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

Stargardt Disease Clinical Trial

— SUSTAIN  

Official title:

Long Term Follow-up for Participants Who Previously Received a Single Intravitreal Injection of MCO-010 Optogenetic Therapy in the STARLIGHT Study

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06048185
• Other study ID #: NTXLTFU-007
• Status: Enrolling by invitation
• Start date: October 19, 2023
• Est. completion date: July 2027

Study information

• Verified date: November 2023
• Source: Nanoscope Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 6
• Est. completion date: July 2027
• Est. primary completion date: May 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Stargardt Disease

Intervention

Biological:
• Gene Therapy Product-MCO-010
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Locations

Country	Name	City	State
United States	Nanoscope Clinical Site	McAllen	Texas
United States	Nanocope Clnical Site	Miami	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Nanoscope Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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	204 weeks
Secondary	Exploration of the long-term impact of MCO-010 on retinal anatomy	Assessment of fundus photography and optical coherence tomography (OCT) outcomes over time	204 weeks
Secondary	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	204 weeks