Trial | NCT04246866  Report issue

Title

First in Human Study to Evaluate the Safety and Tolerability of GEM103 in Geographic Atrophy Secondary to Dry Age Related Macular Degeneration
A Phase 1, Multicenter, Open-label, Single-dose, Dose-escalation Study in Patients With Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD) to Evaluate the Safety, Tolerability, Pharmacodynamics, and Immunogenicity of Intravitreal Injections of GEM103

  • Phase

    Phase 1

  • Study Type

    Interventional

  • Status

    Completed No Results Posted

  • Intervention/Treatment

    GEM103

  • Study Participants

    12
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.
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 Started
Dec 19
2019
Primary Completion
Oct 21
2020
Study Completion
Oct 21
2020
Last Update
Feb 11
2021

Biological GEM103

GEM103

Dose 1 Experimental

A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103

Dose 2 Experimental

A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103

Dose 3 Experimental

A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103

Dose 4 Experimental

A single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103

Criteria 

Inclusion Criteria:

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

Exclusion Criteria:

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)

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
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
Female subjects must not be pregnant or lactating
Current use of medications known to be toxic to the lens, retina, or optic nerve
No Results Posted