Retinitis PigmentosaEXTENDNCT05921162
A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection
An observational study of Retinitis Pigmentosa and Retinal Disease, run by Nanoscope Therapeutics Inc. at 1 site in India.
Trial summary
This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Retinal Disease, Retinal Degeneration
- What is being tested?
- Gene Therapy product:vMCO-I
- How many people
- 11 participants
- Where
- 1 siteIndia
- Study dates
- 14 Jul 2023to May 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: It is expected that a total of 11 study subjects from NSCT/CT/18/01 study will be enrolled. Subjects who meet all the inclusion criteria and for whom none of the exclusion criteria apply will be eligible for enrollment.
Who may be able to join
- The subject population includes subjects who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I), administered via intravitreal injection in a parent study.
Who cannot join
- Subjects who will not consent for study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
India1 site
JPM Rotary Club of Cuttack Eye Hospital and Research Institute
Cuttack, Odisha, 753014
About this study
Official title: A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection
This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection
Read the detailed description
The observational study population comprises of individuals who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection in a parent study.
Enrolled subjects will undergo safety and efficacy assessments for up to 5 years from the vMCO-I administration. At the visits, subjects will have safety and efficacy evaluations. Subjects will have a final visit at 60 months post study drug administration.
- Conditions
- Retinitis Pigmentosa, Retinal Disease, Retinal Degeneration
- Being tested
- Gene Therapy product:vMCO-I
- Keywords
- Optogenetics, Gene Therapy, AAV Vectors, Multicharacteristic Opsin (MCO)-I
- Medical subject headings
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy product:vMCO-IBiological
Safety evaluation to monitor long term effects of previously injected vMCO-I in RP patients
| Study group | Role | What they receive |
|---|---|---|
| Observation of Participants exposed to 1.2E11gc/eye of vMCO-I | This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of vMCO-I No investigational product will be administered in this study. Receives: Biological: Gene Therapy product:vMCO-I | |
| Observation of Participants exposed to 0.6E11gc/eye of vMCO-I | This is a long-term follow-up observational study of participants who previously received 0.6E11gc/eye of vMCO-I No investigational product will be administered in this study. Receives: Biological: Gene Therapy product:vMCO-I |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I
Assessments of Incidences, nature, and severity of treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal anatomy
Measured: 20 Months
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I)
Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)
Measured: 20 Months
Secondary outcomes (2)
Assessment of the effect of vMCO-I on functional vision outcomes
Assessment of the treatment effect on the quality of Life with changes from baseline in activities of daily living using the National Eye Institute (NEI) Visual Function Questionnaire-25
Measured: 20 Months
Assessment of the durability of vMCO-I induced gene reporter expression
Assessment of PK parameters including the Change in fundus fluorescence intensity of reporter (mCherry)
Measured: 20 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2023
- Study start14 Jul 2023
- Primary completionMay 2025 (estimated)
- Study completionMay 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 11 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Samarendra Mohanty, Study director, Nanoscope Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05921162
- Sponsor's protocol ID
- NTXMCO-005
- Acronym
- EXTEND
- Status last verified
- Mar 2025
- First submitted
- 19 Jun 2023
- Last updated
- 25 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection
- ClinicalTrials.gov ID
- NCT05921162
- Acronym / sponsor ID
- EXTEND / NTXMCO-005
- Conditions
- Retinitis Pigmentosa, Retinal Disease, Retinal Degeneration
- Keywords
- Optogenetics, Gene Therapy, AAV Vectors, Multicharacteristic Opsin (MCO)-I
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 11 participants (estimated)
- Lead sponsor
- Nanoscope Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Jul 2023 (actual)
- Primary completion
- May 2025 (estimated)
- Study completion
- May 2025 (estimated)
- First posted
- 27 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study "A Long-Term Follow-Up Study in Subjects Who Received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection" is an observational study and will be conducted following Good Clinical Practice (GCP)- International Conference on Harmonization (ICH) guidelines. Eligible subjects satisfying all inclusion and none of the exclusion criteria will be enrolled. All subject who completed the parent clinical study (NSCT/CT/18/01) will undergo safety and efficacy assessments up to 5 years post study drug injection
Detailed description
The observational study population comprises of individuals who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I) Administered Via Intravitreal Injection in a parent study.
Enrolled subjects will undergo safety and efficacy assessments for up to 5 years from the vMCO-I administration. At the visits, subjects will have safety and efficacy evaluations. Subjects will have a final visit at 60 months post study drug administration.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Observation of Participants exposed to 1.2E11gc/eye of vMCO-I | – | Biological: Gene Therapy product:vMCO-IThis is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of vMCO-I No investigational product will be administered in this study. |
| Observation of Participants exposed to 0.6E11gc/eye of vMCO-I | – | Biological: Gene Therapy product:vMCO-IThis is a long-term follow-up observational study of participants who previously received 0.6E11gc/eye of vMCO-I No investigational product will be administered in this study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- It is expected that a total of 11 study subjects from NSCT/CT/18/01 study will be enrolled. Subjects who meet all the inclusion criteria and for whom none of the exclusion criteria apply will be eligible for enrollment.
Key inclusion criteria (1 listed)
- The subject population includes subjects who received an Adeno-Associated Viral Vector Serotype 2 Containing the Multi-Characteristic Opsin Gene (vMCO-I), administered via intravitreal injection in a parent study.
Key exclusion criteria (1 listed)
- Subjects who will not consent for study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gene Therapy product:vMCO-ISafety evaluation to monitor long term effects of previously injected vMCO-I in RP patients | Biological | Observation of Participants exposed to 0.6E11gc/eye of vMCO-I, Observation of Participants exposed to 1.2E11gc/eye of vMCO-I |
Primary outcome
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I
Time frame: 20 Months
Assessments of Incidences, nature, and severity of treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal anatomy
Evaluation of the long-term efficacy of a single intravitreal injection of Multi-Characteristic Opsin (vMCO-I)
Time frame: 20 Months
Assessment of treatment effect with the change from baseline in Freiburg Visual Acuity (quantitative LogMAR score)
2 secondary outcomes
Assessment of the effect of vMCO-I on functional vision outcomes
Time frame: 20 Months
Assessment of the durability of vMCO-I induced gene reporter expression
Time frame: 20 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2025.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Retinitis Pigmentosa
RecruitingObservational study
Rod and Cone Mediated Function in Retinal Disease
Retinal Degeneration, Retinitis Pigmentosa
RecruitingObservational study
Cell Collection to Study Eye Diseases
Retinal Disease, AMD
Recruiting
Brain Stimulation Effects on Orientation and Mobility Skills in Adults With Vision Impairment
Retinitis Pigmentosa (RP), Rod Cone Dystrophy
RecruitingObservational study
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
Inherited Retinal Degeneration, Retinitis Pigmentosa
Active, not recruitingObservational study
Rate of Progression in EYS Related Retinal Degeneration
Retinitis Pigmentosa, Eye Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Samarendra Mohanty study director
Nanoscope Therapeutics Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinitis Pigmentosa and Retinal Disease, run by Nanoscope Therapeutics Inc. at 1 site in India.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 25 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in India. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of the long-term safety profile of a single intravitreal injection of vMCO-I (time frame: 20 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2025.