RetinoschisisPhase 1/2NCT02317887
Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
A Phase 1/2 study testing RS1 AAV Vector in people with Retinoschisis and X-Linked, run by VegaVect, Inc. at 1 site in United States.
Trial summary
Background: X-linked juvenile retinoschisis (XLRS) is caused by changes in the RS1 gene. These changes cause abnormal function of the eye protein retinoschisin. Without normal retinoschisin, the layers of the retina split and vision is lost. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Retinoschisis, X-Linked
- What is being tested?
- RS1 AAV Vector
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Feb 2015to 16 Oct 2024
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is male with a mutation in the RS1 gene identified by genotyping.
- Participant must be 18 years of age or older.
- Participant must be able to understand and sign the informed consent.
- Participant must be medically able to comply with the study treatment, study testing and procedures and follow-up visits.
- Participant has at least one eye that meets the study eye criteria listed below.
- Participant must agree to use effective barrier (male or female condom) of contraception starting two weeks before and continuing one year after gene transfer.
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- If the participant's partner is able to become pregnant, a second form of effective contraception will be required starting two weeks before and continuing one year after gene transfer.
- Effective methods of contraception for this study include:
- hormonal contraception (birth control pills, injected hormones or vaginal ring),
- intrauterine device,
- barrier methods (condom or diaphragm) combined with spermicide,
- surgical sterilization (hysterectomy or tubal ligation in partner or vasectomy).
Who cannot join
- Participant is actively receiving another study medication/investigational product (IP).
- Participant has previously enrolled in another gene therapy trial.
- Participant is currently taking, or has taken in the last three months, a systemic carbonic anhydrase inhibitor prior to enrollment/baseline 1 testing.
- Participant has any condition that significantly increases risk of systemic corticosteroids or systemic steroid-sparing immuno-modulatory agents, such as HIV, syphilis, tuberculosis, hepatitis B, hepatitis C, or diabetes mellitus (DM).
- Participant has an underlying serious illness that impairs regular follow-up during the study.
- Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years.
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- Participant has pre-existing ocular tumors (excluding non-suspicious nevi).
- Participant has a known allergy to fluorescein dye or other contraindications to obtaining a fluorescein angiogram.
- Participant is on a medication that prevents safe administration of study related drugs.
- Participant has uncontrolled hypertension. (Hypertension judged to be adequately controlled at baseline medical evaluation is not exclusionary.)
- Participant has compromised renal function such that cyclosporine or Cellcept, which could be used to treat any manifest ocular inflammation, would be contraindicated.
- Participant has significant liver disease with elevated liver enzymes (greater than or equal to 2.5 times upper limit of normal [ULN]).
- Participant has low absolute neutrophil count (ANC<1.3 x 10(3)/micro liters).
- Participant has used any biologic immunosuppressive agents within the last three months (within the last six months for rituximab or cyclophosphamide).
- STUDY EYE ELIGIBILITY CRITERIA:
- The participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below.
- STUDY EYE INCLUSION CRITERIA:
- The study eye must have a best-corrected E-ETDRS visual acuity letterscore of less than or equal to 63 (i.e., worse than or equal to 20/63). The visual acuity from the first baseline visit (Baseline 1) will be used for eligibility determination in case of a change in visual acuity at the second baseline visit (Baseline 2).
- Electroretinogram in the study eye with a scotopic combined response demonstrating a subnormal b wave, consistent with retinoschisis.
- STUDY EYE EXCLUSION CRITERIA:
- The study eye has a history of other ocular disease likely to contribute significantly to visual loss or likely to present special risks (e.g., optic neuropathy, advanced glaucoma, uveitis, large bullous schisis cavities or bullous retinal detachment precluding safe intravitreal injection).
- The study eye has lens, cornea, or other media opacities precluding adequate visualization and testing of the retina.
- The study eye has undergone intraocular surgery within six months prior to enrollment.
- The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.
- STUDY EYE SELECTION CRITERIA:
- If both eyes of a participant meet the study eye eligibility criteria, the choice of study eye will be determined as follows:
- The eye with the worse visual acuity will be selected as the study eye.
