Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease (STARLIGHT)
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ClinicalTrials.gov Identifier: NCT05417126 |
Recruitment Status :
Completed
First Posted : June 14, 2022
Last Update Posted : November 9, 2023
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Sponsor:
Nanoscope Therapeutics Inc.
Information provided by (Responsible Party):
Nanoscope Therapeutics Inc.
Tracking Information | |||||||||
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First Submitted Date ICMJE | June 9, 2022 | ||||||||
First Posted Date ICMJE | June 14, 2022 | ||||||||
Last Update Posted Date | November 9, 2023 | ||||||||
Actual Study Start Date ICMJE | July 5, 2022 | ||||||||
Actual Primary Completion Date | August 2, 2023 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
Type, severity, and incidence of ocular and systemic adverse events (AEs) [ Time Frame: 48 weeks ] Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010
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Original Primary Outcome Measures ICMJE | Same as current | ||||||||
Change History | |||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease | ||||||||
Official Title ICMJE | A Phase 2a, Open Label Multicenter Clinical Trial to Evaluate the Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease | ||||||||
Brief Summary | The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease | ||||||||
Detailed Description | This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010. | ||||||||
Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 2 | ||||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: 6 subjects will be enrolled for vMCO-010 treatment Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | Stargardt Disease | ||||||||
Intervention ICMJE | Biological: Gene Therapy-vMCO-010
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
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Study Arms ICMJE | Experimental: Experimental-vMCO-010
Participants receive 1.2E11gc/eye of vMCO-010
Intervention: Biological: Gene Therapy-vMCO-010
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Completed | ||||||||
Actual Enrollment ICMJE |
6 | ||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||
Actual Study Completion Date ICMJE | September 28, 2023 | ||||||||
Actual Primary Completion Date | August 2, 2023 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 16 Years and older (Child, Adult, Older Adult) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||
Listed Location Countries ICMJE | United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number ICMJE | NCT05417126 | ||||||||
Other Study ID Numbers ICMJE | NTXMCO-004 | ||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Nanoscope Therapeutics Inc. | ||||||||
Original Responsible Party | Same as current | ||||||||
Current Study Sponsor ICMJE | Nanoscope Therapeutics Inc. | ||||||||
Original Study Sponsor ICMJE | Same as current | ||||||||
Collaborators ICMJE | Not Provided | ||||||||
Investigators ICMJE |
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PRS Account | Nanoscope Therapeutics Inc. | ||||||||
Verification Date | November 2023 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |