- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04919473
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
June 2, 2021 updated by: Nanoscope Therapeutics Inc.
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
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)
Study Overview
Status
Completed
Intervention / Treatment
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
Study Type
Interventional
Enrollment (Actual)
11
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Odisha
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Cuttack, Odisha, India, 753014
- JPM Rotary Club of Cuttack Eye Hospital and Research Institute
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: vMCO-I High dose
Participants received 3.5E11vg/eye of vMCO-I
|
The vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
|
Experimental: vMCO-I Low Dose
Participants received 1.75E11vg/eye of vMCO-I
|
The vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
16 Weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Evaluate the treatment effect of vMCO-l as assessed by visual acuity
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with the Freiburg Visual Acuity (FrACT) to provide automated, self-paced, monitored measurement
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Light-guided Mobility assays, performed at different light intensities, to provide functional vision measures using the time to find lighted panel
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Light-guided Mobility assays, performed at different light intensities, to provide functional vision measures using the score based on correct choice
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Static Shape recognition assay, performed at different light intensities, to provide visual function measures using size determination threshold
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Static Shape recognition assay, performed at different light intensities, to provide visual function measures using % shape recognition accuracy
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Optical Flow assay, performed at different speeds, to provide visual function measures using the %accuracy in determining direction of flow
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 of parameters measured with Optical Flow assay, performed at different speeds, to provide visual function measures using the Upper speed limit to determine correct optical flow
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire
Time Frame: 52 weeks
|
Assessment of the treatment effect on Quality of Life changes from baseline to Week 52 with Visual Function Questionnaire-25 (VFQ-25).VFQ25 is a 25-item questionnaire with 47 questions, each question has several responses scored on a scale from 0-5, 0-6, or 0-10.
Values are calculated in percentages.
|
52 weeks
|
Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field
Time Frame: 52 weeks
|
Assessment of the treatment effect with the change from baseline to Week 52 with Humphrey Visual Field (30-2).
Visual Field Index (VFI) is calculated in % and Mean Deviation (MD) values are calculated in dB.
|
52 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Santosh Mahapatra, MD, JPM Rotary Club of Cuttack Eye Hospital and Research Institute
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 23, 2019
Primary Completion (Actual)
February 25, 2020
Study Completion (Actual)
October 31, 2020
Study Registration Dates
First Submitted
May 25, 2021
First Submitted That Met QC Criteria
June 2, 2021
First Posted (Actual)
June 9, 2021
Study Record Updates
Last Update Posted (Actual)
June 9, 2021
Last Update Submitted That Met QC Criteria
June 2, 2021
Last Verified
June 1, 2021
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- NSCT/CT/18/01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Yes
IPD Plan Description
The results of the clinical trial will be made available when the study is completed and results are analyzed.
The results will be published on this site and be available to conference presentations and publications.
IPD Sharing Time Frame
6 months after the completion of the study
IPD Sharing 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.
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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