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

NCT ID: NCT04919473

Last Updated: 2021-06-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-23
• Study Completion Date: 2020-10-31

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

Conditions

Retinitis Pigmentosa; Retinal Diseases; Retinal Degeneration

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

vMCO-I High dose

Participants received 3.5E11vg/eye of vMCO-I

Group Type: EXPERIMENTAL

Gene Therapy product:vMCO-I

Intervention Type: BIOLOGICAL

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

vMCO-I Low Dose

Participants received 1.75E11vg/eye of vMCO-I

Group Type: EXPERIMENTAL

Gene Therapy product:vMCO-I

Intervention Type: BIOLOGICAL

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

Interventions

Gene Therapy product:vMCO-I

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Age > 18 years

2. Diagnosis of advanced RP using Fundus Photographs

3. Clinical diagnosis of advanced retinal dystrophy

4. Prior documented (if any) retinal electrophysiological evidence of rod-cone photoreceptor degeneration

5. Snellen's visual acuity equivalent LP/NLP in worse (study) eye

6. Visual acuity in the non-study eye of no-better-than finger counting

7. Presence of retinal bipolar cells and retinal nerve fiber layer on OCT testing

Exclusion Criteria

1. 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.

2. Concurrent participation in another interventional clinical ocular study.

3. Pre-existing eye conditions such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).

4. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.

5. Subjects who are positive for hepatitis B, C, and HIV will be excluded.

6. Subjects who have undergone ocular surgery in the study eye within three months prior to Day 0.

7. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.

8. Known sensitivity to any component of the study agent or medications planned for use in the peri-operative period.

9. Subjects will be excluded if immunological studies show presence of neutralizing antibodies to AAV2 above 1:1000.

10. Presence of narrow iridocorneal angles contraindicating pupillary dilation.

11. Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including OCT, during the study period.

12. 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.

13. Current evidence of retinal detachment assessed by the investigator to significantly affect central vision.

14. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nanoscope Therapeutics Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Santosh Mahapatra, MD

Role: PRINCIPAL_INVESTIGATOR

JPM Rotary Club of Cuttack Eye Hospital and Research Institute

Locations

JPM Rotary Club of Cuttack Eye Hospital and Research Institute

Cuttack, Odisha, India

Countries

India

Other Identifiers

NSCT/CT/18/01

Identifier Type: -

Identifier Source: org_study_id