- If both eyes have visual acuities within five letter differences of one another, the choice of study eye will be determined at the discretion of the Investigator in consultation with the participant.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Background:
- X-linked juvenile retinoschisis (XLRS) is caused by changes in the RS1 gene. These changes cause abnormal function of the eye protein retinoschisin. Without normal retinoschisin, the layers of the retina split and vision is lost. Researchers want to try to introduce a healthy RS1 gene into eye cells, to see if this helps retinal cells make healthy retinoschisin. They will put the gene in a virus. The gene and virus package is known as a gene transfer vector (AAV-RS1 vector).
Read more
Objectives:
- To see if the AAV-RS1 vector is safe to use in patients with X-linked retinoschisis.
Eligibility:
- Adults 18 and older with a mutation of the RS1 gene, 20/63 vision or worse in one eye, and XLRS.
Design:
- Participants will be screened with genetic tests to confirm XLRS. They will have a medical history and physical and eye exams.
- At visits 1-2, participants will have some or all of the following:
- Medical history
- Physical exam
- Blood and urine tests
- Tuberculosis skin test
- Eye exam
- Vision tests (for one test an intravenous line will be placed in the arm. A dye will be injected that will travel to the blood vessels in the eye).
- At visit 3, the AAV-RS1 vector will be injected with a needle in the study eye. Participants pupils will be dilated. They will get numbing eye drops.
- Visits 4-13 will occur in the 18 months after gene transfer. Many of the above tests will be repeated. Participants will discuss any side effects.
- Visits 14-17 will occur yearly between years 2 and 5.
- After year 5, participants will be contacted yearly by phone for up to 15 years.
Read the detailed description
Objective: To evaluate the safety and tolerability of ocular AAV-RS1 vector (AAV8-scRS/IRBPhRS) gene transfer to the retina of participants affected with X-linked juvenile retinoschisis (XLRS).
Study Population: Male participants affected with XLRS will receive ocular gene transfer. A maximum of up to 24 participants may be enrolled.
Design: This is a Phase I/IIa, prospective, dose escalation, single-center study. One eye of each participant will receive the AAV-RS1 gene vector application by intravitreal injection. Participants will be closely monitored in conjunction with DSMC oversight. Participants will be followed for 18 months after which they will continue to be followed for up to 5 years after enrollment, or per FDA requirements, for further safety analysis.
Outcome Measures: The primary outcome is the safety of ocular AAV-RS1 vector as determined from assessment of retinal function, ocular structure and occurrence of adverse events and laboratory tests. Secondary outcomes include changes in visual function, electroretinogram (ERG) responses, visual field measurements, retinal imaging with optical coherence tomography (OCT), and the formation of anti-AAV and anti-RS1 antibodies.
Statistics: No formal sample size calculations are used in this Phase I/IIa dose-escalation study.
- Conditions
- Retinoschisis, X-Linked
- Being tested
- RS1 AAV Vector
- Keywords
- Gene Transfer, X-Linked, Retinoschisis, AAV Vector
- Medical subject headings
- Retinoschisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RS1 AAV VectorBiological
Gene transfer by intravitreal injection of the RS1 AAV vector (AAV8-scRS/IRBPhRS)
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 1e9 vg/eye Receives: Biological: RS1 AAV Vector |
| Cohort 2 | Experimental | 1e10 vg/eye Receives: Biological: RS1 AAV Vector |
| Cohort 3 | Experimental | 1e11 vg/eye Receives: Biological: RS1 AAV Vector |
| Cohort 4 | Experimental | 1e11 vg/eye Receives: Biological: RS1 AAV Vector |
| Cohort 5 | Experimental | 3e11 vg/eye Receives: Biological: RS1 AAV Vector |
| Cohort 6 | Experimental | Not to exceed 6e11 vg/eye Receives: Biological: RS1 AAV Vector |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS
Includes a) substantial functional change (change >=10 electronic visual acuity letters in best-corrected visual acuity from average of baseline visits), b) decrease in electroretinogram response amplitude (>=75% from average of baseline visits), c) severe ocular inflammation beyond inflammation anticipated consequent to an intravitreal injection; d) adverse events deemed clinically-related to the intraocular administration technique, and e) abnormal laboratory findings beyond Grade 1 Common Terminology Criteria for Adverse Events v5.0 and/or clinically significantly different than baseline. a)-d) only includes events occurring in the study eye.
