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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04945772
Other study ID # NTXMCO-002.
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date July 13, 2021
Est. completion date January 18, 2024

Study information

Verified date March 2024
Source Nanoscope Therapeutics Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).


Description:

This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.


Recruitment information / eligibility

Status Completed
Enrollment 27
Est. completion date January 18, 2024
Est. primary completion date February 27, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: 1. Age = 18 years 2. Able to comprehend and give informed consent. 3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing. 4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening. Exclusion Criteria: Subjects are excluded from the study if any of the following criteria apply: 1. Prior participation in gene therapy program 2. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities). 3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function. 4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. 5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).

Study Design


Intervention

Biological:
Gene Therapy Product-MCO-010
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Procedure:
Sham Injection
Sham Injection

Locations

Country Name City State
Puerto Rico Nanoscope Clinical Site Arecibo
United States Nanoscope Clinical Site Beverly Hills California
United States Nanoscope Clinical Site Fargo North Dakota
United States Nanoscope Clinical Site Houston Texas
United States Nanoscope Clinical Site McAllen Texas
United States Nanoscope Clinical Site Pensacola Florida

Sponsors (1)

Lead Sponsor Collaborator
Nanoscope Therapeutics Inc.

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type Measure Description Time frame Safety issue
Other Efficacy of MCO-010 as assessed by a composite of functional assessments. Proportion of subjects demonstrating a =2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52. Week 52
Other Safety of MCO-010. Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs). 100 weeks
Primary Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity. Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52. Week 52
Secondary Efficacy of MCO-010 as assessed by best corrected visual acuity. Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76. Week 76
Secondary Efficacy of MCO-010 as assessed by mobility testing. Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome. Weeks 16,24,32,52,76,100
Secondary Efficacy of MCO-010 as assessed by mobility testing. Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome. Weeks 16,24,32,52,76,100
Secondary Efficacy of MCO-010 as assessed by static shape recognition assay. Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome. Weeks 16,24,32,52,76,100
Secondary Efficacy of MCO-010 as assessed by static shape recognition assay. Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome. Weeks 16,24,32,52,76,100
Secondary Efficacy of MCO-010 as assessed on visual field. Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry. Weeks 16,24,32,52,76,100
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