Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa

NCT04919473 | Completed Phase 1

• 1 other identifier: NSCT/CT/18/01
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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)

Conditions

Retinitis Pigmentosa; Retinal Diseases; Retinal Degeneration

Keywords

Retinitis Pigmentosa; Retinal Degeneration; Optogenetics; Gene Therapy; AAV vectors; Multicharacteristic Opsin (MCO)-I

Outcome Measures

Primary Outcomes (1)

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

  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.

  16 Weeks

Secondary Outcomes (9)

• Evaluate the treatment effect of vMCO-l as assessed by visual acuity

  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 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 Static Shape recognition assay

  52 weeks

Study Arms (2)

vMCO-I High dose

EXPERIMENTAL

Participants received 3.5E11vg/eye of vMCO-I

Biological: Gene Therapy product:vMCO-I

vMCO-I Low Dose

EXPERIMENTAL

Participants received 1.75E11vg/eye of vMCO-I

Biological: Gene Therapy product:vMCO-I

Interventions

Gene Therapy product:vMCO-I BIOLOGICAL

The vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

vMCO-I High dose; vMCO-I Low Dose

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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 0.
• 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 1:1000.
• Presence of narrow iridocorneal angles contraindicating pupillary dilation.
• Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including OCT, during the study period.
• Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by OCT examinations and assessed by the investigator to significantly affect central vision.
• Current evidence of retinal detachment assessed by the investigator to significantly affect central vision.
• Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

Sponsors & Collaborators

• Nanoscope Therapeutics Inc.: lead

Study Sites (1)

JPM Rotary Club of Cuttack Eye Hospital and Research Institute

Cuttack, Odisha, 753014, India

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinal Diseases; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Santosh Mahapatra, MD

  JPM Rotary Club of Cuttack Eye Hospital and Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 25, 2021
• First Posted: June 9, 2021
• Study Start: October 23, 2019
• Primary Completion: February 25, 2020
• Study Completion: October 31, 2020
• Last Updated: June 9, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will share
• Time Frame: 6 months after the completion of the study
• Access Criteria: IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.

Locations

IN (1)