NCT06162585

Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study

Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 18 (estimated)
• Sponsor: Nanoscope Therapeutics Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study

Detailed description

This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 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) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. 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 RESTORE and are not repeated herein. The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.

Conditions

• Retinitis Pigmentosa
• Retinitis
• Retinal Diseases
• Eye Diseases
• Eye Diseases, Hereditary
• Retinal Dystrophies
• Retinal Degeneration

Interventions

Type	Name	Description
BIOLOGICAL	Gene Therapy product-MCO-010	Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients

Timeline

• Start date: 2023-12-08
• Primary completion: 2027-07-01
• Completion: 2027-09-01
• First posted: 2023-12-08
• Last updated: 2025-03-24

Locations

6 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study