We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 6 for:    FOCUS | Geographic Atrophy
Previous Study | Return to List | Next Study

FOCUS: First in Human Study to Evaluate the Safety and Efficacy of GT005 Administered in Subjects With Dry AMD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03846193
Recruitment Status : Active, not recruiting
First Posted : February 19, 2019
Last Update Posted : December 1, 2022
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Gyroscope Therapeutics Limited

Brief Summary:
This is an open label first in human Phase I/II multicentre study of GT005 in subjects with Macular Atrophy due to AMD

Condition or disease Intervention/treatment Phase
Dry Age-related Macular Degeneration Macular Degeneration Retinal Disease Eye Diseases Retinal Degeneration Geographic Atrophy Macular Atrophy Biological: GT005 Device: GT005/ Device: Orbit™ Subretinal Delivery System Phase 1 Phase 2

Detailed Description:
This study will evaluate the safety, the dose response and efficacy (anatomical and functional visual outcomes) of three doses of GT005 administered as a single subretinal injection in genetically defined subjects with Macular Atrophy due to Age-related Macular Degeneration (AMD). Following consent, subjects will undergo a number of ophthalmic and clinical assessments to determine eligibility for inclusion in the study. Once eligibility is confirmed, subjects will be enrolled, receive treatment, and will be followed for 48 weeks, with the option to be followed for a further 4 years in long-term follow up after completing week 48.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 56 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: A dose escalation study of the safety and efficacy of a single subretinal injection of GT005 in subjects with Macular Atrophy due to AMD
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: FOCUS: An Open Label First in Human Phase I/II Multicentre Study to Evaluate the Safety, Dose Response and Efficacy of GT005 Administered as a Single Subretinal Injection in Subjects With Macular Atrophy Due to AMD
Actual Study Start Date : December 17, 2018
Estimated Primary Completion Date : May 23, 2023
Estimated Study Completion Date : January 19, 2027

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: GT005 Dose 1
A single dose of GT005 will be administered via subretinal injection
Biological: GT005
A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Experimental: GT005 Dose 2
A single dose of GT005 will be administered via subretinal injection
Biological: GT005
A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Experimental: GT005 Dose 3
A single dose of GT005 will be administered via subretinal injection
Biological: GT005
A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Experimental: GT005 Dose 1, 2 or 3
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
Biological: GT005
A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Experimental: GT005 Dose 2 with Orbit Subretinal Delivery System
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
Device: GT005/ Device: Orbit™ Subretinal Delivery System

A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Device: Orbit™ Subretinal Delivery System


Experimental: GT005 Dose 3 with Orbit Subretinal Delivery System
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
Device: GT005/ Device: Orbit™ Subretinal Delivery System

A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Device: Orbit™ Subretinal Delivery System


Experimental: GT005 Dose 1, 2 or 3 with Orbit Subretinal Delivery Sysem
A single dose of GT005 will be administered with subretinal injection via suprachoroidal cannulation approach
Device: GT005/ Device: Orbit™ Subretinal Delivery System

A recombinant non-replicating Adeno-Associated Viral (AAV) vector encoding a human complement factor

Device: Orbit™ Subretinal Delivery System





Primary Outcome Measures :
  1. Incidence of ocular and non-ocular Treatment Emergent AEs (TEAEs) and Treatment-Emergent Serious AEs (TESAEs) Severe Adverse Events (TEAE/TESAE) [ Time Frame: 48 weeks ]
    Proportion of patients with TEAEs/TESAEs after subretinal injection of GT005


Secondary Outcome Measures :
  1. Visual Acuity [ Time Frame: 48 weeks ]
    Change from baseline in BCVA (Best Corrected Visual Acuity) and LLVA (Low Luminance Visual Acuity) score via ETDRS (Early Treatment Diabetic Retinopathy Study) chart

  2. Macular Sensitivity [ Time Frame: 48 weeks ]
    Change from baseline in macular sensitivity as assessed by mesopic Microperimetry

  3. Geographic Atrophy [ Time Frame: 48 weeks ]
    Change from baseline in GA size as assessed by fundus autofluorescence

  4. Rate of successful delivery of Balanced Salt Solution (BSS) or BSS PLUS to the subretinal space (US only) [ Time Frame: Day 1 ]
    Proportion of subjects with successful delivery of BSS or BSS PLUS to subretinal space

  5. Rate of successful delivery of GT005 to the subretinal space (US only) [ Time Frame: Day 1 ]
    Proportion of subjects with successful delivery of GT005 to subretinal space

  6. Incidence of device-related AEs and SAEs (US only) [ Time Frame: Day 1 ]
    Proportion of subjects with device related AEs and SAEs after subretinal delivery with Orbit SDS



Information from the National Library of Medicine

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.


