Clinical trial NCT06048185: Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study
Official title: Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Stargardt Disease
Keywords: Eye Diseases
Retinal Degeneration
Retinal Dystrophy
Eye Diseases, Hereditary
Lead sponsor: Nanoscope Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2023-09-21
Enrollment: 6
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population eligible to participate will comprise of the 6 subjects previously enrolled in STARLIGHT.

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

Eligibility criteria: 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

Primary outcomes:
measure: Assessment of the long-term safety profile of a single intravitreal injection of MCO-010
timeFrame: 204 weeks
Secondary outcomes:
measure: Evaluation of the long-term effects on visual acuity of a single intravitreal injection of MCO-010; 1.2E11 gc/eye
timeFrame: 204 weeks
measure: Exploration of the long-term impact of MCO-010 on retinal anatomy
timeFrame: 204 weeks
measure: Assessment of the long-term durability of MCO-010 induced on gene reporter expression
timeFrame: 204 weeks
Other outcomes:
Locations:
city: Miami
country: United States
facility: Nanocope Clnical Site
state: Florida
city: McAllen
country: United States
facility: Nanoscope Clinical Site
state: Texas
Study officials:
affiliation: Nanoscope Therapeutics Inc.
name: Dr. Samarendra Mohanty
role: STUDY_DIRECTOR
References: