Efficacy and Safety Evaluation of GAL-101 Ophthalmic Solution in Non-Foveal Geographic Atrophy Secondary to Non-Neovascular Age-Related Macular Degeneration
- Trial ID
- 2024-519128-26-00
- Protocol
- GAL-101-C0201
- Sponsor
- Galimedix Therapeutics Inc.
Trial statistics
Diseases & Conditions
Objectives
The primary objective of this study is to evaluate the **efficacy** of GAL-101 ophthalmic solution in reducing the rate of change in geographic atrophy (GA) lesion size in patients with non-foveal geographic atrophy secondary to non-neovascular age-related macular degeneration. This is clinically relevant as GA is a progressive condition that leads to vision loss, and slowing the progression of lesion size could potentially preserve visual function and improve quality of life for affected individuals.
Secondary objectives include:
- Evaluating the efficacy of GAL-101 in reducing the rate of change in photoreceptor degeneration (PRD) in eyes with GA.
- Assessing the neuroprotective efficacy of GAL-101 in preserving photoreceptors and retinal function.
- Determining the neuroenhancement efficacy of GAL-101 in improving visual function.
- Evaluating the ocular and systemic safety of GAL-101 ophthalmic solution.
Participants
The clinical trial involves a total of **90 participants** diagnosed with **non-foveal geographic atrophy secondary to non-neovascular age-related macular degeneration**. The study population includes both male and female subjects, aged **55 years and older**, who are not considered part of a vulnerable population. Participants were selected based on their ability to provide written informed consent and comply with the study schedule and assessments. They must have a **best-corrected visual acuity (BCVA)** of 50 letters or more in the study eye, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart, and a refractive error between +3 and -6 diopters spherical equivalent. The trial does not specify any particular lifestyle considerations such as diet or physical activity. Participants are required to have sufficiently clear ocular media and adequate pupillary dilation to permit quality fundus imaging, as well as the capability to complete macular pigment (MP) testing. The geographic atrophy lesions must meet specific size and location criteria, confirmed by a reading center, to ensure the integrity of the retinal pigment epithelium and outer retina in the study eye.
Plans and Procedures
The clinical trial is designed as a **randomized**, double-masked, placebo-controlled study to evaluate the efficacy and safety of GAL-101, a 2% ophthalmic solution, in patients with non-foveal geographic atrophy secondary to non-neovascular age-related macular degeneration. The trial will be conducted over an estimated duration from April 2025 to November 2025. Participants will be randomly assigned to receive either the active treatment or a matching placebo, ensuring that neither the participants nor the investigators know which treatment is being administered, thus maintaining the double-masked nature of the study.
The trial will commence with a screening visit to assess eligibility based on specific inclusion criteria, such as age (≥ 55 years), ability to provide informed consent, and specific ophthalmic conditions. Following successful screening, participants will undergo a baseline visit where initial measurements and assessments will be conducted. The primary endpoint is the comparison between groups of the annual rate of change in the area of geographic atrophy as measured by fundus autofluorescence from baseline to the last on-treatment visit. Secondary endpoints include changes in the area of photoreceptor degeneration and mean sensitivity of grid points using mesopic microperimetry.
Participants will be involved in the study for a maximum treatment period of 24 months, with regular follow-up visits scheduled to monitor safety and efficacy outcomes. These visits will include assessments such as fundus imaging and visual acuity tests. The end-of-study visit will conclude the trial, where final evaluations will be performed to gather comprehensive data on the treatment's impact. Conditions that may lead to early termination from the study include adverse events, withdrawal of consent, or non-compliance with study procedures.
Treatment
The clinical trial involves the administration of **GAL-101 salt**, an **ophthalmic solution** designed for the treatment of non-foveal geographic atrophy secondary to non-neovascular age-related macular degeneration. The active substance in GAL-101 salt is **sodium (R)-2-(2-amino-3-(1H-indol-3-yl)propanamido)-2-methylpropanoate-propan-2-ol**, a chemical compound. The pharmaceutical form of the medication is an ophthalmic solution, and it is administered via the ophthalmic route. The maximum daily dose is 2.4 mg, with a total maximum dose of 1.2 g over a treatment period of 24 weeks. The solution is not formulated for pediatric use and is not classified as an orphan drug.
