Stargardt DiseaseSUSTAINNCT06048185
Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study
An observational study of Stargardt Disease, run by Nanoscope Therapeutics Inc. at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- Gene Therapy Product-MCO-010
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 19 Oct 2023to Jul 2027
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population eligible to participate will comprise of the 6 subjects previously enrolled in STARLIGHT.
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Nanoscope Clinical Site
McAllen, Texas, 78503
Nanocope Clnical Site
Miami, Florida, 33136
About this 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
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.
Read the 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
- Conditions
- Stargardt Disease
- Being tested
- Gene Therapy Product-MCO-010
- Keywords
- Eye Diseases, Retinal Degeneration, Retinal Dystrophy, Eye Diseases, Hereditary
- Medical subject headings
- Stargardt Disease, Eye Diseases, Retinal Degeneration, Retinal Dystrophies, Eye Diseases, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy Product-MCO-010Biological
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Change from baseline in BCVA over time in the study eye and fellow eye
Measured: 204 weeks
Exploration of the long-term impact of MCO-010 on retinal anatomy
Assessment of fundus photography and optical coherence tomography (OCT) outcomes over time
Measured: 204 weeks
Assessment of the long-term durability of MCO-010 induced on gene reporter expression
Change from baseline of fundus fluorescence intensity of reporter (mCherry) over time in the study eye and fellow eye
Measured: 204 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2023
- Study start19 Oct 2023
- Primary completionMay 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dr. Samarendra Mohanty, Study director, Nanoscope Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06048185
- Sponsor's protocol ID
- NTXLTFU-007
- Acronym
- SUSTAIN
- Status last verified
- Mar 2025
- First submitted
- 11 Sep 2023
- Last updated
- 24 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.