontrolled IOP despite the use of two or more topical agents; a history of glaucoma-filtering or valve surgery is also excluded
  • Axial myopia of greater than -8 dioptres in the study eye
  • 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
  • Have a contraindication to specified protocol corticosteroid regimen
  • Have received any investigational and/or approved product(s) 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 in the study eye or systemically
  • Have received a gene or cell therapy at any time
  • 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

Trial locations and contacts

55 sites in 8 countries.

55 sites in 8 countries

United States32 sites

  • Vision Research Center Eye Associates of New Mexico

    Albuquerque, New Mexico, 87109

  • Southeast Retina Center

    Augusta, Georgia, 30909

  • Austin Research Center for Retina, PLLC

    Austin, Texas, 78705

  • The Retina Care Center

    Baltimore, Maryland, 21209

  • Retina Consultants of Houston-TMC

    Bellaire, Texas, 77401

  • Ophthalmic Consultants of Boston (OCB)

    Boston, Massachusetts, 02114

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Texas Retina Associates

    Dallas, Texas, 75231

  • Erie Retinal Surgery, INC

    Erie, Pennsylvania, 16507

  • Oregon Retina

    Eugene, Oregon, 97401

  • VitreoRetinal Associates, P.A.

    Gainesville, Florida, 32607

  • Retina Associates of Southern California

    Huntington Beach, California, 92647

  • Midwest Eye Institute Northside

    Indianapolis, Indiana, 46290

  • Southeastern Retina Associates, PC

    Knoxville, Tennessee, 37922

  • University Retina Macula Associates PC

    Lemont, Illinois, 60439

  • Charles Retina Institute

    Memphis, Tennessee, 38138

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136

  • VitreoRetinal Surgery, PLLC

    Minneapolis, Minnesota, 55435

  • West Virginia University

    Morgantown, West Virginia, 26506

  • Columbia University Medical Center

    New York, New York, 10032

  • Byers Eye Institute at Stanford

    Palo Alto, California, 94303

  • Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19107

  • Retinal Research Institute (retina consultants of AZ)

    Phoenix, Arizona, 85053

  • Casey Eye Institute

    Portland, Oregon, 97239

  • Retina Consultants San Diego

    Poway, California, 92064

  • Sierra Eye Associates

    Reno, Nevada, 89502

  • Retina Associates of Western New York

    Rochester, New York, 14620

  • Retinal Consultants of San Antonio

    San Antonio, Texas, 78240

  • Department of Ophthalmology UW Medicine

    Seattle, Washington, 98104-2499

  • Retina Vitreous Associates of Florida

    St. Petersburg, Florida, 33711

  • Wolfe Eye Clinic

    West Des Moines, Iowa, 50266

Other trials in United States

United Kingdom6 sites

  • Bristol Eye Hospital

    Bristol, BS1 2LX

  • St.Paul's Eye Unit

    Liverpool, L7 8XP

  • Moorfields Eye Hospital - NHS Foundation Trust

    London, EC1V 2PD

  • The Retina Clinic London

    London, W1G 7LB

  • Sheffield Teaching Hospitals NHS Foundation Trust

    Sheffield, S10 2SB

  • Sunderland Eye Infirmary

    Sunderland, SR2 9HP

Other trials in United Kingdom

Germany5 sites

  • Universitaetsklinikum Bonn

    Bonn, 53127

  • Internationale Innovative Ophthalmochirurgie

    Düsseldorf, 40549

  • Universitaetsklinikum Schleswig-Holstein Campus Lübeck

    Lübeck, Schleswig-Holstein, 23538

  • St. Franziskus-Hospital

    Münster, 48145

  • Universitatsklinikum Tübingen

    Tübingen, 72076

Other trials in Germany

Spain5 sites

  • Hospital La Arruzafa

    Córdoba, 14012

  • Clinica Baviera

    Madrid, 28046

  • Clinica Universidad de Navarra - Pamplona

    Pamplona, Navarre, 31008

  • Hospital Universitari General de Catalunya

    Sant Cugat del Vallès, Barcelona, 08195

  • Clinica Oftalvist Valencia

    Valencia, 46100

Other trials in Spain

France3 sites

  • CHU Hôpital F. Mitterrand

    Dijon, Bourgogne-Franche-Comté, 21079

  • Centre Paradis Monticelli

    Marseille, Alpes-Cote d'Azur, 13008

  • CHU de Nantes - Hôtel-Dieu

    Nantes, Pays de la Loire Region, 44000

Other trials in France

Australia2 sites

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

    Melbourne E., Victoria

  • Sydney Hospital and Sydney Eye Hospital

    Sydney, 2000

Other trials in Australia

Netherlands1 site

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA

Other trials in Netherlands

Poland1 site

  • Oftalmika Spolka z ograniczona odpowiedzialnoscia

    Bydgoszcz, 85-631

Other trials in Poland

About this study

Official title: EXPLORE: A Phase II, Outcomes Assessor-masked, Multicentre, Randomised Study 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

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).