The study also includes a **placebo** group, which receives a matching ophthalmic solution. This placebo contains the same excipients as the GAL-101 ophthalmic solution, with **sodium chloride** replacing the active pharmaceutical ingredient (API) to achieve the same osmolarity. The placebo is used to maintain the double-masked nature of the trial, ensuring that neither the participants nor the investigators are aware of the treatment assignments. The placebo is administered in the same manner as the experimental medication, following the same dosing schedule and route of administration.
Efficacy
The efficacy of GAL-101 ophthalmic solution in the treatment of non-foveal geographic atrophy secondary to non-neovascular age-related macular degeneration will be assessed through a series of primary and secondary endpoints. The primary endpoint involves the comparison between groups of the annual rate of change in the area of geographic atrophy (GA) as measured by fundus autofluorescence (FAF) from baseline to the last on-treatment visit. This measurement will provide a quantitative assessment of the treatment's impact on the progression of GA lesions.
Secondary endpoints include the comparison between groups of the annual rate of change in the area of photoreceptor degeneration (PRD) as measured by optical coherence tomography (OCT), and the annual rate of change in mean sensitivity of grid points using mesopic microperimetry (MP). These secondary measures will be evaluated from baseline to the last on-treatment visit, offering additional insights into the treatment's effect on retinal structure and function.
Inclusion and Exclusion Criteria
Inclusion Criteria
- > = 55 years of age
- Willing and able to provide written informed consent
- Willing and able to comply with the study schedule and study assessments
- Able to successfully administer ophthalmic solution or have an appropriate designee (e.g., family member, health care professional) who can administer ophthalmic solution.
- BCVA of > = 50 letters in the study eye using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (i.e., 20/100 Snellen equivalent). Criterion will be confirmed at Baseline.
- Refractive error between +3 and -6 diopters spherical equivalent in the study eye
- Sufficiently clear ocular media and adequate pupillary dilation to permit quality fundus imaging of the study eye, in opinion of the Investigator. Criterion will be confirmed at Baseline
- Willing and capable of completing MP testing of the study eye in the opinion of the Investigator and verified by the reading center
- Previously established diagnosis of non-foveal GA secondary to non-neovascular AMD in the study eye. Specific GA lesion criteria will be confirmed by the reading center: a. Well-delineated cumulative GA area between 1.25 and 12.0 mm2; b. If GA is multifocal, at least 1 lesion >= 0.7 mm2; c. GA lesions must be located outside a >= 100 µm radius from the center point of the fovea (i.e., this area must have intact retinal pigment epithelium [RPE] and outer retina); d. GA lesions must be located (partially or wholly) within a 2000 µm radius from the center point of the fovea; e. GA lesions must be completely located within FAF imaging field (field 2 to 30-degree image centered on the fovea). GA lesion borders must be > 300 µm from image edges. f. GA lesion(s) are not confluent with the optic disc or peripapillary atrophy (peripapillary atrophy may otherwise be present); g. Area of PRD must be cumulatively between 5.0 and 25.0 mm2
Exclusion Criteria
- (Study Eye) Presence or history of choroidal neovascularization (CNV). Criterion will be confirmed at Baseline.
- (Study Eye) History of laser therapy in the macular region, regardless of indication.
- (Study Eye) History of herpes zoster.
- (Study Eye) Ophthalmic disease or condition that requires or is likely to require surgery during the study period.
- (Study Eye) GA with cumulative area < 1.25 mm2
- (Study Eye) Any GA lesion within 100 µm radius from the center point of the fovea.
- (Study Eye) Axial length > 26 mm
- (Study Eye) Any ocular disease or condition other than non-neovascular AMD that may, in the opinion of the Investigator, interfere with study assessments, patient adherence to the study schedule, or interpretation of study data (e.g., epiretinal membrane, macular hole, glaucomatous optic neuropathy, presumed ocular histoplasmosis, etc.)
- (Study Eye) Intraocular surgery (including cataract extraction and crystalline lens replacement) within 3 months before Visit 1a, or yttrium aluminum garnet (YAG) surgery within 2 months before Visit 1a, or planned either during the study period.
