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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

Primary Purpose

Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
GEM103
Sponsored by
Gemini Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dry Age-related Macular Degeneration

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Associated Retina Consultants
  • Retinal Consultants of Arizona
  • California Retina Consultants
  • Retina Vitreous Associates
  • California Retina Consultants
  • California Retina Consultants
  • California Retina Consultants
  • Southeast Retina Center
  • Midwest Eye Institute
  • Pepose Vision Institute
  • Sierra Eye Associates
  • Western Carolina Retinal Associates

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Dose 1

Dose 2

Dose 3

Dose 4

Arm Description

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

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

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

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

Outcomes

Primary Outcome Measures

Incidence and severity of AEs/SAEs following drug administration

Secondary Outcome Measures

GEM103 concentrations in ocular fluids
Changes in complement factor levels compared to baseline after drug administration

Full Information

First Posted
January 6, 2020
Last Updated
February 9, 2021
Sponsor
Gemini Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04246866
Brief Title
First in Human Study to Evaluate the Safety and Tolerability of GEM103 in Geographic Atrophy Secondary to Dry Age Related Macular Degeneration
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
Study Type
Interventional

2. Study Status

Record Verification Date
February 2021
Overall Recruitment Status
Completed
Study Start Date
December 19, 2019 (Actual)
Primary Completion Date
October 21, 2020 (Actual)
Study Completion Date
October 21, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gemini Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration, Retinal Disease, Retinal Degeneration

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Dose 1
Arm Type
Experimental
Arm Description
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103
Arm Title
Dose 2
Arm Type
Experimental
Arm Description
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103
Arm Title
Dose 3
Arm Type
Experimental
Arm Description
A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103
Arm Title
Dose 4
Arm Type
Experimental
Arm Description
A single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103
Intervention Type
Biological
Intervention Name(s)
GEM103
Intervention Description
GEM103
Primary Outcome Measure Information:
Title
Incidence and severity of AEs/SAEs following drug administration
Time Frame
Up to 8 Weeks
Secondary Outcome Measure Information:
Title
GEM103 concentrations in ocular fluids
Time Frame
Up to 8 Weeks
Title
Changes in complement factor levels compared to baseline after drug administration
Time Frame
Up to 8 Weeks
Other Pre-specified Outcome Measures:
Title
Change from baseline in aqueous humor biomarker levels (in ng/mL) of complement factors
Time Frame
Up to 8 Weeks
Title
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
Title
Change from baseline in clinical assessments including area of GA in mm2 as assessed by fundus imaging
Time Frame
Up to 8 Weeks
Title
Change in concentration of GEM103 in blood samples
Time Frame
Up to 8 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
Associated Retina Consultants
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85020
Country
United States
Facility Name
Retinal Consultants of Arizona
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85053
Country
United States
Facility Name
California Retina Consultants
City
Bakersfield
State/Province
California
ZIP/Postal Code
93309
Country
United States
Facility Name
Retina Vitreous Associates
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
California Retina Consultants
City
Oxnard
State/Province
California
ZIP/Postal Code
93036
Country
United States
Facility Name
California Retina Consultants
City
Santa Barbara
State/Province
California
ZIP/Postal Code
93103
Country
United States
Facility Name
California Retina Consultants
City
Santa Maria
State/Province
California
ZIP/Postal Code
93454
Country
United States
Facility Name
Southeast Retina Center
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30909
Country
United States
Facility Name
Midwest Eye Institute
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46290
Country
United States
Facility Name
Pepose Vision Institute
City
Chesterfield
State/Province
Missouri
ZIP/Postal Code
63017
Country
United States
Facility Name
Sierra Eye Associates
City
Reno
State/Province
Nevada
ZIP/Postal Code
89502
Country
United States
Facility Name
Western Carolina Retinal Associates
City
Asheville
State/Province
North Carolina
ZIP/Postal Code
28803
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
36249705
Citation
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.
Results Reference
derived

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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

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