Clinical trial NCT04207983: A 48 Week Study to Evaluate the Efficacy and Safety of Two (2) EYS606 Treatment Regimens in Subjects With Active Chronic Non-infectious Uveitis (CNIU)
Official title: A 48 Week Phase II, Randomized, Open-Label, Multicenter, Study to Evaluate the Efficacy and Safety of Two (2) EYS606 Treatment Regimens in Subjects With Active Chronic Non-infectious Uveitis (CNIU)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Non-infectious Uveitis
Lead sponsor: Eyevensys
Sponsor class: INDUSTRY
First posted: 2019-12-23
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Part I (Safety cohort phase) - no randomization will take place; subjects will be consecutively assigned to one of two sequential safety cohort Regimens and receive two administrations of EYS606

Part II (Randomized comparison phase) - 1:1 randomization to either Treatment Arm A (Regimen 1 or 2 based upon recommendation by the DSMB, n=25) or Treatment Arm B (Regimen 3, n=25)
Primary purpose: TREATMENT

Brief summary: The objective of the study is to evaluate the efficacy and safety of two different treatment regimens of EYS606.

Detailed description: This is a Phase 2, multi-center, randomized open-label interventional study of EYS606 in subjects with active chronic non-infectious uveitis.

The maximum study duration per patient is 51 Weeks (including an up to 3 week screening period + 48 weeks follow-up after treatment).

The study will be conducted in 2 parts. Part I is a safety cohort phase that will enroll up to 6 subjects, Part II is the randomized comparison phase that will enroll up to an additional 50 subjects.

Eligibility criteria: Key Eligibility Criteria:

1. Subject must be 18 years of age or older.
2. Subject must have a diagnosis of chronic non-infectious uveitis of any anatomic subtype (anterior, intermediate, posterior or panuveitis).
3. Subject must have a history of chronic or recurrent non-infectious uveitis requiring or having required treatment with corticosteroids (systemic, periocular or intraocular) and/or systemic immunosuppressive medication(s) in the 12 months prior to the screening visit.
4. Best corrected visual acuity of

   * Study Part I: ≥ 5 and \< 67 ETDRS letters in the study eye (equivalent to less than or equal to 20/50 but better than or equal to 20/800 Snellen).
   * Study Part II: ≥ 5 and \< 77 ETDRS letters in the study eye (equivalent to less than or equal to 20/32 but better than or equal to 20/800 Snellen).
5. At the screening and baseline visits subject must have active chronic non-infectious uveitis as evidenced by at least one or more of the following in the study eye:

   * Active retinal vasculitis (retinal vascular leakage) involving the posterior pole confirmed by the reading center.
   * Vitreous haze grade ≥ 2+ (SUN classification).
   * Anterior chamber cell grade ≥ 2+ (SUN classification); anterior chamber cells must be present for subjects with a diagnosis of chronic anterior non-infectious uveitis.
   * Persistent macular edema (defined as central retinal thickness (CRT) \> 300 microns or \> 320 microns using Zeiss Cirrus and Topcon or Heidelberg Spectralis spectral domain ocular coherence tomography (SD-OCT) instruments, respectively) despite treatment with corticosteroids and/or immunosuppressive therapy for at least 4 weeks prior to screening.
6. Subject receiving concomitant topical and/or systemic corticosteroids or allowed systemic immunosuppressive medications must have maintained the same treatment regimen (dosage/frequency) for at least 2 weeks prior to the baseline (V1) visit, (if applicable).

Primary outcomes:
measure: Time to rescue therapy between the two EYS606 treatment regimens
timeFrame: Week 24
Secondary outcomes:
measure: Proportion (%) of subjects responded to the treatment
timeFrame: Week 8 and 24
measure: Proportion (%) of subjects achieving and maintaining active chronic noninfectious posterior uveitis (CNIU)
timeFrame: Week 24
measure: Median time to control of active CNIU
timeFrame: Each Visit up to Week 48
measure: Median time to loss of treatment effect
timeFrame: Each Visit up to Week 48
measure: Median change in visual acuity
timeFrame: Each Visit up to Week 48
Other outcomes:
Locations:
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Arlington
country: United States
facility: Texas Retina Associates
state: Texas
city: Houston
country: United States
facility: Houston Eye Associates
state: Texas
Study officials:
References:
citation: Touchard E, Benard R, Bigot K, Laffitte JD, Buggage R, Bordet T, Behar-Cohen F. Non-viral ocular gene therapy, pEYS606, for the treatment of non-infectious uveitis: Preclinical evaluation of the medicinal product. J Control Release. 2018 Sep 10;285:244-251. doi: 10.1016/j.jconrel.2018.07.013. Epub 2018 Aug 1.