Layout table for eligibility information
Ages Eligible for Study:   55 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Able and willing to give consent to study participation
  2. 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)
  3. Cohorts 1 to 6: GA lesion(s) total size in the study eye must be ≥1.25mm2 and ≤17.5mm2.

    Cohort 7: GA lesion(s) total size in the study eye must be ≥1.25mm2

  4. GA lesion(s) in the study eye must reside completely within the FAF fundus image
  5. Cohorts 1 to 3: BCVA of ≤50 letters (6/36 Snellen acuity equivalent or worse) using ETDRS charts in the study eye Cohorts 4 to 7: BCVA of ≥24 letters (6/95 and 20/320 Snellen acuity equivalent or better) using ETDRS charts in the study eye
  6. Aged ≥55 years
  7. Able to attend all study visits and complete the study procedures
  8. Women of child-bearing potential need to have a negative urine pregnancy test within two weeks prior to receiving the drug. 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)

Exclusion Criteria:

  1. Have evidence or history of Choroidal Neovascularisation (CNV) in the study eye. Subjects 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
  2. Presence of moderate/severe non-proliferative diabetic retinopathy or worse in the study eye
  3. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye
  4. History of intraocular surgery in the study eye within 12 weeks prior to Screening (Visit 1). Yttrium aluminum garnet capsulotomy is permitted if performed >10 weeks prior to Visit 1
  5. Have clinically significant cataract that may require surgery during the study period in the study eye
  6. Presence of moderate to severe glaucomatous optic neuropathy in the study eye; uncontrolled IOP despite the use of more than two topical agents; a history of glaucoma-filtering or valve surgery is also excluded
  7. Axial myopia of greater than -8 diopters in the study eye
  8. 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
  9. Have received a gene or cell therapy at any time
  10. Have a contraindication to the specified protocol corticosteroid regimen
  11. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
  12. 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
  13. 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
  14. Cohorts 5 to 7 only: presence of metallic objects or implanted stimulator devices in or near the head, including cochlear implants, deep brain stimulators, vagus nerve stimulators, and other implanted electrodes or stimulators

Information from the National Library of Medicine

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): NCT03846193


Locations
Layout table for location information
United States, Florida
Retina Vitreous Associates of Florida
Saint Petersburg, Florida, United States, 33711
United States, Indiana
Midwest Eye Institute
Indianapolis, Indiana, United States, 46290
United States, Iowa
Wolfe Eye Clinic
West Des Moines, Iowa, United States, 50266
United States, Massachusetts
Ophthalamic Consultants of Boston (OCB)
Boston, Massachusetts, United States, 02114
United States, Missouri
Pepose Vision Institute
Chesterfield, Missouri, United States, 63017
United States, Nevada
Sierra Eye Associates
Reno, Nevada, United States, 89502
United States, Ohio
Cincinnati Eye Institute
Cincinnati, Ohio, United States, 45242
United States, Pennsylvania
Mid-Atlantic Retina
Philadelphia, Pennsylvania, United States, 19107
United Kingdom
Bristol Eye Hospital
Bristol, United Kingdom
London Vision Clinic
London, United Kingdom, W1G 7LA
Moorfields Eye Hospital
London, United Kingdom
Manchester Eye Hospital
Manchester, United Kingdom
Oxford University Hospital
Oxford, United Kingdom
Sunderland Eye Infirmary
Sunderland, United Kingdom
Sponsors and Collaborators
Gyroscope Therapeutics Limited
Novartis Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Chief Medical Officer Novartis Pharmaceuticals
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Gyroscope Therapeutics Limited
ClinicalTrials.gov Identifier: NCT03846193    
Other Study ID Numbers: GT005-01
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: December 1, 2022
Last Verified: November 2022

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Gyroscope Therapeutics Limited:
Dry Age-related Macular Degeneration (Dry AMD)
AMD
Atrophic AMD
Geographic Atrophy (GA)
Dry-AMD
Dry AMD
Additional relevant MeSH terms:
Layout table for MeSH terms
Geographic Atrophy
Atrophy
Macular Degeneration
Eye Diseases
Retinal Diseases
Retinal Degeneration
Anetoderma
Pathological Conditions, Anatomical
Eye Diseases, Hereditary
Connective Tissue Diseases
Skin Abnormalities
Skin Diseases