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

NCT06048185 | Active Not Recruiting DMC FDA Drug

• 1 other identifier: NTXLTFU-007
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 2

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.

Conditions

Stargardt Disease

Keywords

Eye Diseases; Retinal Degeneration; Retinal Dystrophy; Eye Diseases, Hereditary

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

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

  204 weeks

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

  204 weeks

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

  204 weeks

Interventions

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

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Nanoscope Therapeutics Inc.: lead

Study Sites (2)

Nanocope Clnical Site

Miami, Florida, 33136, United States

Location

Nanoscope Clinical Site

McAllen, Texas, 78503, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood

MeSH Terms

Conditions

Stargardt Disease; Eye Diseases; Retinal Degeneration; Retinal Dystrophies; Eye Diseases, Hereditary

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Dr. Samarendra Mohanty

  Nanoscope Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2023
• First Posted: September 21, 2023
• Study Start: October 19, 2023
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 24, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: Within a year from the long term monitoring data availability
• 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.

Locations

US (2)