Retinitis PigmentosaPhase 1/2NCT04919473
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
A Phase 1/2 study testing Gene Therapy product:vMCO-I in people with Retinitis Pigmentosa and Retinal Diseases, run by Nanoscope Therapeutics Inc. at 1 site in India.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
- What is being tested?
- Gene Therapy product:vMCO-I
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteIndia
- Study dates
- 23 Oct 2019to 31 Oct 2020
- Phase
- Phase 1/2
- 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
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
India1 site
JPM Rotary Club of Cuttack Eye Hospital and Research Institute
Cuttack, Odisha, 753014
About this study
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
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)
Read the 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
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
- Being tested
- Gene Therapy product:vMCO-I
- Keywords
- Retinitis Pigmentosa, Retinal Degeneration, 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
The vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
| Study group | Role | What they receive |
|---|---|---|
| vMCO-I High dose | Experimental | Participants received 3.5E11vg/eye of vMCO-I Receives: Biological: Gene Therapy product:vMCO-I |
| vMCO-I Low Dose | Experimental | Participants received 1.75E11vg/eye of vMCO-I Receives: Biological: Gene Therapy product:vMCO-I |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
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.
Measured: 16 Weeks
Secondary outcomes (9)
Evaluate the treatment effect of vMCO-l as assessed by visual acuity
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
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
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire
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.
Measured: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field
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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2021
- Study start23 Oct 2019
- Primary completion25 Feb 2020
- Study completion31 Oct 2020
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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanoscope Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Santosh Mahapatra, MD, Principal investigator, JPM Rotary Club of Cuttack Eye Hospital and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04919473
- Sponsor's protocol ID
- NSCT/CT/18/01
- Status last verified
- Jun 2021
- First submitted
- 25 May 2021
- Last updated
- 9 Jun 2021
- 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 9 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04919473
- Acronym / sponsor ID
- NSCT/CT/18/01
- Conditions
- Retinitis Pigmentosa, Retinal Diseases, Retinal Degeneration
- Keywords
- Retinitis Pigmentosa, Retinal Degeneration, Optogenetics, Gene Therapy, AAV vectors, Multicharacteristic Opsin (MCO)-I
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Nanoscope Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Oct 2019 (actual)
- Primary completion
- 25 Feb 2020 (actual)
- Study completion
- 31 Oct 2020 (actual)
- First posted
- 9 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 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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| vMCO-I High dose | Experimental | Biological: Gene Therapy product:vMCO-IParticipants received 3.5E11vg/eye of vMCO-I |
| vMCO-I Low Dose | Experimental | Biological: Gene Therapy product:vMCO-IParticipants received 1.75E11vg/eye of vMCO-I |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (14 listed)
- 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.
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-IThe vMCO-I is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette | Biological | vMCO-I High dose, vMCO-I Low Dose |
Primary outcome
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.
9 secondary outcomes
Evaluate the treatment effect of vMCO-l as assessed by visual acuity
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire
Time frame: 52 weeks
Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field
Time frame: 52 weeks
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 9 Jun 2021.
- 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.
Santosh Mahapatra, MD principal investigator
JPM Rotary Club of Cuttack Eye Hospital and Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Gene Therapy product:vMCO-I in people with Retinitis Pigmentosa and Retinal Diseases, 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 7 inclusion and 14 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 9 Jun 2021. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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). 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 9 Jun 2021.