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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04246866
Recruitment Status : Active, not recruiting
First Posted : January 29, 2020
Last Update Posted : October 9, 2020
Sponsor:
Information provided by (Responsible Party):
Gemini Therapeutics, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Dry Age-related Macular Degeneration Geographic Atrophy Macular Degeneration Retinal Disease Retinal Degeneration Biological: GEM103 Phase 1

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : December 19, 2019
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose 1
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103
 Biological: GEM103
GEM103
Experimental: Dose 2
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103
 Biological: GEM103
GEM103
Experimental: Dose 3
A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103
 Biological: GEM103
GEM103
Experimental: Dose 4
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103
 Biological: GEM103
GEM103


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of AEs/SAEs following drug administration [ Time Frame: Up to 8 Weeks ]

Secondary Outcome Measures :
  1. GEM103 concentrations in ocular fluids [ Time Frame: Up to 8 Weeks ]
  2. Changes in complement factor levels compared to baseline after drug administration [ Time Frame: Up to 8 Weeks ]

Other Outcome Measures:
  1. Change from baseline in aqueous humor biomarker levels (in ng/mL) of complement factors [ Time Frame: Up to 8 Weeks ]
  2. Change from baseline in clinical assessments including BCVA and LLVA scores as assessed by ETDRS Visual Acuity Score in number of letters [ Time Frame: Up to 8 Weeks ]
  3. Change from baseline in clinical assessments including area of GA in mm2 as assessed by fundus imaging [ Time Frame: Up to 8 Weeks ]
  4. Change in concentration of GEM103 in blood samples [ Time Frame: Up to 8 Weeks ]

Eligibility Criteria
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Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04246866


Locations
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United States, Arizona
Associated Retina Consultants
Phoenix, Arizona, United States, 85020
Retinal Consultants of Arizona
Phoenix, Arizona, United States, 85053
United States, California
California Retina Consultants
Bakersfield, California, United States, 93309
Retina Vitreous Associates
Beverly Hills, California, United States, 90211
California Retina Consultants
Oxnard, California, United States, 93036
California Retina Consultants
Santa Barbara, California, United States, 93103
California Retina Consultants
Santa Maria, California, United States, 93454
United States, Georgia
Southeast Retina Center
Augusta, Georgia, United States, 30909
United States, Indiana
Midwest Eye Institute
Indianapolis, Indiana, United States, 46290
United States, Missouri
Pepose Vision Institute
Chesterfield, Missouri, United States, 63017
United States, Nevada
Sierra Eye Associates
Reno, Nevada, United States, 89502
United States, North Carolina
Western Carolina Retinal Associates
Asheville, North Carolina, United States, 28803
Sponsors and Collaborators
Gemini Therapeutics, Inc.
More Information
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Responsible Party: Gemini Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04246866    
Other Study ID Numbers: GEM-CL-10301
First Posted: January 29, 2020    Key Record Dates
Last Update Posted: October 9, 2020
Last Verified: October 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Macular Degeneration
Geographic Atrophy
Retinal Diseases
Retinal Degeneration
 Atrophy
Eye Diseases
Pathological Conditions, Anatomical
Eye Diseases, Hereditary