SUSTAIN: Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
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 Design
Outcome Measures
Primary Outcome Measures
- 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
- 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
- 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
Eligibility Criteria
Criteria
Inclusion Criteria:
- 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.
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Nanocope Clnical Site | Miami | Florida | United States | 33136 |
| 2 | Nanoscope Clinical Site | McAllen | Texas | United States | 78503 |
Sponsors and Collaborators
- Nanoscope Therapeutics Inc.
Investigators
- Study Director: Dr. Samarendra Mohanty, Nanoscope Therapeutics Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- NTXLTFU-007