Measured: Day 0 through Year 2, inclusive
Secondary outcomes (6)
Change in Electroretinography Combined Response Amplitudes
Change in electroretinography combined response amplitudes from average of baseline 1 and 2 measurements. The higher the amplitude, the better.
Measured: Baseline 1 through Year 2
Mean Change in Best Corrected Visual Acuity
Mean change in best corrected visual acuity (BCVA) at Year 2 compared to average of baseline 1 and 2. BCVA is measured via Electronic Visual Acuity (EVA).
Measured: Baseline 1 through Year 2
Median and Distribution of Change in Best-Corrected Visual Acuity
Median and distribution of change in best-corrected visual acuity (BCVA) at Year 2 compared to average of baseline 1 and 2. BCVA is measured via Electronic Visual Acuity (EVA).
Measured: Baseline 1 through Year 2
Formation of Circulating Systemic Anti-AAV or Anti-RS1 Antibodies
Formation of circulating systemic anti-AAV or anti-RS1 antibodies assessed via serologic testing
Measured: Day 0 through end of study participation (Year 5 or Year 7)
Change in Retinal Structure as Measured by Optical Coherence Tomography
Change in retinal structure as measured by optical coherence tomography (OCT) compared to average of baseline 1 and 2. Includes a) quantitative measures of total retinal thickness obtained with the Cirrus OCT using macular cube scans, b) qualitative morphologic changes to macula anatomy investigated using the tracking ability of the Heidelberg Spectralis OCT system and c) length of intact ellipsoid zone on the OCT and any findings of restoration of this reflectivity line.
Measured: Baseline 1 through end of study participation (Year 5 or Year 7)
Change in Central Visual Field Sensitivity as Measured by Microperimetry (MP-1) Visual Field Testing
Change in central visual field sensitivity as measured by microperimetry (MP-1) Visual Field testing compared to average of baseline 1 and 2. Includes mean sensitivities, number of scotomatous points and number of points with a significant change in sensitivity.
Measured: Baseline 1 through end of study participation (Year 5 or Year 7)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2014 (estimated)
- Study start11 Feb 2015
- Primary completion25 Apr 2024
- Study completion16 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for RetinoschisisStudies 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
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- VegaVect, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Eye Institute (NEI)
- Responsible party
- Sponsor
- Investigators
- Laryssa A Huryn, M.D., Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02317887
- Sponsor's protocol ID
- 15EI0038
- Other IDs
- 15-EI-0038
- Status last verified
- Jan 2025
- First submitted
- 16 Dec 2014
- Last updated
- 14 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Vijayasarathy C, Zeng Y, Marangoni D, Dong L, Pan ZH, Simpson EM, Fariss RN, Sieving PA. Targeted Expression of Retinoschisin by Retinal Bipolar Cells in XLRS Promotes Resolution of Retinoschisis Cysts Sans RS1 From Photoreceptors. Invest Ophthalmol Vis Sci. 2022 Oct 3;63(11):8. doi: 10.1167/iovs.63.11.8.
PubMed 36227606Vijayasarathy C, Sardar Pasha SPB, Sieving PA. Of men and mice: Human X-linked retinoschisis and fidelity in mouse modeling. Prog Retin Eye Res. 2022 Mar;87:100999. doi: 10.1016/j.preteyeres.2021.100999. Epub 2021 Aug 11.
PubMed 34390869Mishra A, Vijayasarathy C, Cukras CA, Wiley HE, Sen HN, Zeng Y, Wei LL, Sieving PA. Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial. Mol Ther. 2021 Jun 2;29(6):2030-2040. doi: 10.1016/j.ymthe.2021.02.013. Epub 2021 Feb 15.
PubMed 33601057Cukras C, Wiley HE, Jeffrey BG, Sen HN, Turriff A, Zeng Y, Vijayasarathy C, Marangoni D, Ziccardi L, Kjellstrom S, Park TK, Hiriyanna S, Wright JF, Colosi P, Wu Z, Bush RA, Wei LL, Sieving PA. Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery. Mol Ther. 2018 Sep 5;26(9):2282-2294. doi: 10.1016/j.ymthe.2018.05.025. Epub 2018 Jul 7.
