Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

Limbal Stem Cell Deficiency Clinical Trial

Official title:

A Single-Arm Open-Label Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06452316
• Other study ID #: CSB-C23-001
• Status: Recruiting
• Phase: Phase 1
• Start date: May 15, 2024
• Est. completion date: August 2025

Study information

• Verified date: June 2024
• Source: Claris Biotherapeutics, Inc.
• Contact: Central Operations Representative
• Phone: 919-354-1299
• Email: clinicaltrials[@]clarisbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: August 2025
• Est. primary completion date: August 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Limbal Stem Cell Deficiency

Intervention

Biological:
• CSB-001 Ophthalmic Solution 0.1%
CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

Locations

Country	Name	City	State
United States	Midwest Cornea Associates, LLC	Carmel	Indiana

Sponsors (1)

Lead Sponsor	Collaborator
Claris Biotherapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety as Assessed by Adverse Event Reporting	Incidence of ocular and systemic adverse events	Day 0 through Week 48
Primary	Safety as Assessed by Slit-lamp Biomicroscopy	Change from baseline in slit-lamp parameters	Day 0 through Week 48
Primary	Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity	Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart	Day 0 through Week 48
Secondary	Efficacy as Assessed by Change in Stage of LSCD	Change from baseline in LSCD signs based on imaging (white light, blue light with fluorescein and Wratten filter, and AS-OCT)	Day 0 through Week 48
Secondary	Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity	Visual acuity measured using the EDTRS visual acuity chart	Day 0 through Week 48