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: February 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

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. Adults with bilateral Macular Atrophy due to Age-related Macular Degeneration

2. BCVA of ≤50 letters (6/36 Snellen acuity equivalent)

Exclusion Criteria

1. Evidence or history of neovascular AMD or diabetic retinopathy

2. History of prior surgery to eye (involving macula) or intravitreal drug therapy

3. Having contraindication to oral steroids

4. Significant ocular or non-ocular disease that would impact the subject's ability to participate in the study

5. Participation in another research program or participation with an investigational product within previous 12 weeks

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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