Dry Age-related Macular DegenerationPhase 1NCT04246866
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 study testing GEM103 in people with Dry Age-related Macular Degeneration and Geographic Atrophy, run by Gemini Therapeutics, Inc. at 12 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration
- What is being tested?
- GEM103
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 12 sitesUnited States
- Study dates
- 19 Dec 2019to 21 Oct 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- 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
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Western Carolina Retinal Associates
Asheville, North Carolina, 28803
Southeast Retina Center
Augusta, Georgia, 30909
California Retina Consultants
Bakersfield, California, 93309
Retina Vitreous Associates
Beverly Hills, California, 90211
Pepose Vision Institute
Chesterfield, Missouri, 63017
Midwest Eye Institute
Indianapolis, Indiana, 46290
California Retina Consultants
Oxnard, California, 93036
Associated Retina Consultants
Phoenix, Arizona, 85020
Retinal Consultants of Arizona
Phoenix, Arizona, 85053
Sierra Eye Associates
Reno, Nevada, 89502
California Retina Consultants
Santa Barbara, California, 93103
California Retina Consultants
Santa Maria, California, 93454
About this study
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
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.
Read the 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.
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration, Retinal Disease, Retinal Degeneration
- Being tested
- GEM103
- Medical subject headings
- Geographic Atrophy, Macular Degeneration, Retinal Diseases, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GEM103Biological
GEM103
| Study group | Role | What they receive |
|---|---|---|
| Dose 1 | Experimental | A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103 Receives: Biological: GEM103 |
| Dose 2 | Experimental | A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103 Receives: Biological: GEM103 |
| Dose 3 | Experimental | A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103 Receives: Biological: 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 Receives: Biological: GEM103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of AEs/SAEs following drug administration
Measured: Up to 8 Weeks
Secondary outcomes (2)
GEM103 concentrations in ocular fluids
Measured: Up to 8 Weeks
Changes in complement factor levels compared to baseline after drug administration
Measured: Up to 8 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2020
- Study start19 Dec 2019
- Primary completion21 Oct 2020
- Study completion21 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dry Age-related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gemini Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04246866
- Sponsor's protocol ID
- GEM-CL-10301
- Status last verified
- Feb 2021
- First submitted
- 6 Jan 2020
- Last updated
- 11 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 36249705
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04246866
- Acronym / sponsor ID
- GEM-CL-10301
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Macular Degeneration, Retinal Disease, Retinal Degeneration
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Gemini Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Dec 2019 (actual)
- Primary completion
- 21 Oct 2020 (actual)
- Study completion
- 21 Oct 2020 (actual)
- First posted
- 29 Jan 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dose 1 | Experimental | Biological: GEM103A single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103 |
| Dose 2 | Experimental | Biological: GEM103A single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103 |
| Dose 3 | Experimental | Biological: GEM103A single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103 |
| Dose 4 | Experimental | Biological: GEM103A single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103 |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (10 listed)
- Presence of the following ocular conditions - in the study eye:
- Presence of any of the following ocular conditions - in either 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GEM103GEM103 | Biological | Dose 1, Dose 2, Dose 3, Dose 4 |
Primary outcome
Incidence and severity of AEs/SAEs following drug administration
Time frame: Up to 8 Weeks
2 secondary outcomes
GEM103 concentrations in ocular fluids
Time frame: Up to 8 Weeks
Changes in complement factor levels compared to baseline after drug administration
Time frame: Up to 8 Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2021.
- Derived from this study: PubMed 36249705Khanani 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GEM103 in people with Dry Age-related Macular Degeneration and Geographic Atrophy, run by Gemini Therapeutics, Inc. at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of AEs/SAEs following drug administration (time frame: Up to 8 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04246866, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2021.