Retinitis PigmentosaPhase 2RESTORENCT04945772
Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
A Phase 2 study testing Gene Therapy Product-MCO-010 in people with Retinitis Pigmentosa and Retinitis, run by Nanoscope Therapeutics Inc. at 6 sites in 2 countries.
Trial summary
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010). 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 13 Jul 2021to 18 Jan 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
Who may be able to join
- Age ≥ 18 years
- Able to comprehend and give informed consent.
- Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
- Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.
Who cannot join
- Subjects are excluded from the study if any of the following criteria apply:
- Prior participation in gene therapy program
- Pre-existing conditions in the study eye 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.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
- Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
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: A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).
Read the detailed description
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Being tested
- Gene Therapy Product-MCO-010, Sham Injection
- Keywords
- Retinitis Pigmentosa, Eye Diseases Hereditary, Eye Diseases, Retinal Degeneration, Inherited Retinal Diseases, Rod & cone dystrophies, Optogenetics, Gene Therapy, AAV vectors, Intravitreal Injections, Low Vision, Multi-Characteristic Opsin, No Light Perception, Visual Acuity, Multi-Luminance Y Mobility Test (MLYMT), Multi-Luminance Shape Discrimination Test (MLSDT)
- Medical subject headings
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration, Cone-Rod Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gene Therapy Product-MCO-010Biological
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Sham InjectionProcedure or surgery
Sham Injection
| Study group | Role | What they receive |
|---|---|---|
| MCO-010- High Dose | Experimental | Participants receive 1.2E11gc/eye of MCO-010 Receives: Biological: Gene Therapy Product-MCO-010 |
| MCO-010- Low Dose | Experimental | Participants receive 0.9E11gc/eye of MCO-010 Receives: Biological: Gene Therapy Product-MCO-010 |
| Sham Injection | Sham comparator | Participants receive sham injection Receives: Procedure: Sham Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.
Measured: Week 52
Secondary outcomes (6)
Efficacy of MCO-010 as assessed by best corrected visual acuity.
Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76.
Measured: Week 76
Efficacy of MCO-010 as assessed by mobility testing.
Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome.
Measured: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by mobility testing.
Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome.
Measured: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome.
Measured: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome.
Measured: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed on visual field.
Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry.
Measured: Weeks 16,24,32,52,76,100
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2021
- Study start13 Jul 2021
- Primary completion27 Feb 2023
- Study completion18 Jan 2024
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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Following a 1:1:1 block randomization schema, 9 subjects will be enrolled in each MCO-010 treatment group, and 9 subjects will be enrolled in the sham-controlled group.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dr Samarendra Mohanty, Study chair, Nanoscope Therapeutics Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04945772
- Sponsor's protocol ID
- NTXMCO-002.
- Acronym
- RESTORE
- Status last verified
- Mar 2024
- First submitted
- 15 Jun 2021
- Last updated
- 22 Mar 2024
- Results posted
- No
- Data monitoring committee
- Yes
- 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. 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 22 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
- ClinicalTrials.gov ID
- NCT04945772
- Acronym / sponsor ID
- RESTORE / NTXMCO-002.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies
- Keywords
- Retinitis Pigmentosa, Eye Diseases Hereditary, Eye Diseases, Retinal Degeneration, Inherited Retinal Diseases, Rod & cone dystrophies, Optogenetics, Gene Therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Nanoscope Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Jul 2021 (actual)
- Primary completion
- 27 Feb 2023 (actual)
- Study completion
- 18 Jan 2024 (actual)
- First posted
- 30 Jun 2021 (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.
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (MCO-010).
Detailed description
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 100 weeks following treatment with MCO-010.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Following a 1:1:1 block randomization schema, 9 subjects will be enrolled in each MCO-010 treatment group, and 9 subjects will be enrolled in the sham-controlled group.
| Arm | Type | Receives |
|---|---|---|
| MCO-010- High Dose | Experimental | Biological: Gene Therapy Product-MCO-010Participants receive 1.2E11gc/eye of MCO-010 |
| MCO-010- Low Dose | Experimental | Biological: Gene Therapy Product-MCO-010Participants receive 0.9E11gc/eye of MCO-010 |
| Sham Injection | Sham comparator | Procedure: Sham InjectionParticipants receive sham injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age ≥ 18 years
- Able to comprehend and give informed consent.
- Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
- Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.
Key exclusion criteria (6 listed)
- Prior participation in gene therapy program
- Pre-existing conditions in the study eye 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.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
- Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
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-010The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette | Biological | MCO-010- High Dose, MCO-010- Low Dose |
| Sham InjectionSham Injection | Procedure or surgery | Sham Injection |
Primary outcome
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity.
Time frame: Week 52
Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.
6 secondary outcomes
Efficacy of MCO-010 as assessed by best corrected visual acuity.
Time frame: Week 76
Efficacy of MCO-010 as assessed by mobility testing.
Time frame: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by mobility testing.
Time frame: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Time frame: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed by static shape recognition assay.
Time frame: Weeks 16,24,32,52,76,100
Efficacy of MCO-010 as assessed on visual field.
Time frame: Weeks 16,24,32,52,76,100
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2024.
- Individual participant dataSharing planned: Yes. The results of the clinical trial will be made available when the study is completed. 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.
Dr Samarendra Mohanty study chair
Nanoscope Therapeutics Inc.
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Gene Therapy Product-MCO-010 in people with 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 6 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: finished, as of the sponsor's last update on 22 Mar 2024. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity. (time frame: Week 52). 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 22 Mar 2024.