Read the detailed description

This was a Phase II, outcomes assessor-masked, multicentre, randomized study to assess the safety and efficacy of two doses of GT005 administered as a single-time subretinal injection in subjects with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). Approximately 202 subjects were planned to be randomized to GT005 or the untreated control group.

Subjects entered the study had genotyping and serum complement factor I(CFI) levels assessed. Assessments were either performed at a sponsor-approved laboratory during the EXPLORE screening period or provided through participation in a previous Gyroscope sponsored study. If subjects failed to meet the eligibility criteria for EXPLORE, they were classified as screen failures for this study and could be considered for entry into another Novartis/Gyroscope sponsored study.

After providing the informed consent, subjects underwent ophthalmic and clinical assessments to determine eligibility for inclusion in the study.

Upon confirmation of eligibility, subjects in part 1 were randomized to one of two groups: low dose [2E10 vg], or high dose [2E11 vg]. Within each group, subjects were allocated to GT005 or untreated control based on a 2:1 ratio. Once part 1 enrolment was completed, and the last active subject completed screening and either was screen-failed or randomized, then Part 2 could commence. In part 2, subjects were randomized to the low dose [2E10] group or untreated control based on a 2:1 ratio. The study eye was identified for all subjects.

Subjects were stratified by GA lesion size on fundus autofluorescence (FAF) (≤10 mm2 or >10 mm2) and presence of choroidal neovascularisation (CNV) in the fellow eye (Yes or No). Randomization of study eyes in the GA lesion size upper stratum of >10 mm2 to 17.5 mm2 was capped at 20% of total subjects randomized. Once enrolment capping at 20% based on the upper GA lesion size was reached, eyes that fulfilled the cap criteria were no longer eligible, unless the subject had a CFI rare variant genotype (minor allele frequency ≤1%) previously associated with normal or low serum CFI or had an unreported CFI rare variant genotype. Of all subjects enrolled and randomized in the study, the presence of CNV in the fellow eye was capped at 25% per stratum. A permuted-block randomization method was used to obtain an approximately 2:1 ratio between GT005 and the untreated control groups for each dose group within each stratum.

Following randomization, the investigator was informed of the subject's allocated treatment (GT005 or the untreated control group) and the study eye selected. To minimize bias, all imaging endpoint assessments and grading were performed at a Central Reading Centre (CRC), in a masked fashion. For part 1, the Sponsor, subject, investigators, and study personnel performing clinical assessments remained masked to dose received for those allocated to GT005. For part 2, the Sponsor, investigators, subjects, and study personnel performing clinical assessments were unmasked to dose received, since only the low dose was administered.

Subjects randomized to GT005 underwent a single time subretinal administration of the study drug. Vitreous samples were collected during surgery. Following surgery, a prophylactic steroid regimen was prescribed.

The study consisted of a screening period lasting up to 8 weeks (or up to 12 weeks if agreed by the Sponsor Medical Monitor), followed by a 96-week study period. All subjects were assessed for the occurrence of adverse events (AEs) at each visit and underwent functional visual and retinal imaging,anatomical assessments, and biological sampling as per the schedule of assessments.

This study was conducted in compliance with Independent ethics committees (IECs) / Institutional review board (IRBs), informed consent regulations, the Declaration of Helsinki, International Council on Harmonisation (ICH) Good Clinical Practices (GCP) Guidelines, and the Food and Drug Administration (FDA) guidance.

On 24-Aug-2023, the decision was taken to terminate the study and the GT005 program. The decision was aligned with the recommendation of an independent DMC, which concluded that futility criteria had been met for the HORIZON study (GT005-03) and the overall benefit-risk ratio did not support continuation of the current development program as planned. All GT005- treated subjects, who were willing to be transferred into the long-term safety follow-up study were enrolled in the ORACLE (CPPY988A12203B / NCT05481827) study.

