Clinical trial NCT06289452: Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis
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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: EARLY_PHASE1
Conditions: Retinoschisis
Retinal Disease
Retinal Degeneration
Eye Diseases
Lead sponsor: InnoVec Biotherapeutics Inc.
Sponsor class: INDUSTRY
Collaborators: name: Peking Union Medical College Hospital
First posted: 2024-03-01
Enrollment: 18
Sex: MALE
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.

Eligibility criteria: Inclusion Criteria:

1. Must be willing and able to provide written, signed informed consent.
2. Male individual at least 8 years of age with clinical diagnosis of XLRS caused by mutations in RS1.
3. Best corrected visual acuity (BCVA) in the study eye of less than or equal to 63 (corresponding to a Snellen acuity of 20/63).
4. Must agree to use effective barrier (male or female condom) of contraception before dosing and continuing one year after gene transfer.

Exclusion Criteria:

1. Lens, cornea, or other media opacities in the study eye that preclude adequate visualization and testing of the retina.
2. 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).
3. Any intraocular surgery in the study eye within 6 months prior to screening.
4. Use of topical carbonic anhydrase inhibitors within 3 months prior to screening.
5. Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration.
6. Prior receipt of any AAV gene therapy product.
7. Use of any investigational agent within 3 months prior to screening.

Primary outcomes:
measure: Incidence of DLTs
timeFrame: 4 weeks
measure: Incidence of AEs
timeFrame: 52 weeks
measure: Incidence of SAEs
timeFrame: 52 weeks
Secondary outcomes:
measure: Visual function
timeFrame: 52 weeks
measure: OCT imaging
timeFrame: 52 weeks
measure: ERG parameters
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
References:
citation: 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.
citation: Wang 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.
citation: Sikkink 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.