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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
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Observation of Participants exposed 1.2E11gc/eye of MCO-010 | This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study. |
| |
| Observation of Participants exposed to 0.9E11gc/eye of MCO-010 | This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Gene Therapy product-MCO-010 | Biological | Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients |
|
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010 | 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. | 156 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010 | Change from baseline in BCVA over time in both eyes | 156 Weeks |
| Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010 |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression | 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) | 156 Weeks |
Inclusion Criteria:
Exclusion Criteria:
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This study population will be comprised of the 18 adult subjects with advanced retinitis pigmentosa, previously dosed with MCO-010 in the RESTORE study.
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| Name | Affiliation | Role |
|---|---|---|
| Samuel Barone, MD | Nanoscope Therapeutics Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nanoscope Clinical Site | Beverly Hills | California | 90211 | United States | ||
| Nanoscope Clinical Site |
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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Change from baseline in multi-luminance shape discrimination test (MLSDT) scores |
| 156 Weeks |
| Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010 | Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score | 156 Weeks |
| Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy | Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time | 156 Weeks |
| Pensacola |
| Florida |
| 32503 |
| United States |
| Nanoscope Clinical Site | Fargo | North Dakota | 58103 | United States |
| Nanoscope Clinical Site | Houston | Texas | 77030 | United States |
| Nanoscope Clinical Site | McAllen | Texas | 78503 | United States |
| Nanoscope Clinical Site | Arecibo | 00612 | Puerto Rico |
| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| D012173 | Retinitis |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D015354 | Vision, Low |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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