In both Part 1 and Part 2, patients with geographic atrophy secondary to age-related macular degeneration were enrolled. In Part 1, patients with CFI rare variant genotype and low serum CFI level and in Part 2, patients were enrolled regardless of genotypes. Efficacy results were analyzed by arm and also by part. AE and disposition data were reported per arm, but the parts were combined, according to the analysis plan.

Part 1 enrolled subjects with CFI rare variant; Part 2 enrolled subjects regardless of genotype.

There were no subjects in the high dose arm in Part 2.

Being tested
GT005
Keywords
Geographic atrophy, Retinal disease, Eye disease, Retinal degeneration, Macular atrophy, Dry age-related macular degeneration
Medical subject headings
Geographic Atrophy, Retinal Diseases, Eye Diseases, Retinal Degeneration, Anetoderma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GT005Drug

    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.

Study groups in NCT04437368
Study groupRoleWhat they receive
GT005 Low dose [2E10 vg]ExperimentalGT005 Low dose [2E10 vg] (Parts 1 and 2)
Receives: Drug: GT005
GT005 High dose [2E11 vg]ExperimentalGT005 High dose [2E11 vg] (Part 1)
Receives: Drug: GT005
Untreated controlNo interventionUntreated control (Parts 1 and 2)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1

    The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)

    Measured: Baseline, Weeks 12, 24, 36, and 48

Secondary outcomes (15)

  1. The Change From Baseline in Geographic Atrophy (GA) at Week 72 and Week 96 - Part 1

    The change from baseline to Week 48 in GA area as measured by fundus autofluorescence (FAF)

    Measured: Baseline, Weeks 72 and 96

  2. Summary of Adverse Events - Parts 1 and 2 Combined

    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject.

    A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs.

    Measured: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

  3. Ocular Adverse Events by Primary System Organ Class and Preferred Term for the Study Eye - Parts 1 and 2 Combined

    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject.

    A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs.

    System organ classes are sorted alphabetically, and preferred terms are sorted by decreasing overall frequency within system organ class.

    Measured: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

  4. Non-ocular Adverse Events - Summary - Parts 1 and 2 Combined

    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject.

    A TEAE is defined as any AE that develops after randomization or any AE already present that worsens following randomization. The primary summaries of AEs are based on TEAEs.

    Measured: Adverse events are reported from randomization up to end of study, for a maximum timeframe of approximately 96 weeks.

  5. Change in GA Morphology From Baseline to Week 96 on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence (FAF) - Part 1

    Change in GA morphology on multimodal imaging through Week 96.

    FAF images at Week 5 were introduced via a protocol amendment after most participants had already completed Week 5; therefore, only a minimal number of patients had FAF images taken at Week 5 for Part 1.

    Measured: Baseline, Weeks 5,12,24,36,48,72,96

  6. Change From Baseline to Week 48 in GA Morphology on Colour Fundus Photography (CFP) - Number of Participants With Increase in Fundus Autofluorescence - Part 2

    Change in GA morphology on multimodal imaging through Week 48.

    For the untreated control group, there were no protocol-specified visits for Weeks 5 and 8.

    The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

    Measured: Baseline, Weeks 5,12,24,36,48

  7. Change in Best Corrected Visual Acuity (BCVA) Score From Baseline Through Week 96 Via the Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1

    BCVA was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts.

    Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.

    For the untreated control group, there were no protocol-specified visits for Weeks 1, 5 and 8.

    Measured: Baseline, Weeks 1, 5, 8, 12, 24, 36, 48, 72 and 96

  8. Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, 48, 72 and 96, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 1

    LLD was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts.

    The test was to be performed after BCVA testing, prior to pupil dilation, and distance refraction was to be carried out before Low Luminance Visual Acuity (LLVA) was measured.

    LLVA was to be measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. The LLD was calculated as the difference between BCVA and LLVA.

    Initially, letters were to be read at a distance of 4 metres from the chart. If <20 letters were read at 4 metres, testing at 1 metre should have been performed. LLD was to be reported as number of letters read correctly by the subject.

    Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.

    Measured: Baseline, Weeks 12, 24, 36, 48, 72 and 96

  9. Change in Low Luminance Difference (LLD) Letter Count From Baseline at Weeks 12, 24, 36, and 48, Via Early Treatment for Diabetic Retinopathy (ETDRS) Chart - Part 2

    LLD was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts.

