Retinal Degeneration, Retinal Disease, Retinitis Pigmentosa
Conditions
Keywords
Optogenetics, Gene Therapy, AAV Vectors, Multicharacteristic Opsin (MCO)-I
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
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.
Interventions
BIOLOGICALGene Therapy product:vMCO-I
Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients
Sponsors
Nanoscope Therapeutics Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
Countries
India
Outcome results
None listed