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

Nanoscope Therapeutics

Status: Active, not recruiting

Conditions: Stargardt Disease

Treatments: Biological: Gene Therapy Product-MCO-010

Study type: Observational

Funder types: Industry

Identifiers

NCT06048185

NTXLTFU-007

Details and patient eligibility

About

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.

Full 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

Enrollment: 6 estimated patients

Sex: All

Ages: 16+ years old

Volunteers: No Healthy Volunteers

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