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

Status: Completed
Location: See all (2) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:

• ≥16 years of age

• Able to comprehend and give informed consent.

• Able to comply with testing and all protocol tests.

• 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 PROM1

• In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.

• Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening

Locations
United States
Florida
Nanoscope Clinical Site
Miami
Texas
Nanoscope Clinical Site
Mcallen
Time Frame
Start Date: 2022-07-05
Completion Date: 2023-09-28
Participants
Target number of participants: 6
Treatments
Experimental: Experimental-vMCO-010
Participants receive 1.2E11gc/eye of vMCO-010
Related Therapeutic Areas
Stargardt Macular Degeneration
Sponsors
Leads: Nanoscope Therapeutics Inc.

This content was sourced from clinicaltrials.gov

Similar Clinical Trials

An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)

An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)

Enrollment Status: Recruiting
Publish Date: December 12, 2025
Intervention Type: Biological
Study Phase: Phase 1/Phase 2

A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene

A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene

Enrollment Status: Recruiting
Publish Date: April 29, 2025
Intervention Type: Genetic
Study Phase: Phase 1/Phase 2

A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

Enrollment Status: Recruiting
Publish Date: December 05, 2025
Intervention Type: Drug
Study Phase: Phase 2/Phase 3
View All