Retinitis PigmentosaREMAINNCT06162585
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
An observational study of Retinitis Pigmentosa and Retinitis, run by Nanoscope Therapeutics Inc. at 6 sites in 2 countries.
Trial summary
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Retinitis, Retinal Diseases
- What is being tested?
- Gene Therapy product-MCO-010
- How many people
- 18 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 8 Dec 2023to Sep 2027
- 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: This study population will be comprised of the 18 adult subjects with advanced retinitis pigmentosa, previously dosed with MCO-010 in the RESTORE study.
Who may be able to join
- Previously enrolled in study NTXMCO-002 (RESTORE)
- Able to comprehend and give informed consent.
- Able to comply with testing and all protocol tests.
- Agree to participate for the full 3-year duration of follow-up to the best of their ability and barring any unforeseen circumstances.
Who cannot join
- Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at their final RESTORE study visit
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
United States5 sites
Nanoscope Clinical Site
Beverly Hills, California, 90211
Nanoscope Clinical Site
Fargo, North Dakota, 58103
Nanoscope Clinical Site
Houston, Texas, 77030
Nanoscope Clinical Site
McAllen, Texas, 78503
Nanoscope Clinical Site
Pensacola, Florida, 32503
About this study
Official title: Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study
Read the detailed description
This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for RESTORE and are not repeated herein.
The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Being tested
- Gene Therapy product-MCO-010
- Keywords
- Gene Therapy, AAV Vectors, Low Vision, Intravitreal Injections, Optogenetics, Multi-Characteristic Opsin, Multi-Luminance Y Mobility Test, Multi-Luminance Shape Discrimination Test, Visual Acuity
- Medical subject headings
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration, Vision, Low
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy product-MCO-010Biological
Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients
| Study group | Role | What they receive |
|---|---|---|
| Observation of Participants exposed 1.2E11gc/eye of MCO-010 | This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study. Receives: Biological: Gene Therapy product-MCO-010 | |
| Observation of Participants exposed to 0.9E11gc/eye of MCO-010 | This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study. Receives: Biological: Gene Therapy product-MCO-010 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010
Delayed adverse events. Incidence, nature, and severity of selected adverse events (AEs); all serious adverse events (SAEs); all ocular AEs including intraocular inflammation graded through ocular exam; non-ocular AEs with a common terminology criteria for adverse events (CTCAE) grade of 3 or greater; AEs of special interest (AESIs) including new malignancies, new incidence or exacerbation of any pre-existing neurologic disorder or rheumatologic or other autoimmune disorder, new incidence of hematologic disorder or new infection regardless of suspected relatedness to treatment with MCO-010.
Measured: 156 weeks
Secondary outcomes (4)
Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010
Change from baseline in BCVA over time in both eyes
Measured: 156 Weeks
Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Change from baseline in multi-luminance shape discrimination test (MLSDT) scores
Measured: 156 Weeks
Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score
Measured: 156 Weeks
Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy
Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time
Measured: 156 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2023
- Study start8 Dec 2023
- Primary completionJul 2027 (estimated)
- Study completionSep 2027 (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
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Samuel Barone, MD, Study director, Nanoscope Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06162585
- Sponsor's protocol ID
- NTXLTFU-008
- Acronym
- REMAIN
- Status last verified
- Mar 2025
- First submitted
- 30 Nov 2023
- Last updated
- 24 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- 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 24 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study
- ClinicalTrials.gov ID
- NCT06162585
- Acronym / sponsor ID
- REMAIN / NTXLTFU-008
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Keywords
- Gene Therapy, AAV Vectors, Low Vision, Intravitreal Injections, Optogenetics, Multi-Characteristic Opsin, Multi-Luminance Y Mobility Test, Multi-Luminance Shape Discrimination Test
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Nanoscope Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Dec 2023 (actual)
- Primary completion
- Jul 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 8 Dec 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 will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study
Detailed description
This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for RESTORE and are not repeated herein.
The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Observation of Participants exposed 1.2E11gc/eye of MCO-010 | – | Biological: Gene Therapy product-MCO-010This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study. |
| Observation of Participants exposed to 0.9E11gc/eye of MCO-010 | – | Biological: Gene Therapy product-MCO-010This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 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
- This study population will be comprised of the 18 adult subjects with advanced retinitis pigmentosa, previously dosed with MCO-010 in the RESTORE study.
Key inclusion criteria (4 listed)
- Previously enrolled in study NTXMCO-002 (RESTORE)
- Able to comprehend and give informed consent.
- Able to comply with testing and all protocol tests.
- Agree to participate for the full 3-year duration of follow-up to the best of their ability and barring any unforeseen circumstances.
Key exclusion criteria (1 listed)
- Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at their final RESTORE study visit
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-MCO-010Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients | Biological | Observation of Participants exposed 1.2E11gc/eye of MCO-010, Observation of Participants exposed to 0.9E11gc/eye of MCO-010 |
Primary outcome
Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010
Time frame: 156 weeks
Delayed adverse events. Incidence, nature, and severity of selected adverse events (AEs); all serious adverse events (SAEs); all ocular AEs including intraocular inflammation graded through ocular exam; non-ocular AEs with a common terminology criteria for adverse events (CTCAE) grade of 3 or greater; AEs of special interest (AESIs) including new malignancies, new incidence or exacerbation of any pre-existing neurologic disorder or rheumatologic or other autoimmune disorder, new incidence of hematologic disorder or new infection regardless of suspected relatedness to treatment with MCO-010.
4 secondary outcomes
Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010
Time frame: 156 Weeks
Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Time frame: 156 Weeks
Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
Time frame: 156 Weeks
Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy
Time frame: 156 Weeks
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 24 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.
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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. This study is not recruiting at the moment.
Samuel Barone, MD study director
Nanoscope Therapeutics Inc.
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinitis Pigmentosa and Retinitis, run by Nanoscope Therapeutics Inc. at 6 sites in 2 countries.
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 4 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: under way, not looking for participants, as of the sponsor's last update on 24 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 6 sites in United States, Puerto Rico. 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 of previous treatment with a single intravitreal injection of MCO-010 (time frame: 156 weeks). 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 24 Mar 2025.