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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

65 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-05-15

Study Completion Date

2026-05-31

Brief Summary

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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).

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Detailed Description

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Conditions

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Limbal Stem Cell Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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)

Group Type EXPERIMENTAL

CSB-001 Ophthalmic Solution 0.1%

Intervention Type BIOLOGICAL

CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

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)

Group Type EXPERIMENTAL

CSB-001 Ophthalmic Solution 0.1%

Intervention Type BIOLOGICAL

CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

Interventions

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CSB-001 Ophthalmic Solution 0.1%

CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No