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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
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
| |
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Gene Therapy product:vMCO-I | Biological | Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients |
|
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Samarendra Mohanty | Nanoscope Therapeutics Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| JPM Rotary Club of Cuttack Eye Hospital and Research Institute | Cuttack | Odisha | 753014 | India |
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.
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Within a year from the long term monitoring data availability
IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| D012164 | Retinal Diseases |
| D012162 | Retinal Degeneration |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D030342 | Genetic Diseases, Inborn |
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Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry) |
| 20 Months |
| D009358 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities |