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
1 other identifier
interventional
12
1 country
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Dec 2019
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 19, 2019
CompletedFirst Submitted
Initial submission to the registry
January 6, 2020
CompletedFirst Posted
Study publicly available on registry
January 29, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 21, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
October 21, 2020
CompletedFebruary 11, 2021
February 1, 2021
10 months
January 6, 2020
February 9, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence and severity of AEs/SAEs following drug administration
Up to 8 Weeks
Secondary Outcomes (2)
GEM103 concentrations in ocular fluids
Up to 8 Weeks
Changes in complement factor levels compared to baseline after drug administration
Up to 8 Weeks
Other Outcomes (4)
Change from baseline in aqueous humor biomarker levels (in ng/mL) of complement factors
Up to 8 Weeks
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
Change from baseline in clinical assessments including area of GA in mm2 as assessed by fundus imaging
Up to 8 Weeks
- +1 more other outcomes
Study Arms (4)
Dose 1
EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103
Dose 2
EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103
Dose 3
EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103
Dose 4
EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
Associated Retina Consultants
Phoenix, Arizona, 85020, United States
Retinal Consultants of Arizona
Phoenix, Arizona, 85053, United States
California Retina Consultants
Bakersfield, California, 93309, United States
Retina Vitreous Associates
Beverly Hills, California, 90211, United States
California Retina Consultants
Oxnard, California, 93036, United States
California Retina Consultants
Santa Barbara, California, 93103, United States
California Retina Consultants
Santa Maria, California, 93454, United States
Southeast Retina Center
Augusta, Georgia, 30909, United States
Midwest Eye Institute
Indianapolis, Indiana, 46290, United States
Pepose Vision Institute
Chesterfield, Missouri, 63017, United States
Sierra Eye Associates
Reno, Nevada, 89502, United States
Western Carolina Retinal Associates
Asheville, North Carolina, 28803, United States
Related Publications (1)
Khanani AM, Maturi RK, Bagheri N, Bakall B, Boyer DS, Couvillion SS, Dhoot DS, Holekamp NM, Jamal KN, Marcus DM, Pieramici D, Aziz AA, Patki KC, Bridges WZ Jr, Barone SB. A Phase I, Single Ascending Dose Study of GEM103 (Recombinant Human Complement Factor H) in Patients with Geographic Atrophy. Ophthalmol Sci. 2022 Apr 11;2(2):100154. doi: 10.1016/j.xops.2022.100154. eCollection 2022 Jun.
PMID: 36249705DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 6, 2020
First Posted
January 29, 2020
Study Start
December 19, 2019
Primary Completion
October 21, 2020
Study Completion
October 21, 2020
Last Updated
February 11, 2021
Record last verified: 2021-02
Data Sharing
- IPD Sharing
- Will not share