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

Active, not recruiting

• Conditions: Stargardt Disease

• Registration Number: NCT06048185
• Lead Sponsor: Nanoscope Therapeutics Inc.

Brief Summary

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.

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 Timeline

• Study First Submitted: 2023-09-11
• Study First Submitted QC: 2023-09-15
• Study First Posted: 2023-09-21
• Study Start: 2023-10-19
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-21
• Last Update Posted: 2025-03-24
• Primary Completion: 2027-05
• Study Completion: 2027-07

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 6

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessment of the long-term safety profile of a single intravitreal injection of MCO-010	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 Outcome Measures

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

Trial Locations

Locations (2)

Nanocope Clnical Site; 🇺🇸 Miami, Florida, United States

Nanoscope Clinical Site; 🇺🇸 McAllen, Texas, United States