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

NCT05921162 | Enrolling By Invitation

• 1 other identifier: NTXMCO-005
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 1

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

Conditions

Retinitis Pigmentosa; Retinal Disease; Retinal Degeneration

Keywords

Optogenetics; Gene Therapy; AAV Vectors; Multicharacteristic Opsin (MCO)-I

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Assessment of the effect of vMCO-I on functional vision outcomes

  20 Months

• Assessment of the durability of vMCO-I induced gene reporter expression

  20 Months

Study Arms (2)

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

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

Interventions

Gene Therapy product:vMCO-I BIOLOGICAL

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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Subjects who will not consent for study.

Sponsors & Collaborators

• Nanoscope Therapeutics Inc.: lead

Study Sites (1)

JPM Rotary Club of Cuttack Eye Hospital and Research Institute

Cuttack, Odisha, 753014, India

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinal Diseases; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Samarendra Mohanty

  Nanoscope Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 19, 2023
• First Posted: June 27, 2023
• Study Start: July 14, 2023
• Primary Completion: May 1, 2025
• Study Completion: May 1, 2025
• Last Updated: March 25, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: Within a year from the long term monitoring data availability
• 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.

Locations

IN (1)