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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03846193
Recruitment Status : Recruiting
First Posted : February 19, 2019
Last Update Posted : November 4, 2020
Sponsor:
Information provided by (Responsible Party):
Gyroscope Therapeutics

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 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: 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 : February 2022
Estimated Study Completion Date : February 2025

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
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

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
A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor




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


Secondary Outcome Measures :
  1. Geographic Atrophy [ Time Frame: 48 weeks ]
    Change from baseline in GA size measured in mm2

  2. Retinal Drusen Volume [ Time Frame: 48 weeks ]
    Change from baseline in GA size measured in mm3

  3. Macular Sensitivity [ Time Frame: 48 weeks ]
    Change from baseline in macular sensitivity measured in decibels (DB)

  4. Monocular Reading Speed [ Time Frame: 48 weeks ]
    Change from baseline in monocular reading speed measured in words/min

  5. Vector Shedding [ Time Frame: 48 weeks ]
    Change from baseline in vector titres measured in vector genome/ml

  6. Complement factors [ Time Frame: 48 weeks ]
    Change from baseline in levels of complement factors, measured in nanograms/ml



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.


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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. Presence of Bilateral GA due to AMD on Colour Fundus Photography (CFP)
  3. Cohorts 1 to 3: GA lesions total size in the treatment eye must be ≥1.25mm2 and ≤17.5mm2 Cohorts 4 to 6: GA lesions total size must be ≥1.25mm2 and ≤17.5mm2 for both eyes.
  4. For all cohorts: the GA lesion in the treatment 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 treatment eye Cohorts 4 to 6: BCVA of ≥34 letters (6/60 Snellen acuity equivalent or better) using ETDRS charts in both eyes
  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.

Exclusion Criteria

  1. Have evidence or history of CNV in either eye
  2. Presence of moderate/severe or worse non-proliferative, diabetic retinopathy in either eye
  3. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in either eye
  4. Have clinically significant cataract that may require surgery during the study period in either eye
  5. Presence of moderate to severe glaucomatous optic neuropathy, uncontrolled IOP, use of two or more topical agents to control IOP; or a history of glaucoma-filtering surgery in either eye
  6. Axial myopia of greater than -8 diopters in the either eye
  7. 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
  8. Have received a gene or cell therapy at any time
  9. Have a contraindication to the corticosteroid regimen
  10. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant
  11. Have a history or presence of cutaneous squamous cell carcinoma
  12. 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
  13. Cohorts 5 and 6 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


Contacts
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Contact: Gyroscope Therapeutics +4401438532142 clinicaltrials@gyroscopetx.com

Locations
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United States, Iowa
Wolfe Eye Clinic Recruiting
West Des Moines, Iowa, United States, 50266
United States, Massachusetts
Ophthalamic Consultants of Boston (OCB) Recruiting
Boston, Massachusetts, United States, 02114
United States, Nevada
Sierra Eye Associates Recruiting
Reno, Nevada, United States, 89502
United Kingdom
Bristol Eye Hospital Recruiting
Bristol, United Kingdom
Contact: Bristol         
London Vision Clinic Recruiting
London, United Kingdom, W1G 7LA
Moorfields Eye Hospital Recruiting
London, United Kingdom
Contact: London         
Manchester Eye Hospital Recruiting
Manchester, United Kingdom
Contact: Manchester         
Oxford University Hospital Recruiting
Oxford, United Kingdom
Contact: Oxford         
Sunderland Eye Infirmary Recruiting
Sunderland, United Kingdom
Contact: Sunderland         
Sponsors and Collaborators
Gyroscope Therapeutics
Investigators
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Study Director: Chief Medical Officer Gyroscope Therapeutics Gyroscope Therapeutics
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Responsible Party: Gyroscope Therapeutics
ClinicalTrials.gov Identifier: NCT03846193    
Other Study ID Numbers: GT005-01
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: November 4, 2020
Last Verified: November 2020

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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:
Dry Age-related Macular Degeneration (Dry AMD)
AMD
Atrophic AMD
Geographic Atrophy (GA)
Dry-AMD
Dry AMD
Additional relevant MeSH terms:
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Macular Degeneration
Eye Diseases
Geographic Atrophy
Retinal Diseases
Retinal Degeneration
Anetoderma
Atrophy
Pathological Conditions, Anatomical
Eye Diseases, Hereditary
Connective Tissue Diseases
Skin Abnormalities
Skin Diseases