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

Retinitis Pigmentosa Clinical Trial

— EXTEND  

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05921162
• Other study ID #: NTXMCO-005
• Status: Enrolling by invitation
• Start date: July 14, 2023
• Est. completion date: December 2024

Study information

• Verified date: July 2023
• Source: Nanoscope Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 11
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Retinal Degeneration
• Retinal Disease
• Retinal Diseases
• Retinitis
• Retinitis Pigmentosa

Intervention

Biological:
• Gene Therapy product:vMCO-I
Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients

Locations

Country	Name	City	State
India	JPM Rotary Club of Cuttack Eye Hospital and Research Institute	Cuttack	Odisha

Sponsors (1)

Lead Sponsor	Collaborator
Nanoscope Therapeutics Inc.

Country where clinical trial is conducted

India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	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
Secondary	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