First in Human Study to Evaluate the Safety and Tolerability of GEM103 in Geographic Atrophy Secondary to Dry Age Related Macular Degeneration

Phase 1

Completed

• Conditions: Retinal Degeneration; Dry Age-related Macular Degeneration; Macular Degeneration; Geographic Atrophy; Retinal Disease
• Interventions: Biological: GEM103

• Registration Number: NCT04246866
• Lead Sponsor: Gemini Therapeutics, Inc.

Brief Summary

The study is designed to identify the maximum tolerated dose (MTD) for intravitreal (IVT) administration of GEM103 in subjects with geographic atrophy (GA) secondary to dry AMD. Safety and tolerability of a single dose of GEM103 will be assessed based on the occurrence of dose-limiting toxicities (DLTs). Each subject will be followed for safety, pharmacokinetic (PK), clinical, and biomarker evaluations. Three escalating dose cohorts are planned.

Detailed Description

This is a Phase 1, multicenter, open-label, single-dose, dose-escalation study in subjects with GA secondary to dry AMD to investigate the safety, tolerability, pharmacodynamics (PD), and immunogenicity of IVT injections of GEM103 to a single eye.

The study is designed to identify the MTD for IVT administration of GEM103. Safety and tolerability of a single dose of GEM103 will be assessed based on the occurrence of DLTs. Three escalating dose cohorts are planned.

Subjects will undergo clinical and ophthalmic assessments for inclusion eligibility into the study. Enrolled subjects will receive GEM103 and be followed for safety, PK, clinical, and biomarker evaluations.

Study Timeline

• Study Start: 2019-12-19
• Study First Submitted: 2020-01-06
• Study First Submitted QC: 2020-01-28
• Study First Posted: 2020-01-29
• Primary Completion: 2020-10-21
• Study Completion: 2020-10-21
• Status Verified: 2021-02
• Last Update Submitted: 2021-02-09
• Last Update Posted: 2021-02-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. At least 50 years old at the time of signed informed consent
2. Able to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment and provide informed consent prior to initiation of any study procedure
3. Best corrected visual acuity (BCVA) in study eye between 5-45 letters
4. Confirmed diagnosis of central GA in the study eye and eligible total GA lesion size

Exclusion Criteria

1. Presence of the following ocular conditions - in the study eye:

   • Exudative AMD or choroidal neovascularization (CNV)
   • Any ocular disease or condition that could impact the subject's ability to participate in the study or be a contraindication to IVT injection
   • Any intraocular surgery (with the exception of intraocular lens replacement surgery more than 3 months prior to consent)

2. Presence of any of the following ocular conditions - in either eye:

   • History of herpetic infection
   • Ongoing treatment with antiangiogenic therapies in the fellow eye or completed treatment in the study eye

3. Any prior or ongoing medical condition (e.g. ocular other than dry AMD, systemic, psychiatric) or clinically significant screening laboratory value that may present a safety risk, interfere with study compliance, interfere with consistent study follow-up, or confound data interpretation throughout the longitudinal follow-up period

4. Female subjects must not be pregnant or lactating

5. Current use of medications known to be toxic to the lens, retina, or optic nerve

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Dose 1	GEM103	A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103
Dose 3	GEM103	A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103
Dose 4	GEM103	A single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103
Dose 2	GEM103	A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103

Primary Outcome Measures

Name	Time	Method
Incidence and severity of AEs/SAEs following drug administration	Up to 8 Weeks

Secondary Outcome Measures

Name	Time	Method
Changes in complement factor levels compared to baseline after drug administration	Up to 8 Weeks
GEM103 concentrations in ocular fluids	Up to 8 Weeks

Trial Locations

Locations (9)

California Retina Consultants; 🇺🇸 Santa Maria, California, United States

Retina Vitreous Associates; 🇺🇸 Beverly Hills, California, United States

Pepose Vision Institute; 🇺🇸 Chesterfield, Missouri, United States

Sierra Eye Associates; 🇺🇸 Reno, Nevada, United States

Western Carolina Retinal Associates; 🇺🇸 Asheville, North Carolina, United States

Southeast Retina Center; 🇺🇸 Augusta, Georgia, United States

Associated Retina Consultants; 🇺🇸 Phoenix, Arizona, United States

Retinal Consultants of Arizona; 🇺🇸 Phoenix, Arizona, United States

Midwest Eye Institute; 🇺🇸 Indianapolis, Indiana, United States