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

Overview

This is an open label first in human Phase 1/2 multicentre study of GT005 in subjects with Macular Atrophy due to AMD

Full Title of Study: “FocuS: An Open Label First in Human Phase 1/2 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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2021

Detailed Description

This study will evaluate the safety, the dose response and efficacy (anatomical and functional visual outcomes) of two 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.

Interventions

  • Biological: GT005
    • A recombinant non-replicating adeno-associated viral (AAV) vector encoding a human complement factor

Arms, Groups and Cohorts

  • Experimental: GT005 Dose 1
    • A single dose of GT005 will be administered via subretinal injection
  • Experimental: GT005 Dose 2
    • A single dose of GT005 will be administered via subretinal injection
  • Experimental: GT005 Dose 3
    • A single dose of GT005 will be administered via subretinal injection
  • Experimental: GT005 Dose 4
    • A single dose of GT005 will be administered via subretinal injection. This dose will be determined by dose levels determined to be safe and tolerable in Arms 1,2 and 3.

Clinical Trial Outcome Measures

Primary Measures

  • 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 Measures

  • Geographical Atrophy (GA) Size
    • Time Frame: 48 weeks
    • Change from baseline in GA size measured in mm2
  • Retinal Drusen Volume
    • Time Frame: 48 weeks
    • Change from baseline in retinal drusen volume measured in mm3
  • Macular Sensitivity
    • Time Frame: 48 weeks
    • Change from baseline in macular sensitivity measured in decibels (DB)
  • Monocular Reading Speed
    • Time Frame: 48 weeks
    • Change from baseline in monocular reading speed measured in words/min
  • Vector Shedding
    • Time Frame: 48 weeks
    • Change from baseline in vector titres measured in vector genome/ml
  • Complement factors
    • Time Frame: 48 weeks
    • Change from baseline in levels of complement factors, measured in nanograms/ml

Participating in This Clinical Trial

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. GA lesions total size in the study eye must be ≥1.25mm2 and ≤17.5mm2 for the study eye

4. The GA lesion 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 Cohort 4: BCVA of ≥34 letters (20/200 Snellen acuity equivalent or better) using ETDRS charts in the study eye

6. Aged ≥18 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. Evidence or history of neovascular AMD or diabetic retinopathy [either eye]

2. History of significant ocular surgery which, in the opinion of the Investigator, may either put the subject at risk if participating in the study, or may influence the results of the study, or the subject's ability to participate in the study [either eye]

3. Concomitant disease(s) limiting the subject's ability to undergo retinal surgery [study eye]

4. Clinically significant cataract that may require surgery during the study period [study eye]

5. Any sign of macular changes that might indicate a separate pathology independent of AMD, such as pathological myopia (e.g.>6 dioptres of myopia) or history of significant amblyopia [either eye]

6. Any other significant ocular or non-ocular disease/disorder which, in the opinion of the Investigator, may either put the subject at risk if participating in the study, or may influence the results of the study, or the subject's ability to participate in the study [either eye]

7. Having a contraindication to oral prednisolone/prednisone, such as gastric ulcer, or significant side effects

8. Current participation in another research study, including observational studies

9. Participation in another research study involving an investigational product in the previous 12 weeks or received a gene/cell based therapy at any time previously [either eye]

10. Any known sensitivity to GT005 formulation component or excipients

11. Unwilling to use two forms of contraception (one of which being a barrier method) 90 days for men and 30 days for women post-dosing, if relevant

12. History or presence of cutaneous squamous cell carcinoma

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Gyroscope Therapeutics
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Chief Medical Officer Gyroscope Therapeutics, Study Director, Gyroscope Therapeutics
  • Overall Contact(s)
    • Gyroscope Therapeutics, +4401438532142, clinicaltrials@gyroscopetx.com

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