RetinoschisisEarly Phase 1NCT06289452
Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis
A Early Phase 1 study testing IVB102 Injection in people with Retinoschisis and Retinal Disease, run by InnoVec Biotherapeutics Inc. at 1 site in China.
Trial summary
The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS. Read more about this study
- Who is it for?
- Ages 8 years and older, men only
- Conditions the study is for
- Retinoschisis, Retinal Disease, Retinal Degeneration
- What is being tested?
- IVB102 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteChina
- Study dates
- 8 Mar 2024to 31 Dec 2029
- Phase
- Early Phase 1
- 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 8 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Must be willing and able to provide written, signed informed consent.
- Male individual at least 8 years of age with clinical diagnosis of XLRS caused by mutations in RS1.
- Best corrected visual acuity (BCVA) in the study eye of less than or equal to 63 (corresponding to a Snellen acuity of 20/63).
- Must agree to use effective barrier (male or female condom) of contraception before dosing and continuing one year after gene transfer.
Who cannot join
- Lens, cornea, or other media opacities in the study eye that preclude adequate visualization and testing of the retina.
- Pre-existing eye conditions that would contribute significantly to visual loss or increase the risk of an intravitreal injection (e.g. DR、RVO or large retinal detachment).
- Any intraocular surgery in the study eye within 6 months prior to screening.
- Use of topical carbonic anhydrase inhibitors within 3 months prior to screening.
- Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration.
- Prior receipt of any AAV gene therapy product.
- Use of any investigational agent within 3 months prior to screening.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100142
About this study
Official title: An Open-label, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of IVB102 Injection in Subjects With X-linked Retinoschisis (XLRS)
The goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.
Read the detailed description
This is a open label, dose escalation, single-center study. One eye of each participant will receive a single IVB102 injection by intravitreal injection. Participants will be followed for 52 weeks after which they will continue to be followed for up to 5 years after enrollment.
- Conditions
- Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases
- Being tested
- IVB102 Injection
- Medical subject headings
- Retinoschisis, Retinal Diseases, Retinal Degeneration, Eye Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVB102 InjectionGenetic
Gene transfer by intravitreal injection of the RS1 AAV vector
IVB102 InjectionGenetic
Gene transfer by intravitreal injection of the RS1 AAV vector
IVB102 InjectionGenetic
Gene transfer by intravitreal injection of the RS1 AAV vector
| Study group | Role | What they receive |
|---|---|---|
| IVB102 Treatment Arm（Low dose） | Experimental | Intraocular injection of a single low dose of IVB102 Receives: Genetic: IVB102 Injection |
| IVB102 Treatment Arm（Intermediate dose） | Experimental | Intraocular injection of a single intermediate dose of IVB102 Receives: Genetic: IVB102 Injection |
| IVB102 Treatment Arm（High dose） | Experimental | Intraocular injection of a single high dose of IVB102 Receives: Genetic: IVB102 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of DLTs
Number and proportion of dose limited toxicity（DLTs）
Measured: 4 weeks
Incidence of AEs
Number and severity of overall and ocular Adverse Events (AEs)
Measured: 52 weeks
Incidence of SAEs
Number and severity of overall and ocular Serious Adverse Events (SAEs)
Measured: 52 weeks
Secondary outcomes (3)
Visual function
Change from baseline in BCVA(Best Corrected Visual Acuity)(ETDRS)
Measured: 52 weeks
OCT imaging
Change from baseline in CST(Central Retinal Thickness)(OCT)
Measured: 52 weeks
ERG parameters
Change from baseline in response amplitudes measured using Electroretinogram (ERG)
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 posted1 Mar 2024
- Study start8 Mar 2024
- Primary completion31 Dec 2025
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- InnoVec Biotherapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Peking Union Medical College Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06289452
- Sponsor's protocol ID
- IVB102-101
- Status last verified
- Nov 2026
- First submitted
- 25 Feb 2024
- Last updated
- 4 Nov 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Pimenides D, George ND, Yates JR, Bradshaw K, Roberts SA, Moore AT, Trump D. X-linked retinoschisis: clinical phenotype and RS1 genotype in 86 UK patients. J Med Genet. 2005 Jun;42(6):e35. doi: 10.1136/jmg.2004.029769.
PubMed 15937075Wang XF, Chen FF, Zhou X, Cheng XX, Xie ZG. A novel mutation in RS1 and clinical manifestations in a Chinese twin family with congenital retinoschisis. Front Genet. 2022 Sep 23;13:993157. doi: 10.3389/fgene.2022.993157. eCollection 2022.
