Phase 2 Clinical Trial to Evaluate OC-01 Nasal Spray in Subjects With Neurotrophic Keratopathy

Oyster Point Pharma

Status and phase: Completed; Phase 2

Conditions: Neurotrophic Keratopathy

Treatments: Drug: Placebo (vehicle) nasal spray; Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml

Study type: Interventional

Funder types: Industry

Identifiers

NCT04957758

OPP-102

Details and patient eligibility

About

The objective of this study is to evaluate the safety and effectiveness of OC-01 (varenicline) nasal spray as compared to placebo nasal spray for mean change from baseline in corneal fluorescein staining in subjects with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) neurotrophic keratopathy (NK) in one or both eyes.

Full description

Protocol OPP-102 is a Phase 2, multicenter, randomized, controlled, double- masked study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in subjects with NK. Approximately 100 subjects at least 18 years of age with a physicians' diagnosis of Stage 1 NK as defined by the Mackie Criteria and meeting all other study eligibility criteria will be randomized 1:1 and will receive OC-01 (varenicline)/ nasal spray or placebo nasal spray for 8 weeks three times daily (TID) as follows:

• OC-01 (varenicline) nasal spray, 1.2 mg/mL
• Placebo (vehicle control) nasal spray All doses will be delivered as a 50 microliter (µL) nasal spray.

Enrollment: 113 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Be at least 18 years of age at Visit 1.
2. Patients with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) NK in one or both eyes, as defined by the Mackie Criteria.
3. Evidence of decreased corneal sensitivity (≤ 4 cm using the Cochet-Bonnet aesthesiometer) in at least 1 corneal quadrant.
4. Schirmer's test without anesthesia ≥3 mm/ 5 minutes in the affected eye.
5. If a female is of childbearing potential, they must: use an acceptable means of birth control (acceptable methods of contraception include hormonal - oral, implantable, injectable, or transdermal contraceptives, mechanical - spermicide in conjunction with a barrier such as a diaphragm or condom, IUD, or surgical sterilization of partner), and have a negative urine pregnancy test on Day 1.

Exclusion criteria

1. Have Stage 2 or Stage 3 NK affecting one or both eyes.
2. Have ocular graft versus host disease or Stevens-Johnson syndrome.
3. Have any active ocular infection (COVID-19 conjunctivitis, bacterial, viral, fungal, or protozoal) or active ocular inflammation not related to NK in the affected eye.
4. Be currently receiving autologous serum tears, amniotic membrane, cenegermin, fresh frozen plasma or cord blood derivative tears.
5. Have severe blepharitis and/or severe meibomian gland disease in the study eye.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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113 participants in 2 patient groups, including a placebo group

OC-01 (varenicline) nasal spray, 1.2 mg/mL

Active Comparator group

Description:

OC-01 (varenicline) nasal spray, 1.2 mg/mL

Treatment:

Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml

Placebo (vehicle control) nasal spray

Placebo Comparator group

Description:

Placebo (vehicle control) nasal spray

Treatment:

Drug: Placebo (vehicle) nasal spray