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

Macular Degeneration Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04246866
• Other study ID #: GEM-CL-10301
• Status: Completed
• Phase: Phase 1
• Start date: December 19, 2019
• Est. completion date: October 21, 2020

Study information

• Verified date: February 2021
• Source: Gemini Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: October 21, 2020
• Est. primary completion date: October 21, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Atrophy
• Dry Age-related Macular Degeneration
• Geographic Atrophy
• Macular Degeneration
• Retinal Degeneration
• Retinal Disease
• Retinal Diseases

Intervention

Biological:
• GEM103
GEM103

Locations

Country	Name	City	State
United States	Western Carolina Retinal Associates	Asheville	North Carolina
United States	Southeast Retina Center	Augusta	Georgia
United States	California Retina Consultants	Bakersfield	California
United States	Retina Vitreous Associates	Beverly Hills	California
United States	Pepose Vision Institute	Chesterfield	Missouri
United States	Midwest Eye Institute	Indianapolis	Indiana
United States	California Retina Consultants	Oxnard	California
United States	Associated Retina Consultants	Phoenix	Arizona
United States	Retinal Consultants of Arizona	Phoenix	Arizona
United States	Sierra Eye Associates	Reno	Nevada
United States	California Retina Consultants	Santa Barbara	California
United States	California Retina Consultants	Santa Maria	California

Sponsors (1)

Lead Sponsor	Collaborator
Gemini Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change from baseline in aqueous humor biomarker levels (in ng/mL) of complement factors		Up to 8 Weeks
Other	Change from baseline in clinical assessments including BCVA and LLVA scores as assessed by ETDRS Visual Acuity Score in number of letters		Up to 8 Weeks
Other	Change from baseline in clinical assessments including area of GA in mm2 as assessed by fundus imaging		Up to 8 Weeks
Other	Change in concentration of GEM103 in blood samples		Up to 8 Weeks
Primary	Incidence and severity of AEs/SAEs following drug administration		Up to 8 Weeks
Secondary	GEM103 concentrations in ocular fluids		Up to 8 Weeks
Secondary	Changes in complement factor levels compared to baseline after drug administration		Up to 8 Weeks