- (Study Eye) Use of any pharmacologic (e.g., pegcetacoplan or avacincaptad pegol) or device (e.g., photobiomodulation) intervention intended for the treatment of non-neovascular AMD or other macular disease within 6 months before Visit 1a, or planned use during the study period
- (Study Eye) Use of any prescription or over-the-counter ophthalmic medication within 1 month before Visit 1a or planned use during the study period Note: a) Ophthalmic solutions used during study assessments are exempt and allowed b) Intraocular pressure (IOP)-lowering therapies are exempt and allowed if patient’s IOP is well controlled, in the Investigator’s opinion, using a single bottle, the treatment has been stable for >3 months, IOP is ≤26 mmHg at Visit 1a, and the treatment is not expected to change during the study period. Combination therapy using a single bottle is allowed c) Artificial tears and lifitegrast or cyclosporine eye drops are exempt and allowed d) Approved therapies for non-neovascular age-related macular degeneration are exempt and allowed at any time after randomization e) Approved therapies for neovascular AMD are exempt and allowed at any time after randomization if CNV was newly detected f) For local eye irritation or inflammation, short course ophthalmic treatments containing corticosteroids (i.e., fluoromethalone or loteprednol) are exempt and allowed
- (Study Eye) Use of rigid contact lenses within 1 month before Visit 1a or planned use during the study period.
- (Non-study Eye) BCVA of < 5 letters using ETDRS chart (i.e., 20/800 Snellen equivalent)
- (Either Eye) History of uveitis
- (Either Eye) GA Secondary to any condition other than non-neovascular AMD
- (Either Eye) History of active ocular infection or inflamation within 3 months before Visit 1a or Baseline. Criterion will be confirmed at Baseline. Note: Acute conjunctivitis is only exclusionary within 1 month before Visit 1a or Baseline.
- (Either Eye) Underwent investigational treatment for AMD within 6 months before Visit 1a.
- (General Exclusion Criteria) History of therapeutic radiation to the cranium.
- (General Exclusion Criteria) Known allergy or hypersensitivity to the IMP or any of its excipients.
- (General Exclusion Criteria) History of malignant disease. Note: Patients with active malignancies, defined as presence of detectable cancer or undergoing treatment for cancer, are not eligible for the study. Note: Patients may be eligible based on their overall health status, as determined by the Investigator in consultation with the Medical Monitor(s) and Sponsor, if they have undergone surgical treatment resulting in pathologically confirmed complete resection of the cancer or are in complete remission with no evidence of cancer, and off all therapies (excluding prophylactic therapies).
- (General Exclusion Criteria) Use of hydroxychloroquine within 1 month before Visit 1a, or planned use during the study period.
- (General Exclusion Criteria) Participated in other IMP study or treatment with any other IMP within 1 month or 5 times the half-life of the IMP/relevant metabolites (whichever is longer) before Visit 1a or plan to participate in any other IMP study during the study period
- (General Exclusion Criteria) Use of lutein > 10 mg per day or zeaxanthin > 2 mg per day within 1 month before Visit 1a, or planned use during the study period.
- (General Exclusion Criteria) Any medical condition (including mental), in the opinion of the Investigator, that could interfere with study assessments, patient adherence to the study schedule, or interpretation of study data, or uncontrolled Grade 3 hypertension as defined in 2023 European Society of Hypertension Guidelines (systolic, ≥180 mmHg; diastolic, ≥110 mmHg)
- (General Exclusion Criteria) Screening laboratory values, in the opinion of the Investigator, that make the patient unsuitable for study participation.
- (General Exclusion Criteria) Pregnant, nursing, or planning a pregnancy during the study. Criterion will be confirmed at Baseline.
- (General Exclusion Criteria) Unwilling or unable to use an acceptable method of contraception throughout the study if a woman of childbearing potential (WOCBP) or if a sexual partner of a WOCBP.
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 France | Recruiting | 30 Apr 2025 | 20 |
 Germany | Recruiting | 30 Apr 2025 | 10 |
 Ireland | Recruiting | 30 Apr 2025 | 20 |
 Italy | Recruiting | 30 Apr 2025 | 15 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
GAL-101 ophtalmic solution matching placebo. It contains the excipients as GAL-101 ophtalmic solution plus Sodium Chloride instead of the API, which allows to achieve the same osmolarity as GAL-101 ophtalmic solution | Placebo | N/A | — | — | — | N/A |
GAL-101 salt | Test | OPHTHALMIC SOLUTION | OPHTHALMIC | 2.4 | 24 | PRD11763332 |