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

Sponsor

Nanoscope Therapeutics Inc. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05921162

Collaborator

(none)

Enrollment

Location

Anticipated Duration (Months)

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Retinitis Pigmentosa Retinal Disease Retinal Degeneration	Biological: Gene Therapy product:vMCO-I

Detailed 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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

11 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Anticipated Study Start Date :

Jun 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2024

Anticipated Study Completion Date :

Dec 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Observation of Participants exposed to 1.2E11gc/eye of vMCO-I 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.	Biological: Gene Therapy product:vMCO-I Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients
Observation of Participants exposed to 0.6E11gc/eye of vMCO-I 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.	Biological: Gene Therapy product:vMCO-I Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients

Arm

Intervention/Treatment

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

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.

Biological: Gene Therapy product:vMCO-I

Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients

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

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.

Biological: Gene Therapy product:vMCO-I

Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients

Outcome Measures

Primary Outcome Measures

Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I [20 Months]
Assessments of Incidences, nature, and severity of treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal anatomy
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I) [20 Months]
Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)

Secondary Outcome Measures

Assessment of the effect of vMCO-I on functional vision outcomes [20 Months]
Assessment of the treatment effect on the quality of Life with changes from baseline in activities of daily living using the National Eye Institute (NEI) Visual Function Questionnaire-25
Assessment of the durability of vMCO-I induced gene reporter expression [20 Months]
Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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.