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

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

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

Study Overview

• Status: Enrolling by invitation
• Conditions: Retinal Degeneration; Retinitis Pigmentosa; Retinal Disease
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Estimated): 11

Contacts and Locations

Study Locations

• India
  • Odisha
    • Cuttack, Odisha, India, 753014
      • JPM Rotary Club of Cuttack Eye Hospital and Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

It is expected that a total of 11 study subjects from NSCT/CT/18/01 study will be enrolled. Subjects who meet all the inclusion criteria and for whom none of the exclusion criteria apply will be eligible for enrollment.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I	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	20 Months
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I)	Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)	20 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of the effect of vMCO-I on functional vision outcomes	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	20 Months
Assessment of the durability of vMCO-I induced gene reporter expression	Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry)	20 Months

Collaborators and Investigators

• Sponsor: Nanoscope Therapeutics Inc.
• Investigators: Study Director: Samarendra Mohanty, Nanoscope Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): July 14, 2023
• Primary Completion (Estimated): December 1, 2024
• Study Completion (Estimated): December 1, 2024

Study Registration Dates

• First Submitted: June 19, 2023
• First Submitted That Met QC Criteria: June 19, 2023
• First Posted (Actual): June 27, 2023

Study Record Updates

• Last Update Posted (Actual): August 2, 2023
• Last Update Submitted That Met QC Criteria: July 31, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Keywords: Gene Therapy; AAV Vectors; Optogenetics; Multicharacteristic Opsin (MCO)-I
• Additional Relevant MeSH Terms: Eye Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Diseases; Retinitis; Retinitis Pigmentosa; Retinal Degeneration
• Other Study ID Numbers: NTXMCO-005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The results of the clinical trial will be made available when the study is completed and results are analyzed. The results will be published on this site and be available to conference presentations and publications.
• IPD Sharing Time Frame: Within a year from the long term monitoring data availability
• IPD Sharing Access Criteria: IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No