PubMed 36212125Sikkink SK, Biswas S, Parry NR, Stanga PE, Trump D. X-linked retinoschisis: an update. J Med Genet. 2007 Apr;44(4):225-32. doi: 10.1136/jmg.2006.047340. Epub 2006 Dec 15.
PubMed 17172462
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of IVB102 Injection in Subjects With X-linked Retinoschisis (XLRS)
- ClinicalTrials.gov ID
- NCT06289452
- Acronym / sponsor ID
- IVB102-101
- Conditions
- Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- InnoVec Biotherapeutics Inc.Industry (pharma, biotech, devices)
- Collaborators
- Peking Union Medical College Hospital
- Study start
- 8 Mar 2024 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 1 Mar 2024 (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 goal of this clinical trial is to evaluate the safety and efficacy of IVB102 injection in subjects with XLRS.
Detailed description
This is a open label, dose escalation, single-center study. One eye of each participant will receive a single IVB102 injection by intravitreal injection. Participants will be followed for 52 weeks after which they will continue to be followed for up to 5 years after enrollment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| IVB102 Treatment Arm（Low dose） | Experimental | Genetic: IVB102 InjectionIntraocular injection of a single low dose of IVB102 |
| IVB102 Treatment Arm（Intermediate dose） | Experimental | Genetic: IVB102 InjectionIntraocular injection of a single intermediate dose of IVB102 |
| IVB102 Treatment Arm（High dose） | Experimental | Genetic: IVB102 InjectionIntraocular injection of a single high dose of IVB102 |
Who may qualify
- Ages
- 8 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Must be willing and able to provide written, signed informed consent.
- Male individual at least 8 years of age with clinical diagnosis of XLRS caused by mutations in RS1.
- Best corrected visual acuity (BCVA) in the study eye of less than or equal to 63 (corresponding to a Snellen acuity of 20/63).
- Must agree to use effective barrier (male or female condom) of contraception before dosing and continuing one year after gene transfer.
Key exclusion criteria (7 listed)
- Lens, cornea, or other media opacities in the study eye that preclude adequate visualization and testing of the retina.
- Pre-existing eye conditions that would contribute significantly to visual loss or increase the risk of an intravitreal injection (e.g. DR、RVO or large retinal detachment).
- Any intraocular surgery in the study eye within 6 months prior to screening.
- Use of topical carbonic anhydrase inhibitors within 3 months prior to screening.
- Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration.
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 |
|---|---|---|
| IVB102 InjectionGene transfer by intravitreal injection of the RS1 AAV vector | Genetic | IVB102 Treatment Arm（Low dose） |
| IVB102 InjectionGene transfer by intravitreal injection of the RS1 AAV vector | Genetic | IVB102 Treatment Arm（Intermediate dose） |
| IVB102 InjectionGene transfer by intravitreal injection of the RS1 AAV vector | Genetic | IVB102 Treatment Arm（High dose） |
Primary outcome
Incidence of DLTs
Time frame: 4 weeks
Number and proportion of dose limited toxicity（DLTs）
Incidence of AEs
Time frame: 52 weeks
Number and severity of overall and ocular Adverse Events (AEs)
Incidence of SAEs
Time frame: 52 weeks
Number and severity of overall and ocular Serious Adverse Events (SAEs)
3 secondary outcomes
Visual function
Time frame: 52 weeks
OCT imaging
Time frame: 52 weeks
ERG parameters
Time frame: 52 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 4 Nov 2026.
- Background: PubMed 15937075Pimenides D, George ND, Yates JR, Bradshaw K, Roberts SA, Moore AT, Trump D. X-linked retinoschisis: clinical phenotype and RS1 genotype in 86 UK patients. J Med Genet. 2005 Jun;42(6):e35. doi: 10.1136/jmg.2004.029769.
- Background: PubMed 36212125Wang XF, Chen FF, Zhou X, Cheng XX, Xie ZG. A novel mutation in RS1 and clinical manifestations in a Chinese twin family with congenital retinoschisis. Front Genet. 2022 Sep 23;13:993157. doi: 10.3389/fgene.2022.993157. eCollection 2022.
- Background: PubMed 17172462Sikkink SK, Biswas S, Parry NR, Stanga PE, Trump D. X-linked retinoschisis: an update. J Med Genet. 2007 Apr;44(4):225-32. doi: 10.1136/jmg.2006.047340. Epub 2006 Dec 15.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing IVB102 Injection in people with Retinoschisis and Retinal Disease, run by InnoVec Biotherapeutics Inc. at 1 site in China.
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 7 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 4 Nov 2026. 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 China. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Incidence of DLTs (time frame: 4 weeks). The study lists 3 primary outcomes in all. 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 NCT06289452, which also lists 3 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 4 Nov 2026.