Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Purpose
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).
Condition
- Limbal Stem Cell Deficiency
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Have an area of the central 5 mm zone of cornea affected, presence of at least one clinical sign observed by slit-lamp examination with white light and/or cobalt blue light, fluorescein, and a Wratten filter, and at least one clinical sign observed with AS-OCT. - If dosing with Acthar at time of Screening Visit, must have a history of stable dosing for approximately 8 weeks, plan to maintain the same Acthar treatment dose and regimen, and have demonstrated little to no improvement in severity of LSCD. - Sponsor written confirmation of qualifying LSCD diagnosis. - Inflammation associated with LSCD or other non-infectious inflammation that will not interfere with subject's participation.
Exclusion Criteria
- Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye. - Ocular surgery within 30 days of Day 0 where investigator assesses that healing is not considered complete/stable. - Planned ocular surgery on or before Week 20 visit. - Presence or history of any ocular or systemic disorder, condition, or procedure that might hinder the efficacy of the study treatment or its evaluation, or could interfere with the interpretation of study results, in the opinion of the investigator. Note: Other inclusion/exclusion criteria apply.
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Control |
Observation (non-interventional) for 20 weeks followed by one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days followed by observation (non-interventional) for 52 weeks (total participation of 96 weeks) |
|
|
Experimental CSB-001 QID |
One drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days followed by observation (non-interventional) for 72 weeks (total participation of 96 weeks) |
|
Recruiting Locations
Loma Linda University Eye Institute
Loma Linda 5367696, California 5332921 92354
Loma Linda 5367696, California 5332921 92354
Contact:
Study Coordinator
Study Coordinator
Midwest Cornea Associates, LLC
Carmel 4255466, Indiana 4921868 46290
Carmel 4255466, Indiana 4921868 46290
Contact:
Study Coordinator
Study Coordinator
Francis Price Jr, MD
Indianapolis 4259418, Indiana 4921868 46260
Indianapolis 4259418, Indiana 4921868 46260
Contact:
Study Coordinator
Study Coordinator
Minnesota Eye Consultants
Minnetonka 5037784, Minnesota 5037779 55305
Minnetonka 5037784, Minnesota 5037779 55305
Contact:
Study Coordinator
Study Coordinator
Legacy Devers Eye Institute
Portland 5746545, Oregon 5744337 97210
Portland 5746545, Oregon 5744337 97210
Contact:
Study Coordinator
Study Coordinator
Stuart A. Terry, MD PA
San Antonio 4726206, Texas 4736286 78212
San Antonio 4726206, Texas 4736286 78212
Contact:
Study Coordinator
Study Coordinator
More Details
- NCT ID
- NCT06452316
- Status
- Recruiting
- Sponsor
- Claris Biotherapeutics, Inc.