Thyroid Eye Disease (TED)
Conditions
Brief summary
The goal of this study is to evaluate the safety, tolerability, and efficacy of KRIYA-586 (VV-14305) in treating thyroid eye disease (TED).
Interventions
GENETICVV-14305
VV-14305 will be administered via peribulbar injection.
OTHERSham (No Treatment)
Sham solution such as Saline
Sponsors
Kriya Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Must be 18 to 80 years of age (inclusive) at Screening. 2. Must be euthyroid (defined as normal thyroid-stimulating hormone) or have mild hyper- or hypothyroidism (being managed to bring them to a euthyroid state). 3. Best Corrected Visual Acuity (BCVA) score of 20/60 or better at Screening with no history of deterioration noted in the 3 months prior to Screening. 4. Must be willing and able to cease product use 10 Days prior to VV-14305 (or sham) peribulbar injection if using non-steroidal anti-inflammatory drug (NSAIDs), antiplatelet/anticoagulant medications or any herbal supplements, vitamins, or multivitamins with antiplatelet/anticoagulant properties. Product use may resume following the administration of VV-14305 or sham. 5. The study eye and the fellow eye must fall within the pre-defined degree of proptosis and clinical activity score (CAS) as measured at Screening. 6. Participants must be diagnosed with chronic moderate to severe TED at Screening.
Exclusion criteria
1. History of serious ocular condition(s) other than TED, including but not limited to uveitis, dry age-related macular degeneration (AMD), and wet AMD; Other orbital or ophthalmic diseases, including inflammatory conditions, optic neuropathy, tumors, glaucoma with visual field defect or visual field loss, that in the opinion of the Investigator and/or Medical Monitor is clinically significant. 2. Any medical, cognitive, or psychiatric condition that, in the opinion of the Investigator, could contraindicate the use of the investigational drug, make consistent study assessment and follow-up over the 12-month Post-Treatment Follow-up Period unlikely, or would make the participant an unsafe study candidate. 3. Diagnosed with diabetes (HbA1c ≥6.5%). 4. History of malignancy requiring chemotherapy and/or radiation in the 12 months prior to Screening, except for successfully treated nonmelanoma skin cancers (e.g., basal cell, squamous cell carcinomas), cervical intraepithelial neoplasia, and localized prostate cancer. 5. Known allergy or condition that would contraindicate the use of required study medications. 6. Any Screening assessment or laboratory value that, in the opinion of the Investigator and/or Medical Monitor, is clinically significant and renders the subject not suitable for study participation. 7. Any vaccination or planned vaccination 30 days prior to dosing, 4 weeks post dosing or during period of immunosuppression. 8. Participant requires or, in the opinion of the Investigator, is likely to require immediate surgical ophthalmological/orbital intervention or irradiation of either eye; has had any prior surgery for TED including orbital decompression, strabismus surgery and any eyelid surgery on either eye. 9. Prior participation in other investigational drug, biologic or device clinical trials within 30 days prior to Screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Safety and Tolerability of VV-14305 in participants with TED | 52 Weeks | Incidence and severity of ocular and non-ocular adverse events, clinical laboratory values, physical examinations, vital signs, and ophthalmic examinations |
| Part 2: Safety of VV-14305 in Participants with TED Compared to Sham | 52 Weeks | Incidence and severity of ocular and non-ocular adverse events, clinical laboratory values, physical examinations, vital signs, and ophthalmic examinations |
| Part 2: Efficacy of VV-14305 in participants with TED compared to Sham | 36 Weeks | Percentage of participants with reduction in proptosis in the study eye as measured by exophthalmometer |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Efficacy associated with VV-14305 in participants with TED | 36 Weeks | Percentage of participants with reduction in proptosis in the study eye as measured by exophthalmometer |
| Efficacy associated with VV-14305 in participants with TED (as compared to Sham for Part 2) | 36 Weeks | Percentage of participants with Baseline diplopia \>0 who have a ≥1 grade reduction |
| Effect of VV-14305 on quality of life (as compared to Sham for Part 2) | 12, 24, 36, and 52 Weeks | Mean change from Baseline in overall score in the Graves Orbitopathy Quality of Life (GO-QoL) questionnaire, which includes two subscales: Visual Functioning and Appearance (scale range 0-100), where higher scores indicate better quality of life |
| Pharmacokinetics (PK) of VV-14305 transgene product | 12, 24, 36, and 52 Weeks | Peak and steady-state concentrations of adeno-associated virus (AAV) vector-mediated transgene product in serum |
Countries
New Zealand, United States
Contacts
CONTACTVP, Medical Affairs
Outcome results
None listed