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NCT Number: NCT06048185

Non-interventional Long Term Follow-up Study of Participants Previously Enrolled 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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Stargardt Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases Retinal Dystrophies

Age range

16 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Nanocope Clnical Site, Miami, Florida, United States

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subjects are eligible to be included in the study only if all of the following criteria apply:
  • Previously enrolled in study NTXMCO-004 study
  • Able to comprehend and give informed consent.
  • Able to comply with testing and all protocol tests.
  • 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

Treatment and study plan

Gene Therapy Product-MCO-010

Biological

The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Primary outcomes

  1. Assessment of the long-term safety profile of a single intravitreal injection of MCO-010

    Time frame: 204 weeks

    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.

Secondary outcomes

  1. Evaluation of the long-term effects on visual acuity of a single intravitreal injection of MCO-010; 1.2E11 gc/eye

    Time frame: 204 weeks

    Change from baseline in BCVA over time in the study eye and fellow eye

  2. Exploration of the long-term impact of MCO-010 on retinal anatomy

    Time frame: 204 weeks

    Assessment of fundus photography and optical coherence tomography (OCT) outcomes over time

  3. Assessment of the long-term durability of MCO-010 induced on gene reporter expression

    Time frame: 204 weeks

    Change from baseline of fundus fluorescence intensity of reporter (mCherry) over time in the study eye and fellow eye

Sponsors and collaborators

Lead sponsor

Nanoscope Therapeutics Inc.

Industry

Registry information

Official study title

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

Acronym: SUSTAIN

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Sep 21, 2023
Registry last updated
Mar 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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