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

Nanoscope Therapeutics

Status: Active, not recruiting

Conditions: Eye Diseases, Hereditary; Retinal Degeneration; Retinal Dystrophies; Retinitis Pigmentosa; Retinitis; Eye Diseases; Retinal Diseases

Treatments: Biological: Gene Therapy product-MCO-010

Study type: Observational

Funder types: Industry

Identifiers

NCT06162585

NTXLTFU-008

Details and patient eligibility

About

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

Full 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.

Enrollment: 18 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Previously enrolled in study NTXMCO-002 (RESTORE)
• Able to comprehend and give informed consent.
• Able to comply with testing and all protocol tests.
• Agree to participate for the full 3-year duration of follow-up 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 their final RESTORE study visit

Trial design

18 participants in 2 patient groups

Observation of Participants exposed 1.2E11gc/eye of MCO-010

Description:

This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study.

Treatment:

Biological: Gene Therapy product-MCO-010

Observation of Participants exposed to 0.9E11gc/eye of MCO-010

Description:

This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study.

Treatment:

Biological: Gene Therapy product-MCO-010