A Single-Center, Randomized, Controlled, Masked Clinical Trial to Evaluate the Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation

Molecule Varenicline solution nasal spray (formerly known as OC-01)
Therapeutic focus Dry eye disease
Phase 2
Status Completed
Participants 18
Completion Date September 30, 2019

Note: This clinical trial was not included in the submission which resulted in Tyrvaya® approval.
https://clinicaltrials.gov/study/NCT03688802.

Trial Design1

Note: The FDA-approved dosage of varenicline solution nasal spray is 0.03 mg BID.2 This clinical trial was not included in the submission which resulted in Tyrvaya® approval.
*Target enrollment was 45. However, enrollment was slower than expected, and the study was ended prior to completing target enrollment.
FDA, Food and Drug Administration.
1. Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561; 2. TYRVAYA®(varenicline solution) nasal spray. Prescribing Information. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213978s005lbl.pdf.

Inclusion Criteria

  • A diagnosis of dry eye disease
  • ≥18 years old
  • Used and/or desired to use an artificial tear substitute for dry eye symptoms within 6 months before visit 1
  • Ocular Surface Disease Index (OSDI) ≥23 with ≤3 responses of “not applicable” at screening​
    • Corneal fluorescein staining (CFS) score of ≥2 in at least 1 corneal region, or a sum of ≥4 for all corneal regions
    • Baseline Schirmer’s test score (STS) (with topical anesthesia) of ≤12 mm/5 minutes, with a cotton swab nasal stimulation STS of ≥7 mm more in the same eye
    • <20-mm difference between the study and fellow eye STS
Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561.

Exclusion Criteria

  • Clinically significant corneal epithelial defects at visit 1 before performing Schirmer’s test
  • Nasal or sinus surgery (including history of application of nasal cautery) or significant trauma to these areas
  • Vascularized polyp, severely deviated septum, or severe nasal airway obstruction (as confirmed by intranasal examination performed before visit 1)
  • Current use of nasal continuous positive airway pressure (CPAP) therapy
  • Any intraocular surgery or extraocular surgery in either eye within 3 months or refractive surgery within 12 months of visit 1
  • A corneal transplant in either eye
  • Contact lens use within 7 days before visit 1
Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561.

Primary Endpoints

Change in pre- and post-treatment:

  • Goblet cell area in both eyes
  • Goblet cell perimeter in both eyes
  • Meibomian gland area in both eyes
Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561.

Safety Endpoints

  • Treatment emergent adverse events*
*Defined as adverse events that were new or had worsened in severity compared with first study drug use. All adverse events were coded using the Medical Dictionary for Regulatory Activities, version 22.0.
Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561.

Baseline Characteristics1

Note: The FDA-approved dosage of varenicline solution nasal spray is 0.03 mg BID.2 *Data presented as mean (SD), unless otherwise specified.
EDS, eye dryness score; FDA, Food and Drug Administration; OSDI, Ocular Surface Disease Index; SD, standard deviation; STS, Schirmer's test score; VNS, varenicline solution nasal spray.
1. Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561; 2. TYRVAYA®(varenicline solution) nasal spray. Prescribing Information. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213978s005lbl.pdf.

Efficacy Outcome Measures – Primary Endpoint1

Note: The FDA-approved dosage of varenicline solution nasal spray is 0.03 mg BID.2 Outcomes were compared using an ANCOVA model with treatment group and baseline measurements (meibomian cell area, goblet cell area, and goblet cell perimeter) as covariates.1
ANCOVA, analysis of covariance; FDA, Food and Drug Administration; SD, standard deviation; VNS, varenicline solution nasal spray.
1. Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561; 2. TYRVAYA®(varenicline solution) nasal spray. Prescribing Information. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213978s005lbl.pdf.

Efficacy Outcome Measures – Primary Endpoint1

Note: The FDA-approved dosage of varenicline solution nasal spray is 0.03 mg BID.2 Outcomes were compared using an ANCOVA model with treatment group and baseline measurements (meibomian cell area, goblet cell area, and goblet cell perimeter) as covariates.1
ANCOVA, analysis of covariance; FDA, Food and Drug Administration; SD, standard deviation; VNS, varenicline solution nasal spray.
1. Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561; 2. TYRVAYA®(varenicline solution) nasal spray. Prescribing Information. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213978s005lbl.pdf.

Safety Outcome Measures*1

Note: The FDA-approved dosage of varenicline solution nasal spray is 0.03 mg BID.2 *Data presented as n (%); †Patients reporting more than one event were counted only once at the maximum severity reported.
AE, adverse event; FDA, Food and Drug Administration; SAE, serious adverse event; TEAE, treatment-emergent adverse event; VNS, varenicline solution nasal spray.
1. Dieckmann G.M. et al.Ophthalmol Ther. 2022;11:1551–1561; 2. TYRVAYA®(varenicline solution) nasal spray. Prescribing Information. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/213978s005lbl.pdf.