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Trial record 1 of 2 for:    FOCUS | Geographic Atrophy
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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 : September 28, 2020
Sponsor:
Information provided by (Responsible Party):
Gyroscope Therapeutics

Brief Summary:
This is an open label first in human Phase 1/2 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 Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 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 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
Actual Study Start Date : December 17, 2018
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

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 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.
Biological: 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. Geographical Atrophy (GA) Size [ 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 retinal drusen volume 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:   18 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. 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

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, Massachusetts
Ophthalamic Consultants of Boston (OCB) Recruiting
Boston, Massachusetts, United States, 02114
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: September 28, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Gyroscope Therapeutics:
Dry AMD
AMD
Atrophic AMD
Geographic Atrophy (GA)
Additional relevant MeSH terms:
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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