Geographic Atrophy Due to Age-Related Macular Degeneration
Conditions
Keywords
Geographic Atrophy, TO-O-1007, Intravitreal Implant, TO-I-1007
Brief summary
This study will evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of TO-O-1007, a biodegradable sustained-release intravitreal implant, for the treatment of Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). A long-acting intravitreal implant providing sustained delivery of API over 6 months may slow the progression of GA lesions while reducing treatment burden associated with frequent intravitreal injections. TO-O-1007 may offer a convenient treatment option with the potential to preserve retinal structure and visual function while maintaining an acceptable safety profile.
Detailed description
TO-O-1007 is a biodegradable sustained-release intravitreal implant, that is injected into the vitreous cavity of the eye. The implant is designed to gradually degrade over time, providing sustained ocular delivery of API for up to 6 months following a single administration. By continuously inhibiting C3/C5 activation, TO-O-1007 is intended to slow the progression of Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD), with the goal of preserving retinal structure and visual function. In this study, TO-O-1007 will be evaluated for its safety, tolerability, pharmacokinetics, and preliminary efficacy to determine whether it may offer a durable treatment option that reduces treatment burden compared with currently available therapies requiring more frequent intravitreal injections.
Interventions
DRUGTO-O-1007 (low-dose)
TO-O-1007 (low-dose): Participants will receive one TO-O-1007 implant administered by intravitreal injection into the study eye.
DRUGTO-O-1007 (high-dose)
TO-O-1007 (high-dose): Participants will receive two TO-O-1007 implants administered by intravitreal injection into the study eye.
PROCEDURESham injection
Sham injection: No injection is given. It is a sham injection to keep the participant masked.
Sponsors
Theratocular Biotek Co.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Masking description
Phase IIa: single masked (25 Participants)
Intervention model description
This is a Phase I/IIa interventional clinical trial evaluating the safety, tolerability, and preliminary efficacy of TO-O-1007, a biodegradable sustained-release intravitreal implant, in patients with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). Phase I is an open-label, single ascending dose study enrolling 6 participants across low- and high-dose cohorts, with Safety Review Committee (SRC) oversight before dose escalation. Phase IIa is a randomized, single-masked, proof-of-activity study enrolling 25 participants assigned to low-dose TO-O-1007, high-dose TO-O-1007, or sham control. Participants will be followed for 6 months plus 1 month of safety follow-up.
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Adults ≥50 years, able to provide informed consent and comply with study procedures. 2. Non-foveal GA secondary to dry AMD, confirmed by the central reading center. 3. Total GA area 2.5-17.5 mm² by FAF (≥1.25 mm² for at least one lesion if multifocal). 4. BCVA ≥24 ETDRS letters (\~20/320 Snellen) in the study eye. 5. IOP ≤21 mmHg in the study eye.
Exclusion criteria
1. Recent participation in intravitreal, GA, or investigational treatment studies. 2. Pregnant or nursing women. 3. High myopia (\>8 D or axial length \>27 mm). 4. Prior AMD treatment or active/secondary CNV in the study eye. 5. Current/prior uveitis. 6. Recent anti-complement therapy. 7. Use of prohibited medications, investigational products, or immunosuppressive therapies.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Treatment-Emergent Adverse Events (TEAEs) | Up to 6 months treatment duration | Number and percentage of participants experiencing at least one treatment-emergent adverse event, including serious adverse events and adverse events of special interest. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Geographic Atrophy Lesion Growth | Baseline, Week 12, and Week 24. | Change in the growth rate of square root-transformed total Geographic Atrophy lesion area in the study eye, assessed by fundus autofluorescence (FAF) imaging through Week 24. |
| Visual Function Assessments | Week 12 and Week 24. | Mean change from baseline in BCVA, LL-BCVA, and low-luminance deficit (LLD), assessed using ETDRS charts under photopic and low-luminance conditions through Week 24. |
| Exposure endpoint (only for phase I) | Day 1 pre-dose, Week 2, Week 4, Week 8, Week 12, Week 16, Week 20, Week 24 and Week 28 post first dosing. | Plasma concentrations of TO-O-1007 |
Countries
Australia
Contacts
CONTACTWeian Chien
CONTACTChung-Hsin Tsai
PRINCIPAL_INVESTIGATORDr. Andrew Chang
Sydney Retina Clinic & Day Surgery
Outcome results
None listed