Back to resultsA Study of EXG102-031 in Patients With wAMD (Everest)
Primary Purpose
Neovascular (Wet) Age-related Macular Degeneration (nAMD)
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EXG102-031
Sponsored by
About this trial
This is an interventional treatment trial for Neovascular (Wet) Age-related Macular Degeneration (nAMD)
Eligibility Criteria
Inclusion Criteria: Male or female, age ≥ 50 years of age; Diagnosis of nAMD and current active lesion in the study eye at Screening; An ETDRS BCVA letter scores between 73 and 19 letters in the study eye; Response to anti-VEGF treatment during Screening; The study eye must be a pseudophakic lens (post-cataract surgery status); and Voluntarily agree to participate in the clinical trial, understand the trial procedures, and be capable of signing the informed consent form before screening. Exclusion Criteria: Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye; Presence in the study eye of CNV or macular edema due to causes other than AMD; Presence in the study eye of scarring, fibrosis or atrophy involving the macula; Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters; Active ocular infection in either eye; Presence of advanced glaucoma or uncontrolled glaucoma in the study eye; History of intraocular surgery in the study eye within 90 days of screening; or Prior receipt of any ocular or systemic gene therapy agent.
Sites / Locations
- Erie Retina ResearchRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
EXG102-031
Arm Description
Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of two dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into.
Outcomes
Primary Outcome Measures
Evaluation of safety and tolerability
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Secondary Outcome Measures
Evaluation of potential efficacy
Change from baseline in best corrected visual acuity (BCVA) measured by ETDRS method
Evaluation of potential safety
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Evaluation of supplementary therapy injections received
Average number of doses of anti-vascular endothelial growth factor (VEGF) supplemental therapy received
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05903794
Brief Title
A Study of EXG102-031 in Patients With wAMD (Everest)
Official Title
An Open-label, Dose-escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With EXG102-031 in Participants With Neovascular Age-related Macular Degeneration.
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
July 24, 2023 (Actual)
Primary Completion Date
December 31, 2024 (Anticipated)
Study Completion Date
December 31, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Exegenesis Bio
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
In neovascular (wet) age-related macular degeneration (nAMD), the macula, or the part of the eye that provides the clear, detailed central vision, is being affected by abnormal blood vessel growth and leakage. This leakage affects the vision over time and can lead to severe blurriness or blinding. EXG102-031 was made to block the extra vessel formation which would lead to less leakage affecting the vision. Before EXG102-031 can be tested for its efficacy (if it makes vision better), it must be tested to see if it is safely tolerated to confirm it can continue to be studied in more patients with nAMD.
Detailed Description
Age-related macular degeneration (AMD) is a major cause of blindness and visual impairment in older adults. The wet form of AMD, also called neovascular AMD (nAMD) usually causes faster vision loss than the dry form. The most common current treatments of nAMD are products that inhibit vascular endothelial growth factor (VEGF) (including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron) and are delivered by intravitreal injections at 4 to 16 week intervals and continued indefinitely. This Phase I, open-label, multiple-cohort, dose-escalation study is designed to evaluate the safety and tolerability of EXG102-031 gene therapy in subjects with previously treated nAMD. Safety will be assessed over 52 weeks after the administration of EXG102-031, and study participants will be followed for a total of five years after they receive the investigational administration of EXG102-031.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neovascular (Wet) Age-related Macular Degeneration (nAMD)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Model Description
Dose-escalation Study with two cohorts of three participants in each.
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
EXG102-031
Arm Type
Experimental
Arm Description
Each participant will receive a single subretinal injection of EXG102-031 in the study eye. Participants will be enrolled into one of two dosage groups sequentially, and the dose administered will be determined based on which study group the participant is enrolled into.
Intervention Type
Biological
Intervention Name(s)
EXG102-031
Intervention Description
EXG102-031 is a recombinant adeno-associated virus (rAAV) expressing an angiopoietin domain and VEGF receptor (ABD-VEGFR) fusion protein. EXG102-031 will be administered by subretinal injecting into one eye of each participant.
Primary Outcome Measure Information:
Title
Evaluation of safety and tolerability
Description
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Time Frame
Throughout 52 weeks
Secondary Outcome Measure Information:
Title
Evaluation of potential efficacy
Description
Change from baseline in best corrected visual acuity (BCVA) measured by ETDRS method
Time Frame
52 weeks post administration
Title
Evaluation of potential safety
Description
Frequency, type, and intensity of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs)
Time Frame
Through week 24
Title
Evaluation of supplementary therapy injections received
Description
Average number of doses of anti-vascular endothelial growth factor (VEGF) supplemental therapy received
Time Frame
Throughout 52 weeks post administration
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female, age ≥ 50 years of age;
Diagnosis of nAMD and current active lesion in the study eye at Screening;
An ETDRS BCVA letter scores between 73 and 19 letters in the study eye;
Response to anti-VEGF treatment during Screening;
The study eye must be a pseudophakic lens (post-cataract surgery status); and
Voluntarily agree to participate in the clinical trial, understand the trial procedures, and be capable of signing the informed consent form before screening.
Exclusion Criteria:
Presence of any ocular disease or history of disease in the study eye other than nAMD that may affect central visual acuity and/or macular detection, including retinal detachment, or in the opinion of the investigator could limit VA improvement in the study eye;
Presence in the study eye of CNV or macular edema due to causes other than AMD;
Presence in the study eye of scarring, fibrosis or atrophy involving the macula;
Subretinal hemorrhage accumulating in the center of the macula of the test eye, with an area of hemorrhage ≥ 4 optic disc diameters;
Active ocular infection in either eye;
Presence of advanced glaucoma or uncontrolled glaucoma in the study eye;
History of intraocular surgery in the study eye within 90 days of screening; or
Prior receipt of any ocular or systemic gene therapy agent.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Anna Oughton, PharmD
Phone
12153536621
Email
quality-exg102-031@exegenesisbio.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Arshad Khanani, MD
Organizational Affiliation
Sierra Eye Associates
Official's Role
Principal Investigator
Facility Information:
Facility Name
Erie Retina Research
City
Erie
State/Province
Pennsylvania
ZIP/Postal Code
16507
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bethany Bielak-Scott
Phone
814-456-4241
Ext
5408
Email
b.scott.research@pm.me
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study of EXG102-031 in Patients With wAMD (Everest)
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