Stargardt DiseasePhase 2STARLIGHTNCT05417126
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
A Phase 2 study testing Gene Therapy-vMCO-010 in people with Stargardt Disease, run by Nanoscope Therapeutics Inc. at 2 sites in United States.
Trial summary
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- Gene Therapy-vMCO-010
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 5 Jul 2022to 28 Sep 2023
- Phase
- Phase 2
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- ≥16 years of age
- Able to comprehend and give informed consent.
- Able to comply with testing and all protocol tests.
- Documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1
- In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.
- Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening
Who cannot join
- Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
- Received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as ARGUS-II) or sub-retinal injections.
- Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
- Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
- Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
- Resting heart rate outside specified limits upon repeated measurement.
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- History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
- Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
- Any major surgical procedure within one month of trial entry or anticipated during the trial.
- Clinically significant abnormal lab results at screening
- Known serious allergies to the fluorescein dye used in angiography or intraocular pressure measurement, povidone iodine, or to the components of the vMCO-010 formulation
- In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
- Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, history of uveitis, corneal or lenticular opacities).
- Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
- Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..
- Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
- Presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including OCT, during the study period.
- Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
- Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
- Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Nanoscope Clinical Site
McAllen, Texas, 78503
Nanoscope Clinical Site
Miami, Florida, 33136
About this study
Official title: A Phase 2a, Open Label Multicenter Clinical Trial to Evaluate the Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
Read the detailed description
This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010.
- Conditions
- Stargardt Disease
- Being tested
- Gene Therapy-vMCO-010
- Keywords
- Eye Diseases, Retinal Degeneration, Retinal Dystrophy, Eye Diseases, Hereditary
- Medical subject headings
- Stargardt Disease, Eye Diseases, Retinal Degeneration, Retinal Dystrophies, Eye Diseases, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy-vMCO-010Biological
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
| Study group | Role | What they receive |
|---|---|---|
| Experimental-vMCO-010 | Experimental | Participants receive 1.2E11gc/eye of vMCO-010 Receives: Biological: Gene Therapy-vMCO-010 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Type, severity, and incidence of ocular and systemic adverse events (AEs)
Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010
Measured: 48 weeks
Secondary outcomes (4)
Effect of vMCO-010 as assessed by visual acuity
Change from baseline in BCVA at Weeks 12, 24, 48 in the study eye and the fellow eye
Measured: 48 weeks
Effect of vMCO-010 on Light-guided Mobility
Change from baseline in Multi-Luminance Mobility Test at weeks 12, 24, 48 in the study eye and the fellow eye
Measured: 48 Weeks
Effect of vMCO-010 on determination of shape
Change from baseline in accuracy in determination of shape using Low Vision Multi-Parameter Test (LVMPT) at weeks 12, 24, 48 in the study eye and the fellow eye
Measured: 48 Weeks
Effect of vMCO-010 on determination of optical flow
Change from baseline in accuracy in determination of optical flow using the LVMPT at weeks 12, 24 and 48 in the study eye and the fellow eye
Measured: 48 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2022
- Study start5 Jul 2022
- Primary completion2 Aug 2023
- Study completion28 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 6 subjects will be enrolled for vMCO-010 treatment
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dr Samarendra Mohanty, Study director, Nanoscope Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05417126
- Sponsor's protocol ID
- NTXMCO-004
- Acronym
- STARLIGHT
- Status last verified
- Nov 2023
- First submitted
- 9 Jun 2022
- Last updated
- 9 Nov 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.
Study at a glance
From the registry record, last updated by the sponsor on 9 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Open Label Multicenter Clinical Trial to Evaluate the Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
- ClinicalTrials.gov ID
- NCT05417126
- Acronym / sponsor ID
- STARLIGHT / NTXMCO-004
- Conditions
- Stargardt Disease
- Keywords
- Eye Diseases, Retinal Degeneration, Retinal Dystrophy, Eye Diseases, Hereditary
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Nanoscope Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jul 2022 (actual)
- Primary completion
- 2 Aug 2023 (actual)
- Study completion
- 28 Sep 2023 (actual)
- First posted
- 14 Jun 2022 (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 purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
Detailed description
This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 6 subjects will be enrolled for vMCO-010 treatment
| Arm | Type | Receives |
|---|---|---|
| Experimental-vMCO-010 | Experimental | Biological: Gene Therapy-vMCO-010Participants receive 1.2E11gc/eye of vMCO-010 |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- ≥16 years of age
- Able to comprehend and give informed consent.
- Able to comply with testing and all protocol tests.
- Documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1
- In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.
Key exclusion criteria (21 listed)
- Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
- Received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as ARGUS-II) or sub-retinal injections.
- Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
- Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
- Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
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 |
|---|---|---|
| Gene Therapy-vMCO-010The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette | Biological | Experimental-vMCO-010 |
Primary outcome
Type, severity, and incidence of ocular and systemic adverse events (AEs)
Time frame: 48 weeks
Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010
4 secondary outcomes
Effect of vMCO-010 as assessed by visual acuity
Time frame: 48 weeks
Effect of vMCO-010 on Light-guided Mobility
Time frame: 48 Weeks
Effect of vMCO-010 on determination of shape
Time frame: 48 Weeks
Effect of vMCO-010 on determination of optical flow
Time frame: 48 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 9 Nov 2023.
- Individual participant dataSharing planned: Yes. The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.
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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.
Dr Samarendra Mohanty study director
Nanoscope Therapeutics Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Gene Therapy-vMCO-010 in people with Stargardt Disease, run by Nanoscope Therapeutics Inc. at 2 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 6 inclusion and 21 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 9 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Type, severity, and incidence of ocular and systemic adverse events (AEs) (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Nov 2023.