A Phase I/IIa Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa

Nanoscope Therapeutics Inc.1 site in 1 country11 target enrollmentOctober 23, 2019

ConditionsRetinitis Pigmentosa Retinal Diseases Retinal Degeneration

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Retinitis Pigmentosa
Sponsor: Nanoscope Therapeutics Inc.
Enrollment: 11
Locations: 1
Primary Endpoint: The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
Status: Completed
Last Updated: 4 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)

Detailed Description

This open label dose-escalation study evaluated 2 dose levels in up to 11 subjects of retinitis pigmentosa (3 in low dose and 8 in high dose per dose) with active vMCO-010. Subjects with confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination and dilated fundus examination, were considered for participation in this study. The primary endpoint for this study is safety and tolerability of vMCO-I at 16 weeks. All subjects were assessed for 52 weeks following treatment with vMCO-I

Registry

clinicaltrials.gov

Start Date

October 23, 2019

End Date

October 31, 2020

Last Updated

4 years ago

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Nanoscope Therapeutics Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Age \> 18 years
•Diagnosis of advanced RP using Fundus Photographs
•Clinical diagnosis of advanced retinal dystrophy
•Prior documented (if any) retinal electrophysiological evidence of rod-cone photoreceptor degeneration
•Snellen's visual acuity equivalent LP/NLP in worse (study) eye
•Visual acuity in the non-study eye of no-better-than finger counting
•Presence of retinal bipolar cells and retinal nerve fiber layer on OCT testing

Exclusion Criteria

•Prior participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy or any gene or stem cell therapy in the past six months.
•Concurrent participation in another interventional clinical ocular study.
•Pre-existing eye conditions such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).
•Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
•Subjects who are positive for hepatitis B, C, and HIV will be excluded.
•Subjects who have undergone ocular surgery in the study eye within three months prior to Day
•Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
•Known sensitivity to any component of the study agent or medications planned for use in the peri-operative period.
•Subjects will be excluded if immunological studies show presence of neutralizing antibodies to AAV2 above 1:
•Presence of narrow iridocorneal angles contraindicating pupillary dilation.

Outcomes

Primary Outcomes

The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa

Time Frame: 16 Weeks

Safety and tolerability of vMCO-l treatment at Week 16, by assessments based on local and systemic safety issues, as assessed by incidence of Adverse Events.

Secondary Outcomes

Evaluate the treatment effect of vMCO-l as assessed by visual acuity(52 weeks)
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay(52 weeks)
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays(52 weeks)
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay(52 weeks)
Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire(52 weeks)
Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field(52 weeks)