    The test was to be performed after BCVA testing, prior to pupil dilation, and distance refraction was to be carried out before Low Luminance Visual Acuity (LLVA) was measured.

    LLVA was to be measured by placing a 2.0-log-unit neutral density filter over the front of each eye and having the subject read the normally illuminated ETDRS chart. The LLD was calculated as the difference between BCVA and LLVA.

    Initially, letters were to be read at a distance of 4 metres from the chart. If <20 letters were read at 4 metres, testing at 1 metre should have been performed. LLD was to be reported as number of letters read correctly by the subject.

    Min and max possible scores are 0-100 respectively. A higher score represents better visual functioning.

    Measured: Baseline, Weeks 12, 24, 36, and 48

  10. Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 1

    The maximum reading speed (MRS) represents the highest reading speed an individual can achieve when print size is not a limiting factor. Essentially, it measures how quickly a person can read text when the print is large enough to be easily readable.

    A higher count represents better visual functioning.

    Measured: Baseline, Weeks 24, 36, 48, 72 and 96

  11. Change From Baseline at Weeks 24, 36 and 48 in Reading Performance, Measured as the Maximum Reading Speed (Words Per Minute), as Assessed by Minnesota Low-vision Reading Test (MNRead) Chart - Part 2

    A higher count represents better visual functioning.

    The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

    Measured: Baseline, Weeks 24, 36 and 48

  12. Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Functional Reading Independence (FRI) Index - Part 1

    The FRI index is a patient-reported outcome measure developed specifically for use in GA patients. The FRI index evaluates the level of independence subjects have in performing everyday activities that require reading, such as writing a cheque or reading a prescription.

    Scores derived from the index range from 1 (unable to do) to 4 (total independence).

    A higher score represents better visual functioning.

    Measured: Baseline, Weeks 24, 36, 48, 72 and 96

  13. Change From Baseline at Weeks 24, 36 and 48 in Functional Reading Independence (FRI) Index - Part 2

    The FRI index is a patient-reported outcome measure developed specifically for use in GA patients. The FRI index evaluates the level of independence subjects have in performing everyday activities that require reading, such as writing a cheque or reading a prescription.

    Scores derived from the index range from 1 (unable to do) to 4 (total independence).

    A higher score represents better visual functioning.

    The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

    Measured: Baseline, Weeks 24, 36 and 48

  14. Change From Baseline at Weeks 24, 36, 48, 72 and 96 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 1

    The National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) measures the influence of visual disability and visual symptoms on general health domains.

    The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better visual functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A composite score is derived based on the average of the 11 subscales.

    Measured: Baseline, Weeks 24, 36, 48, 72 and 96

  15. Change From Baseline at Weeks 24, 36 and 48 in Patient Reported Outcomes (Visual Function Questionnaire-25) - Composite Score - Part 2

    The National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) measures the influence of visual disability and visual symptoms on general health domains.

    The NEI VFQ-25 consists of a base set of 25 vision-targeted questions representing 11 vision-related constructs, plus an additional single-item general health rating question. All items are scored so that a high score represents better visual functioning. Each item is then converted to a 0 to 100 scale so that the lowest and highest possible scores are set at 0 and 100 points, respectively. A composite score is derived based on the average of the 11 subscales.

    The untreated control participants were immediately discontinued from the study when the study was early terminated; therefore Week 36 and 48 data in part 2 were not applicable for these the untreated control group.

    Measured: Baseline, Weeks 24, 36, and 48

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jun 2020
  2. Study start26 Apr 2019
  3. Primary completion5 Apr 2024
  4. Study completion5 Apr 2024

Why it stopped: Terminated for interim analysis demonstrating futility (trial highly unlikely to meet efficacy outcome). The trial is not ending early because of medical problems or concerns.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This is a Phase 2, outcomes assessor-masked multicentre, randomised 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.
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
98 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04437368
Sponsor's protocol ID
GT005-02
Other IDs
CPPY988A12202, 2019-003421-22, 2019
Acronym
EXPLORE
Status last verified
Jan 2026
First submitted
3 Jun 2020
Last updated
27 Jan 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 37314061

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GT005 in people with Dry Age-related Macular Degeneration, run by Gyroscope Therapeutics Limited at 55 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 27 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 55 sites in United States, United Kingdom, Germany, Spain, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1 (time frame: Baseline, Weeks 12, 24, 36, and 48). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04437368, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jan 2026.

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