HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
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|ClinicalTrials.gov Identifier: NCT04566445|
Recruitment Status : Active, not recruiting
First Posted : September 28, 2020
Last Update Posted : December 16, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Dry Age-related Macular Degeneration||Drug: GT005: Medium Dose Drug: GT005: High Dose||Phase 2|
This is a Phase 2, 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.
The trial includes a screening period of up to 8 weeks and a 96-week study period.
Subjects will be randomised to one of two groups; GT005 or the untreated control group.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||255 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||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.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||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.|
|Official Title:||HORIZON: 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 Geographic Atrophy Secondary to Dry Age-related Macular Degeneration|
|Actual Study Start Date :||September 28, 2020|
|Estimated Primary Completion Date :||September 18, 2024|
|Estimated Study Completion Date :||September 17, 2025|
Experimental: GT005 Medium Dose
Approximately 83 subjects are planned, with subjects randomised to GT005 Medium Dose.
Drug: GT005: Medium Dose
The study will test two doses of GT005: Medium Dose and High Dose.
Experimental: GT005 High Dose
Approximately 83 subjects are planned, with subjects randomised to GT005 High Dose.
Drug: GT005: High Dose
The study will test two doses of GT005: Medium Dose and High Dose.
No Intervention: Untreated control
Approximately 83 subjects are planned, with subjects randomised to untreated control.
- Progression of geographic atrophy [ Time Frame: 72 weeks ]The change from baseline to Week 72 in GA area as measured by fundus autofluorescence (FAF)
- Progression of geographic atrophy [ Time Frame: 96 weeks ]The change from baseline through Week 96 in GA area as measured by fundus autofluorescence (FAF)
- Evaluation of the safety and tolerability of GT005 [ Time Frame: 96 weeks ]Frequency of treatment emergent adverse events (AEs)
- Evaluation of the effect of GT005 on retinal anatomical measures [ Time Frame: 96 weeks ]Change in retinal morphology on multimodal imaging
- Evaluation of the effect of GT005 on functional measures [ Time Frame: 96 weeks ]Change in best corrected visual acuity (BCVA) Score via the early treatment for diabetic retinopathy (ETDRS) chart
- Evaluation of the effect of GT005 on functional measures [ Time Frame: 96 weeks ]Change in low luminance difference (LLD) via the ETDRS chart
- Evaluation of the effect of GT005 on visual function [ Time Frame: 96 weeks ]Change in reading performance as assessed by Minnesota low-vision reading test (MNRead) chart
- Evaluation of the effect of GT005 on visual function [ Time Frame: 96 weeks ]Change in functional reading independence (FRI) index
- Evaluation of the effect of GT005 on patient-reported outcomes [ Time Frame: 96 weeks ]Change in quality of life measured on the Visual Function Questionnaire-25 (VFQ-25)
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||55 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Able and willing to give written informed consent
- Age ≥55 years
- a. In Stage 1: 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; b. In Stage 2: Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, that is non-foveal, as determined by the central reading centre, or has a CFI rare variant genotype and meets inclusion criteria 3a, and a diagnosis of AMD in the contralateral eye (except if monocular)
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04566445
|Responsible Party:||Gyroscope Therapeutics Limited|
|Other Study ID Numbers:||
|First Posted:||September 28, 2020 Key Record Dates|
|Last Update Posted:||December 16, 2022|
|Last Verified:||December 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Dry age-related macular degeneration