PubMed 30196853
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/IIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
- ClinicalTrials.gov ID
- NCT02317887
- Acronym / sponsor ID
- 15EI0038
- Conditions
- Retinoschisis, X-Linked
- Keywords
- Gene Transfer, X-Linked, Retinoschisis, AAV Vector
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- VegaVect, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Eye Institute (NEI)
- Study start
- 11 Feb 2015 (actual)
- Primary completion
- 25 Apr 2024 (actual)
- Study completion
- 16 Oct 2024 (actual)
- First posted
- 17 Dec 2014 (estimated)
- Results
- Posted 14 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- X-linked juvenile retinoschisis (XLRS) is caused by changes in the RS1 gene. These changes cause abnormal function of the eye protein retinoschisin. Without normal retinoschisin, the layers of the retina split and vision is lost. Researchers want to try to introduce a healthy RS1 gene into eye cells, to see if this helps retinal cells make healthy retinoschisin. They will put the gene in a virus. The gene and virus package is known as a gene transfer vector (AAV-RS1 vector).
Objectives:
- To see if the AAV-RS1 vector is safe to use in patients with X-linked retinoschisis.
Eligibility:
- Adults 18 and older with a mutation of the RS1 gene, 20/63 vision or worse in one eye, and XLRS.
Design:
- Participants will be screened with genetic tests to confirm XLRS. They will have a medical history and physical and eye exams.
- At visits 1-2, participants will have some or all of the following:
- Medical history
- Physical exam
- Blood and urine tests
- Tuberculosis skin test
- Eye exam
- Vision tests (for one test an intravenous line will be placed in the arm. A dye will be injected that will travel to the blood vessels in the eye).
- At visit 3, the AAV-RS1 vector will be injected with a needle in the study eye. Participants pupils will be dilated. They will get numbing eye drops.
- Visits 4-13 will occur in the 18 months after gene transfer. Many of the above tests will be repeated. Participants will discuss any side effects.
- Visits 14-17 will occur yearly between years 2 and 5.
- After year 5, participants will be contacted yearly by phone for up to 15 years.
Detailed description
Objective: To evaluate the safety and tolerability of ocular AAV-RS1 vector (AAV8-scRS/IRBPhRS) gene transfer to the retina of participants affected with X-linked juvenile retinoschisis (XLRS).
Study Population: Male participants affected with XLRS will receive ocular gene transfer. A maximum of up to 24 participants may be enrolled.
Design: This is a Phase I/IIa, prospective, dose escalation, single-center study. One eye of each participant will receive the AAV-RS1 gene vector application by intravitreal injection. Participants will be closely monitored in conjunction with DSMC oversight. Participants will be followed for 18 months after which they will continue to be followed for up to 5 years after enrollment, or per FDA requirements, for further safety analysis.
Outcome Measures: The primary outcome is the safety of ocular AAV-RS1 vector as determined from assessment of retinal function, ocular structure and occurrence of adverse events and laboratory tests. Secondary outcomes include changes in visual function, electroretinogram (ERG) responses, visual field measurements, retinal imaging with optical coherence tomography (OCT), and the formation of anti-AAV and anti-RS1 antibodies.
Statistics: No formal sample size calculations are used in this Phase I/IIa dose-escalation study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: RS1 AAV Vector1e9 vg/eye |
| Cohort 2 | Experimental | Biological: RS1 AAV Vector1e10 vg/eye |
| Cohort 3 | Experimental | Biological: RS1 AAV Vector1e11 vg/eye |
| Cohort 4 | Experimental | Biological: RS1 AAV Vector1e11 vg/eye |
| Cohort 5 | Experimental | Biological: RS1 AAV Vector3e11 vg/eye |
| Cohort 6 | Experimental | Biological: RS1 AAV VectorNot to exceed 6e11 vg/eye |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Participant is male with a mutation in the RS1 gene identified by genotyping.
- Participant must be 18 years of age or older.
- Participant must be able to understand and sign the informed consent.
- Participant must be medically able to comply with the study treatment, study testing and procedures and follow-up visits.
- Participant has at least one eye that meets the study eye criteria listed below.
Key exclusion criteria (28 listed)
- Participant is actively receiving another study medication/investigational product (IP).
- Participant has previously enrolled in another gene therapy trial.
- Participant is currently taking, or has taken in the last three months, a systemic carbonic anhydrase inhibitor prior to enrollment/baseline 1 testing.
