A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection (EXTEND)

Nanoscope Therapeutics

Status: Invitation-only

Conditions: Retinal Degeneration; Retinitis Pigmentosa; Retinal Disease

Treatments: Biological: Gene Therapy product:vMCO-I

Study type: Observational

Funder types: Industry

Identifiers

NCT05921162

NTXMCO-005

Details and patient eligibility

About

This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection

Full description

The observational study population comprises of individuals who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection in a parent study.

Enrolled subjects will undergo safety and efficacy assessments for up to 5 years from the vMCO-I administration. At the visits, subjects will have safety and efficacy evaluations. Subjects will have a final visit at 60 months post study drug administration.

Enrollment: 11 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• The subject population includes subjects who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I), administered via intravitreal injection in a parent study.

Exclusion criteria

• Subjects who will not consent for study.

Trial design

11 participants in 2 patient groups

Observation of Participants exposed to 1.2E11gc/eye of vMCO-I

Description:

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

Treatment:

Biological: Gene Therapy product:vMCO-I

Observation of Participants exposed to 0.6E11gc/eye of vMCO-I

Description:

This is a long-term follow-up observational study of participants who previously received 0.6E11gc/eye of vMCO-I No investigational product will be administered in this study.

Treatment:

Biological: Gene Therapy product:vMCO-I