Evaluation of K9 in Subjects With Thyroid Eye Disease (TED)
NCT Number: NCT06467435
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1
Conditions: Thyroid Eye Disease
Interventions: DRUG: Kamuvudine-9|DRUG: Kamuvudine-9
Sponsor: Peter Timoney
Collaborators: Inflammasome Therapeutics
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 8
Study Design: Allocation: NON_RANDOMIZED|Intervention Model: PARALLEL|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08-29
Completion Date: 2025-05
Locations: University of Kentucky, Lexington, Kentucky, 40506, United States

Summary:
The objective of this clinical trial is to evaluate the plasma pharmacokinetics of K9 in healthy subjects (Cohort 1) and the safety and treatment efficacy of K9 in patients with active Thyroid Eye Disease (Cohort 2). Participants will receive study medication one time or for up to 4 weeks. Participants will have blood drawn and/or complete eye exams and questionnaires.

The planned duration of this study is 6 weeks.

Primary Outcome Measures: Adverse Events, Frequency of participants experiencing ocular or systemic adverse events., 6 weeks|plasma concentrations of K9, plasma concentrations measured after a single oral dose in healthy adults. For each of these time points 5 ml of blood will be drawn., Pharmacokinetic samples collected pre-dose and at 0.5, 1, 2, 3, 4, 6, 8, and 24 hours after dosing
Secondary Outcome Measures: Change in Standardized Patient Evaluation of Eye Dryness (SPEED) symptoms, The SPEED Questionnaire will be used to track progression of dry eye symptoms. The SPEED questionnaire has eight questions. The frequency and severity of symptoms are rated on a numeric scale. Scores range from 0 to 28 with higher scores equating to more symptoms., Baseline (Day 1) and weeks 1, 4, and 6|Change in Diplopia, Diplopia is assessed by the Bahn-Gorman Scale. The Bahn-Gorman scale is a subjective grading scheme that uses a scale of 0-3 to assess the severity of diplopia. A higher score equates to more severe diplopia., Baseline (Day 1) and weeks 1, 4, and 6|Change in Study Visit in Clinical Activity Score (CAS),, Clinical Activity Score (7 point scale) with higher score equating to more symptoms, Baseline (Day 1) and weeks 1, 4, and 6|Change in proptosis, Hertel exophthalmometry will be used to measure the globe position of the eye by calculating the distance from the lateral orbital rim to the surface of the cornea in millimeters (mm), Baseline (Day 1) and weeks 1, 4, and 6|Change in Graves' ophthalmopathy-specific quality-of-life scale (GO-QOL), The GO-QOL is a self-administered health-related QOL questionnaire specifically designed and validated in patients with TED. It includes 2 subscale GO-QOL scores: (1) visual functioning and (2) appearance-related, with the 2 weighted equally to create an overall score. Both the subscales and overall score are transformed to a scale of 0 to 100, 0 indicating worst health and 100 indicating best health., Baseline (Day 1) and weeks 1, 4, and 6|Change in upper eyelid retraction, upper eyelid retraction will be determined by measuring the margin reflex distance (MRD1) in millimeters. The MRD1 is the measurement in millimetres from the light reflex on the patient's cornea to the level of the centre of the upper eyelid margin, with the patient gazing in the primary position., Baseline (Day 1) and weeks 1, 4, and 6|Change in lower eyelid retraction, Lower eyelid retraction is measured by the distance from the inferior limbus to the lower eyelid, or by the margin reflex distance 2 (MRD2). The distance between the corneal light reflex to the central portion of the lower eyelid is measured in millimeters, Baseline (Day 1) and weeks 1, 4, and 6|Change in inflammasome output cytokines IL-1B, measured by ELISA, Baseline (Day 1) and weeks 1, 4, and 6|Change in inflammasome output cytokines IL-18, measured by ELISA, Baseline (Day 1) and weeks 1, 4, and 6