Eye Diseases, Eye Diseases, Hereditary, Retinal Degeneration, Retinal Diseases, Retinal Dystrophies, Retinitis, Retinitis Pigmentosa
Conditions
Keywords
Gene Therapy, AAV Vectors, Low Vision, Intravitreal Injections, Optogenetics, Multi-Characteristic Opsin, Multi-Luminance Y Mobility Test, Multi-Luminance Shape Discrimination Test, Visual Acuity
Brief summary
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study
Detailed description
This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for RESTORE and are not repeated herein. The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.
Interventions
BIOLOGICALGene Therapy product-MCO-010
Safety evaluation to monitor long term effects of previously injected MCO-010 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
* Previously enrolled in study NTXMCO-002 (RESTORE) * Able to comprehend and give informed consent. * Able to comply with testing and all protocol tests. * Agree to participate for the full 3-year duration of follow-up to the best of their ability and barring any unforeseen circumstances.
Exclusion criteria
* Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at their final RESTORE study visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010 | 156 weeks | Delayed adverse events. Incidence, nature, and severity of selected adverse events (AEs); all serious adverse events (SAEs); all ocular AEs including intraocular inflammation graded through ocular exam; non-ocular AEs with a common terminology criteria for adverse events (CTCAE) grade of 3 or greater; AEs of special interest (AESIs) including new malignancies, new incidence or exacerbation of any pre-existing neurologic disorder or rheumatologic or other autoimmune disorder, new incidence of hematologic disorder or new infection regardless of suspected relatedness to treatment with MCO-010. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010 | 156 Weeks | Change from baseline in BCVA over time in both eyes |
| Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010 | 156 Weeks | Change from baseline in multi-luminance shape discrimination test (MLSDT) scores |
| Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010 | 156 Weeks | Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score |
| Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy | 156 Weeks | Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time |
Other
| Measure | Time frame | Description |
|---|---|---|
| Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression | 156 Weeks | PK parameters including change from baseline of fundus fluorescence intensity of reporter over time and PD correlation of gene expression with the efficacy measures in the study eye and fellow eye (selected sites) |
Countries
Puerto Rico, United States
Outcome results
None listed