DT-168 for people with Fuchs endothelial corneal dystrophy who are having a corneal transplant

A Phase 2, Multicenter, Open-Label Study to Assess the Pharmacodynamics, Safety, and Tolerability of DT-168 Ophthalmic Solution in Patients With Fuchs Endothelial Corneal Dystrophy Undergoing Keratoplasty

This study will test whether DT-168 eye drops are safe, well tolerated, and how they act in people with Fuchs endothelial corneal dystrophy who are scheduled for corneal transplant.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (estimated)
• Ages: 30 Years and up
• Sex: All
• Sponsor: Design Therapeutics, Inc. (Industry-sponsored)
• Locations: 2 sites (Indianapolis, Indiana and 1 other locations)
• Trial ID: NCT07024693 on ClinicalTrials.gov

What this trial studies

This Phase 2 interventional study administers DT-168 ophthalmic solution to patients with Fuchs endothelial corneal dystrophy who are scheduled to undergo keratoplasty, with the goal of characterizing pharmacodynamics, safety, and tolerability. Eligible participants must be at least 30 years old, have a documented diagnosis of FECD in the study eye, and carry a confirmed TCF4 allele CTG expansion. Treatments are given in the pre-keratoplasty period with regular in-person assessments for ocular measurements, pharmacodynamic sampling, and safety monitoring at study visits. Keratoplasty is performed as planned independent of study participation, and outcomes focus on corneal pharmacodynamic markers, adverse events, and participant-reported tolerability.

Who should consider this trial

Good fit: Ideal candidates are adults aged 30 or older with documented FECD in the study eye, a confirmed TCF4 CTG expansion in at least one allele, who are scheduled for keratoplasty and can give informed consent.

Not a fit: Patients without the TCF4 CTG expansion, those not planning keratoplasty, or those with other significant ocular or medical conditions that would confound results are unlikely to benefit from this study.

Why it matters

Potential benefit: If successful, DT-168 eye drops could offer a safe preoperative treatment that improves corneal biology or preserves endothelial function before transplant.

How similar studies have performed: This approach is relatively novel and there are limited prior clinical data showing success with DT-168 specifically.

Eligibility criteria

Inclusion Criteria:

* Participant is scheduled to undergo a keratoplasty for management of FECD independent of this study.
* ≥30 years of age (inclusive).
* Documented diagnosis of FECD in the study eligible eye.
* Confirmation of positive TCF4 allele 18.1 CTG expansion in at least one allele.
* Capable of giving signed informed consent.

Exclusion Criteria:

* Any ocular or medical condition in the study eligible eye which is clinically significant or would confound study results or data interpretation.
* Any clinically significant medical, nonmedical, and psychiatric disorders that could put the participant at higher risk for participation in the study, impair the participant's ability to participate in the study, or interfere with interpretation of the participant's study results, in the opinion of the investigator.
* Concurrent or anticipated need for treatment for FECD, eg, Muro 128 (2% or 5%) during the course of the study in the study eye. Provided the subject undergoes a washout of Muro drops for at least 24 hours prior to the Baseline Visit, they will not be excluded.
* Concurrent or anticipated use of topical corticosteroids in the study eye.
* Concurrent use with Rhopressa®, Rocklatan®, or any other ocular Rho Kinase inhibitor.
* Known contraindication, allergy, or hypersensitivity to any of the treatments, anesthetics, or diagnostic agents or components thereof which may be used during the study.
* Use of contact lenses in the study eye within 7 days prior to the Baseline Visit or planned use during the study.
* Recent (within 30 days of the Screening Visit) or ongoing participation in any other investigational interventional clinical study.
* Female participant is pregnant, planning a pregnancy, or breast-feeding.
* Participant is unwilling to comply with the contraceptive requirements, as per protocol.

Where this trial is running

Indianapolis, Indiana and 1 other locations

• DTX-168-201 Study Site — Indianapolis, Indiana, United States (Recruiting)
• DTX-168-201 Study Site — Grand Rapids, Michigan, United States (Recruiting)

Study contacts

• Study coordinator: Mark Daniels

Conditions Fuchs Endothelial Corneal Dystrophy; Fuchs