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Varenicline

Phase 3

Dry Eye Disease | Small molecule | Ophthalmology |Milestone Pharmaceuticals Inc.|Last Updated: May 1, 2023

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment349

FDA Designations

No designations recorded

Clinical trial landscape

Varenicline · 2 trials · 1 indication

Phase 3 1Phase 1 1
NCT05378945Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray on Signs and Symptoms of Dry Eye DiseaseDry Eye Disease
COMPLETED340 Analytics
PHASE3COMPLETED
Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray on Signs and Symptoms of Dry Eye Disease
Dry Eye DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Schirmer's Test Score
28 days

Percentage of subjects who achieve ≥10 mm improvement in Schirmer's Test Score from baseline

Cmax of varenicline after the first dosing at Visit 1 [Day 1] and after the last dosing at Visit 3 [Day 28], respectively.
Visit 1 [Day 1]: From predose of the 1st dosing to 12 hours post-dose. Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose.

To evaluate the Cmax of varenicline in adult Chinese with dry eye disease (DED) after the first dosing at Visit 1 \[Day 1\] and after the last dosing at Visit 3 \[Day 28\], respectively.

Tmax of varenicline after the first dosing at Visit 1 [Day 1] and after the last dosing at Visit 3 [Day 28], respectively.
Visit 1 [Day 1]: From predose of the 1st dosing to 12 hours post-dose. Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose.

To evaluate the Tmax of varenicline in adult Chinese subjects with DED after the first dosing at Visit 1 \[Day 1\] and after the last dosing at Visit 3 \[Day 28\], respectively.

AUCtau(Area under the concentration-time curve during a dosing interval) of varenicline after the first dosing at Visit 1 [Day 1] and after the last dosing at Visit 3 [Day 28], respectively.
Visit 1 [Day 1]: From predose of the 1st dosing to 12 hours post-dose. Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose.

To evaluate the AUCtau of varenicline in adult Chinese subjects with DED after the first dosing at Visit 1 \[Day 1\] and after the last dosing at Visit 3 \[Day 28\], respectively.

AUC0-last(Area under the concentration-time curve from time 0 to the time of the last measured non-zero concentration) of varenicline after the first dosing at Visit 1 [Day 1] and after the last dosing at Visit 3 [Day 28], respectively.
Visit 1 [Day 1]: From predose of the 1st dosing to 12 hours post-dose. Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose.

To evaluate the AUC0-last of varenicline in adult Chinese subjects with DED after the first dosing at Visit 1 \[Day 1\] and after the last dosing at Visit 3 \[Day 28\], respectively.

T1/2 of varenicline after the last dosing at Visit 3 [Day 28]
Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose

To evaluate the T1/2 of varenicline after the last dosing at Visit 3 \[Day 28\] in adult Chinese subjects with DED.

λz of varenicline after the last dosing at Visit 3 [Day 28]
Visit 3 [Day 28]: From predose of the last dosing to 120 hours post-dose

To evaluate the λz of varenicline after the last dosing at Visit 3 \[Day 28\] in adult Chinese subjects with DED.

Secondary Endpoints

Mean change from Baseline in Schirmer's Test Score (STS)
28 days
Mean change from Baseline in Eye Dryness Score (EDS)
28 days/ 14 days/ 7days
Change from baseline in Schirmer's Test Score (STS) at Visit 1 [Day 1], Visit 2 [Day 14] and Visit 3 [Day 28]
From Day 1 to Day 28
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
OC-01EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
Varenicline Tartrate Nasal SprayDRUGIntranasal delivery of OC-01 (varenicline solution) 0.6 mg/mL twice a day (BID) for 28 days
Placebo (vehicle)DRUGIntranasal delivery of placebo (vehicle) twice a day (BID) for 28 days
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: * Have used and/or desired to use an artificial tear substitute for dry eye symptoms within 6 months prior to the Screening Visit Exclusion Criteria: * Have had any intraocular surgery (such as cataract surgery) or extraocular surgery in either eye within three months or refra...

Countries:China
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Competitive Landscape -Dry Eye Disease 7 trials

Frequently asked questions about Varenicline

What is Varenicline used for in Dry Eye Disease?

Varenicline, developed as OC-01 nasal spray, is an investigational small molecule being studied for the treatment of Dry Eye Disease. It is delivered as a nasal spray and is currently in clinical development, with Phase 3 trials completed. The drug is not yet approved and remains investigational.

What does Varenicline target?

Varenicline is a small molecule that acts as a nicotinic acetylcholine receptor agonist. It is being studied for its effects on the signs and symptoms of Dry Eye Disease when administered as a nasal spray. The exact mechanism in this context is under investigation.

Who makes Varenicline?

Varenicline is being developed by Milestone Pharmaceuticals Inc., a biopharmaceutical company traded on the NASDAQ under the ticker MIST. The company is conducting clinical trials for the drug as a treatment for Dry Eye Disease.

What phase is Varenicline in?

Varenicline is in Phase 3 clinical development for Dry Eye Disease. A Phase 3 trial has been completed, along with a Phase 1 pharmacokinetic study. The drug is investigational and has not received FDA approval.

What clinical trials is Varenicline in?

Varenicline has been studied in two completed clinical trials in China. NCT05378945 is a Phase 3 trial evaluating the efficacy and safety of OC-01 nasal spray in 340 adults with Dry Eye Disease. NCT05576415 is a Phase 1 trial assessing pharmacokinetics, pharmacodynamics, safety, and tolerability in 9 adults.

Is Varenicline the same as OC-01?

Yes, Varenicline is the same as OC-01. The drug is referred to as OC-01 in clinical trial titles, and both names refer to the same investigational nasal spray being developed for Dry Eye Disease.