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) (ELECTRO)

• ClinicalTrials.gov Identifier: NCT04207983
• Recruitment Status: Completed
• First Posted: December 23, 2019
• Last Update Posted: March 10, 2022
• Sponsor: Eyevensys
• Information provided by (Responsible Party): Eyevensys

Tracking Information

• First Submitted Date: December 19, 2019
• First Posted Date: December 23, 2019
• Last Update Posted Date: March 10, 2022
• Actual Study Start Date: February 3, 2020
• Actual Primary Completion Date: October 5, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 19, 2019)

Time to rescue therapy between the two EYS606 treatment regimens [ Time Frame: Week 24 ]

Assessment of efficacy measured as time to rescue therapy required after treatment with EYS606

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 19, 2019)

• Proportion (%) of subjects responded to the treatment [ Time Frame: Week 8 and 24 ]
  Measured as an improvement in anterior cell grade and vitreous haze grade according to the SUN scale, retinal vessel leakage using fluorescein angiography, central retinal thickness using ocular coherence tomography, and an increase in visual acuity using EDTRS compared to baseline
• Proportion (%) of subjects achieving and maintaining active chronic noninfectious posterior uveitis (CNIU) [ Time Frame: Week 24 ]
  Measured as an improvement in anterior cell grade and vitreous haze grade according to the SUN scale, retinal vessel leakage using fluorescein angiography, central retinal thickness using ocular coherence tomography, and an increase in visual acuity using EDTRS compared to baseline
• Median time to control of active CNIU [ Time Frame: Each Visit up to Week 48 ]
  Measured as an improvement in anterior cell grade and vitreous haze grade according to the SUN scale, retinal vessel leakage using fluorescein angiography, and central retinal thickness using ocular coherence tomography
• Median time to loss of treatment effect [ Time Frame: Each Visit up to Week 48 ]
  Measured as an worsening in anterior cell grade and vitreous haze grade according to the SUN scale, retinal vessel leakage using fluorescein angiography, central retinal thickness using ocular coherence tomography, decrease in visual acuity using EDTRS, and any increase in the frequency of dose of specified concomitant medications
• Median change in visual acuity [ Time Frame: Each Visit up to Week 48 ]
  Measured in change from baseline in best-corrected visual acuity using EDTRS

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• 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.

• Study Type: Interventional
• Study Phase: Phase 2

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
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)

Masking: None (Open Label)
Primary Purpose: Treatment

• Condition: Non-infectious Uveitis

Intervention

Combination Product: EYS606

EYS606 is a DNA plasmid solution administered by electrotransfection into the ciliary muscle

Study Arms

• Experimental: Treatment Arm A (Re-administration)
  Two administrations of EYS606 (135μg pEYS606/90 μL). The frequency between the two administrations will be determined by the DSMB upon completion of the Part I safety cohorts.
  Intervention: Combination Product: EYS606
• Experimental: Treatment Arm B (Single administration)
  One administration of EYS606 (135μg pEYS606/90 μL) at the baseline visit (V1).
  Intervention: Combination Product: EYS606

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 9, 2022): 3
• Original Estimated Enrollment (submitted: December 19, 2019): 56
• Actual Study Completion Date: October 5, 2021
• Actual Primary Completion Date: October 5, 2021 (Final data collection date for primary outcome measure)

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).

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04207983
• Other Study ID Numbers: EYS606-CT2
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Eyevensys
• Original Responsible Party: Same as current
• Current Study Sponsor: Eyevensys
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Eyevensys
• Verification Date: March 2022