Clinical trial NCT04919473: Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Retinitis Pigmentosa
Retinal Diseases
Retinal Degeneration
Keywords: Retinitis Pigmentosa
Retinal Degeneration
Optogenetics
Gene Therapy
AAV vectors
Multicharacteristic Opsin (MCO)-I
Lead sponsor: Nanoscope Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2021-06-09
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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

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.

Primary outcomes:
measure: The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
timeFrame: 16 Weeks
Secondary outcomes:
measure: Evaluate the treatment effect of vMCO-l as assessed by visual acuity
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire
timeFrame: 52 weeks
measure: Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Cuttack
country: India
facility: JPM Rotary Club of Cuttack Eye Hospital and Research Institute
state: Odisha
Study officials:
affiliation: JPM Rotary Club of Cuttack Eye Hospital and Research Institute
name: Santosh Mahapatra, MD
role: PRINCIPAL_INVESTIGATOR
References: