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/NCT04437368




Registration number
NCT04437368
Ethics application status
Date submitted
3/06/2020
Date registered
18/06/2020
Date last updated
8/07/2024

Titles & IDs
Public title
EXPLORE: A Phase II Study to Evaluate the Safety and Efficacy of Two Doses of GT005
Scientific 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
Secondary ID [1] 0 0
CPPY988A12202
Secondary ID [2] 0 0
GT005-02
Universal Trial Number (UTN)
Trial acronym
EXPLORE
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; Low Dose
Treatment: Drugs - GT005; High Dose
Treatment: Drugs - GT005; Low Dose

Experimental: Part 1 - GT005 Low Dose - Approximately 25 subjects are planned, with subjects randomised to GT005 Low Dose.

Experimental: Part 1 - GT005 High Dose - Approximately 25 subjects are planned, with subjects randomised to GT005 High Dose.

No intervention: Part 1 - Untreated control - Approximately 25 subjects are planned, with subjects randomised to untreated control.

Experimental: Part 2 - GT005 Low Dose - Approximately 116 subjects are planned, with subjects randomised to Part 2 - GT005 Low Dose.

No intervention: Part 2 - Untreated control - Approximately 54 subjects are planned, with subjects randomised to untreated control.


Treatment: Drugs: GT005; Low Dose
Part 1 of the study will test two doses of GT005: Low Dose and High Dose.

Treatment: Drugs: GT005; High Dose
Part 1 of the study will test two doses of GT005: Low Dose and High Dose.

Treatment: Drugs: GT005; Low Dose
Part 2 of the study will test one dose: Low 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
48 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. 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)
4. Have GA lesion(s) total size between or equal to 1.25mm2 to 17.5mm2 in the study eye
5. The GA lesion(s) 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, defined as either:

1. Non-exudative/sub-clinical fellow eye CNV identified at Screening, or
2. Known history of fellow eye CNV with either =2 years since diagnosis or with no active treatment required in 6 months prior to Screening
7. Have a BCVA of 24 letters (6/95 and 20/320 Snellen acuity equivalent) or better, using ETDRS charts, in the study eye
8. 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
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. 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)
Minimum age
55 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subjects who have a clinical diagnosis of Stargardt Disease or other retinal dystrophies, confirmed by the central reading centre
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 Screening (Visit 1). Yttrium aluminium garnet capsulotomy is permitted if performed >10 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 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
8. Axial myopia of greater than -8 dioptres in the study eye
9. 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
10. Have a contraindication to specified protocol corticosteroid regimen
11. 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
12. Have received a gene or cell therapy at any time
13. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
14. 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

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 assessing the outcomes
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
14/07/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/04/2024
Sample size
Target
Accrual to date
Final
98
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The University of Melbourne - The Centre for Eye Research Australia (CERA) - Melbourne E.
Recruitment hospital [2] 0 0
Sydney Hospital and Sydney Eye Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Melbourne E.
Recruitment postcode(s) [2] 0 0
2000 - Sydney
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
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Nevada
Country [12] 0 0
United States of America
State/province [12] 0 0
New Mexico
Country [13] 0 0
United States of America
State/province [13] 0 0
New York
Country [14] 0 0
United States of America
State/province [14] 0 0
Ohio
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
Pennsylvania
Country [17] 0 0
United States of America
State/province [17] 0 0
Tennessee
Country [18] 0 0
United States of America
State/province [18] 0 0
Texas
Country [19] 0 0
United States of America
State/province [19] 0 0
Washington
Country [20] 0 0
United States of America
State/province [20] 0 0
West Virginia
Country [21] 0 0
France
State/province [21] 0 0
Alpes-Cote d'Azur
Country [22] 0 0
France
State/province [22] 0 0
Bourgogne-Franche-Comté
Country [23] 0 0
France
State/province [23] 0 0
Pays De La Loire
Country [24] 0 0
Germany
State/province [24] 0 0
Schleswig-Holstein
Country [25] 0 0
Germany
State/province [25] 0 0
Bonn
Country [26] 0 0
Germany
State/province [26] 0 0
Düsseldorf
Country [27] 0 0
Germany
State/province [27] 0 0
Münster
Country [28] 0 0
Germany
State/province [28] 0 0
Tübingen
Country [29] 0 0
Netherlands
State/province [29] 0 0
Nijmegen
Country [30] 0 0
Poland
State/province [30] 0 0
Bydgoszcz
Country [31] 0 0
Spain
State/province [31] 0 0
Barcelona
Country [32] 0 0
Spain
State/province [32] 0 0
Navarra
Country [33] 0 0
Spain
State/province [33] 0 0
Córdoba
Country [34] 0 0
Spain
State/province [34] 0 0
Madrid
Country [35] 0 0
Spain
State/province [35] 0 0
Valencia
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Bristol
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Liverpool
Country [38] 0 0
United Kingdom
State/province [38] 0 0
London
Country [39] 0 0
United Kingdom
State/province [39] 0 0
Sheffield
Country [40] 0 0
United Kingdom
State/province [40] 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/NCT04437368