Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease (STARLIGHT)

November 8, 2023 updated by: Nanoscope Therapeutics Inc.

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Nanoscope Clinical Site
    • Texas
      • McAllen, Texas, United States, 78503
        • Nanoscope Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. ≥16 years of age
  2. Able to comprehend and give informed consent.
  3. Able to comply with testing and all protocol tests.
  4. 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
  5. 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.
  6. Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening

Exclusion Criteria:

  1. 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).
  2. 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.
  3. 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
  4. Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
  5. Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
  6. Resting heart rate outside specified limits upon repeated measurement.
  7. History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
  8. Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
  9. Any major surgical procedure within one month of trial entry or anticipated during the trial.
  10. Clinically significant abnormal lab results at screening
  11. 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
  12. In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
  13. 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).
  14. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
  15. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..
  16. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
  17. 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.
  18. Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
  19. Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
  20. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
  21. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Experimental-vMCO-010
Participants receive 1.2E11gc/eye of vMCO-010
Biological: Gene Therapy-vMCO-010
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
48 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Effect of vMCO-010 as assessed by visual acuity
Time Frame: 48 weeks
Change from baseline in BCVA at Weeks 12, 24, 48 in the study eye and the fellow eye
48 weeks
Effect of vMCO-010 on Light-guided Mobility
Time Frame: 48 Weeks
Change from baseline in Multi-Luminance Mobility Test at weeks 12, 24, 48 in the study eye and the fellow eye
48 Weeks
Effect of vMCO-010 on determination of shape
Time Frame: 48 Weeks
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
48 Weeks
Effect of vMCO-010 on determination of optical flow
Time Frame: 48 Weeks
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
48 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Nanoscope Therapeutics Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Dr Samarendra Mohanty, Nanoscope Therapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 5, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 2, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 28, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 9, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 9, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 14, 2022

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 9, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 8, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NTXMCO-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

IPD Sharing Time Frame

Within 12 Months of study completion

IPD Sharing Access Criteria

Safety and efficacy Results

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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