Clinical trial NCT06452316: Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Official title: An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Limbal Stem Cell Deficiency
Lead sponsor: Claris Biotherapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-06-11
Enrollment: 65
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Safety as Assessed by Adverse Event Reporting
timeFrame: Day 0 through Week 96
measure: Safety as Assessed by Slit-lamp Biomicroscopy
timeFrame: Day 0 through Week 96
measure: Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
timeFrame: Day 0 through Week 96
Secondary outcomes:
measure: Efficacy as Assessed by Change in Stage of LSCD
timeFrame: Day 0 through Week 96
measure: Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity
timeFrame: Day 0 through Week 96
Other outcomes:
Locations:
city: Loma Linda
country: United States
facility: Loma Linda University Eye Institute
state: California
city: Carmel
country: United States
facility: Midwest Cornea Associates, LLC
state: Indiana
city: Indianapolis
country: United States
facility: Francis Price Jr, MD
state: Indiana
city: Minnetonka
country: United States
facility: Minnesota Eye Consultants
state: Minnesota
city: Portland
country: United States
facility: Legacy Devers Eye Institute
state: Oregon
city: San Antonio
country: United States
facility: Stuart A. Terry, MD PA
state: Texas
Study officials:
References: