Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study (SUSTAIN)

Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study

The current study is a non-interventional long-term safety follow-up of the subjects who completed STARLIGHT, in accordance with FDA guidance on recipients of human gene therapy products.

Study Overview

• Status: Active, not recruiting
• Conditions: Stargardt Disease
• Intervention / Treatment: Biological: Gene Therapy Product-MCO-010

Detailed Description

This study is designed to follow subjects with Stargardt Disease (SD) previously enrolled in study NTXMCO-004 (STARLIGHT, NCT05417126). In that study, all 6 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT). MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for STARLIGHT and are not repeated herein

• Study Type: Observational
• Enrollment (Estimated): 6

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population eligible to participate will comprise of the 6 subjects previously enrolled in STARLIGHT.

Description

Inclusion Criteria:

• Subjects are eligible to be included in the study only if all of the following criteria apply:

  1. Previously enrolled in study NTXMCO-004 study
  2. Able to comprehend and give informed consent.
  3. Able to comply with testing and all protocol tests.
  4. Agree to participate for the full 4-year study duration to the best of their ability and barring any unforeseen circumstances.

Exclusion Criteria:

• Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at the Week 48 visit for the STARLIGHT study

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of the long-term safety profile of a single intravitreal injection of MCO-010	Assessment of incidences, nature, and severity of treatment emergent adverse events (TEAEs), serious adverse events (SAEs); intraocular inflammation graded through ocular exam; clinical examination, and intraocular pressure.	204 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the long-term effects on visual acuity of a single intravitreal injection of MCO-010; 1.2E11 gc/eye	Change from baseline in BCVA over time in the study eye and fellow eye	204 weeks
Exploration of the long-term impact of MCO-010 on retinal anatomy	Assessment of fundus photography and optical coherence tomography (OCT) outcomes over time	204 weeks
Assessment of the long-term durability of MCO-010 induced on gene reporter expression	Change from baseline of fundus fluorescence intensity of reporter (mCherry) over time in the study eye and fellow eye	204 weeks

Collaborators and Investigators

• Sponsor: Nanoscope Therapeutics Inc.
• Investigators: Study Director: Dr. Samarendra Mohanty, Nanoscope Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): October 19, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): July 1, 2027

Study Registration Dates

• First Submitted: September 11, 2023
• First Submitted That Met QC Criteria: September 15, 2023
• First Posted (Actual): September 21, 2023

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 21, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophy; Retinal Degeneration
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Eye Diseases; Eye Diseases, Hereditary; Retinal Diseases; Retinal Degeneration; Macular Degeneration; Stargardt Disease
• Other Study ID Numbers: NTXLTFU-007

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 a year from the long term monitoring data availability
• IPD Sharing Access Criteria: IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No