Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04566445




Registration number
NCT04566445
Ethics application status
Date submitted
9/09/2020
Date registered
28/09/2020
Date last updated
8/07/2024

Titles & IDs
Public title
HORIZON: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
Scientific 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
Secondary ID [1] 0 0
CPPY988A12201
Secondary ID [2] 0 0
GT005-03
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dry Age-related Macular Degeneration 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GT005: Medium Dose
Treatment: Drugs - GT005: High Dose

Experimental: GT005 Medium Dose - Approximately 83 subjects are planned, with subjects randomised to GT005 Medium Dose.

Experimental: GT005 High Dose - Approximately 83 subjects are planned, with subjects randomised to GT005 High Dose.

No intervention: Untreated control - Approximately 83 subjects are planned, with subjects randomised to untreated control.


Treatment: Drugs: GT005: Medium Dose
The study will test two doses of GT005: Medium Dose and High Dose.

Treatment: Drugs: GT005: High Dose
The study will test two doses of GT005: Medium Dose and High Dose.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Progression of geographic atrophy
Timepoint [1] 0 0
72 weeks
Secondary outcome [1] 0 0
Progression of geographic atrophy
Timepoint [1] 0 0
96 weeks
Secondary outcome [2] 0 0
Evaluation of the safety and tolerability of GT005
Timepoint [2] 0 0
96 weeks
Secondary outcome [3] 0 0
Evaluation of the effect of GT005 on retinal anatomical measures
Timepoint [3] 0 0
96 weeks
Secondary outcome [4] 0 0
Evaluation of the effect of GT005 on functional measures
Timepoint [4] 0 0
96 weeks
Secondary outcome [5] 0 0
Evaluation of the effect of GT005 on functional measures
Timepoint [5] 0 0
96 weeks
Secondary outcome [6] 0 0
Evaluation of the effect of GT005 on visual function
Timepoint [6] 0 0
96 weeks
Secondary outcome [7] 0 0
Evaluation of the effect of GT005 on visual function
Timepoint [7] 0 0
96 weeks
Secondary outcome [8] 0 0
Evaluation of the effect of GT005 on patient-reported outcomes
Timepoint [8] 0 0
96 weeks

Eligibility
Key inclusion criteria
1. Able and willing to give written informed consent
2. Age =55 years
3. 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)
4. GA lesion(s) within an acceptable size on FAF, in the study eye
5. The GA lesion in the study eye must reside completely within the FAF image
6. Up to 25% of the enrolled study population are permitted to have CNV in the fellow eye
7. Have a BCVA of =24 letters (6/95 or 20/320 Snellen acuity equivalent), using ETDRS charts, in the study eye
8. 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
9. Able to attend all study visits and complete the study procedures
10. 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
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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
2. Have a history, or evidence, of CNV in the study eye
3. Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in the study eye
4. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye
5. History of intraocular surgery in the study eye within 12 weeks prior to Visit 1
6. Have clinically significant cataract that may require surgery during the study period in the study eye
7. 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
8. Axial myopia of greater than -8 diopters in the study eye
9. 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
10. Have received a gene or cell therapy at any time.
11. Have a contraindication to the protocol specified corticosteroid regimen
12. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
13. 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
14. 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/09/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
10/06/2024
Sample size
Target
Accrual to date
Final
255
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Sydney Hospital and Sydney Eye Hospital - Sydney
Recruitment hospital [2] 0 0
The University of Melbourne - The Centre for Eye Research Australia (CERA) - Melbourne E.
Recruitment postcode(s) [1] 0 0
2000 - Sydney
Recruitment postcode(s) [2] 0 0
3002 - Melbourne E.
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
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United States of America
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Florida
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United States of America
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Georgia
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United States of America
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Illinois
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United States of America
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Indiana
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United States of America
State/province [8] 0 0
Iowa
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United States of America
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Kansas
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United States of America
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Maryland
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United States of America
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Massachusetts
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United States of America
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Michigan
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Nevada
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
Country [17] 0 0
United States of America
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Oregon
Country [18] 0 0
United States of America
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Pennsylvania
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United States of America
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South Carolina
Country [20] 0 0
United States of America
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Tennessee
Country [21] 0 0
United States of America
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Texas
Country [22] 0 0
United States of America
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Utah
Country [23] 0 0
United States of America
State/province [23] 0 0
Washington
Country [24] 0 0
France
State/province [24] 0 0
Dijon
Country [25] 0 0
France
State/province [25] 0 0
Marseille
Country [26] 0 0
France
State/province [26] 0 0
Nantes
Country [27] 0 0
Germany
State/province [27] 0 0
Bonn
Country [28] 0 0
Germany
State/province [28] 0 0
Düsseldorf
Country [29] 0 0
Germany
State/province [29] 0 0
Lübeck
Country [30] 0 0
Germany
State/province [30] 0 0
Tuebingen
Country [31] 0 0
Poland
State/province [31] 0 0
Bydgoszcz
Country [32] 0 0
Spain
State/province [32] 0 0
Barcelona
Country [33] 0 0
Spain
State/province [33] 0 0
Madrid
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Spain
State/province [34] 0 0
Pamplona
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United Kingdom
State/province [35] 0 0
London
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United Kingdom
State/province [36] 0 0
Oxford
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Sunderland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Gyroscope Therapeutics Limited
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Novartis Pharmaceuticals
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT04566445