- Participant has any condition that significantly increases risk of systemic corticosteroids or systemic steroid-sparing immuno-modulatory agents, such as HIV, syphilis, tuberculosis, hepatitis B, hepatitis C, or diabetes mellitus (DM).
- Participant has an underlying serious illness that impairs regular follow-up during the 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 |
|---|---|---|
| RS1 AAV VectorGene transfer by intravitreal injection of the RS1 AAV vector (AAV8-scRS/IRBPhRS) | Biological | Cohort 1, Cohort 2, Cohort 3, Cohort 4, Cohort 5, Cohort 6 |
Primary outcome
Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS
Time frame: Day 0 through Year 2, inclusive
Includes a) substantial functional change (change >=10 electronic visual acuity letters in best-corrected visual acuity from average of baseline visits), b) decrease in electroretinogram response amplitude (>=75% from average of baseline visits), c) severe ocular inflammation beyond inflammation anticipated consequent to an intravitreal injection; d) adverse events deemed clinically-related to the intraocular administration technique, and e) abnormal laboratory findings beyond Grade 1 Common Terminology Criteria for Adverse Events v5.0 and/or clinically significantly different than baseline. a)-d) only includes events occurring in the study eye.
6 secondary outcomes
Change in Electroretinography Combined Response Amplitudes
Time frame: Baseline 1 through Year 2
Mean Change in Best Corrected Visual Acuity
Time frame: Baseline 1 through Year 2
Median and Distribution of Change in Best-Corrected Visual Acuity
Time frame: Baseline 1 through Year 2
Formation of Circulating Systemic Anti-AAV or Anti-RS1 Antibodies
Time frame: Day 0 through end of study participation (Year 5 or Year 7)
Change in Retinal Structure as Measured by Optical Coherence Tomography
Time frame: Baseline 1 through end of study participation (Year 5 or Year 7)
Change in Central Visual Field Sensitivity as Measured by Microperimetry (MP-1) Visual Field Testing
Time frame: Baseline 1 through end of study participation (Year 5 or Year 7)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36227606Vijayasarathy C, Zeng Y, Marangoni D, Dong L, Pan ZH, Simpson EM, Fariss RN, Sieving PA. Targeted Expression of Retinoschisin by Retinal Bipolar Cells in XLRS Promotes Resolution of Retinoschisis Cysts Sans RS1 From Photoreceptors. Invest Ophthalmol Vis Sci. 2022 Oct 3;63(11):8. doi: 10.1167/iovs.63.11.8.
- Derived from this study: PubMed 34390869Vijayasarathy C, Sardar Pasha SPB, Sieving PA. Of men and mice: Human X-linked retinoschisis and fidelity in mouse modeling. Prog Retin Eye Res. 2022 Mar;87:100999. doi: 10.1016/j.preteyeres.2021.100999. Epub 2021 Aug 11.
- Derived from this study: PubMed 33601057Mishra A, Vijayasarathy C, Cukras CA, Wiley HE, Sen HN, Zeng Y, Wei LL, Sieving PA. Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial. Mol Ther. 2021 Jun 2;29(6):2030-2040. doi: 10.1016/j.ymthe.2021.02.013. Epub 2021 Feb 15.
- Derived from this study: PubMed 30196853Cukras C, Wiley HE, Jeffrey BG, Sen HN, Turriff A, Zeng Y, Vijayasarathy C, Marangoni D, Ziccardi L, Kjellstrom S, Park TK, Hiriyanna S, Wright JF, Colosi P, Wu Z, Bush RA, Wei LL, Sieving PA. Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery. Mol Ther. 2018 Sep 5;26(9):2282-2294. doi: 10.1016/j.ymthe.2018.05.025. Epub 2018 Jul 7.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Laryssa A Huryn, M.D. principal investigator
National Eye Institute (NEI)
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 RS1 AAV Vector in people with Retinoschisis and X-Linked, run by VegaVect, Inc. at 1 site in United States.
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 12 inclusion and 28 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 14 Feb 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 United States. 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?
Yes. Results are posted in the registry (first posted 14 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS (time frame: Day 0 through Year 2, inclusive). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02317887, which also lists 4 linked publications.
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 14 Feb 2025.