28 Participants Needed

DT-168 for Fuchs' Dystrophy

Recruiting at 1 trial location
MD
Overseen ByMark Daniels
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Design Therapeutics, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new eye drop called DT-168 to evaluate its safety and effectiveness for individuals with Fuchs' endothelial corneal dystrophy, a condition where the inner layer of the cornea deteriorates. The goal is to assist those planning to undergo a corneal transplant due to this condition. The trial seeks participants diagnosed with Fuchs' dystrophy who are scheduled for corneal transplant surgery. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in eye care.

Will I have to stop taking my current medications?

You may need to stop using certain eye medications like Muro 128, topical corticosteroids, and Rho Kinase inhibitors before joining the study. Specifically, if you're using Muro 128, you'll need to stop at least 24 hours before the baseline visit.

Is there any evidence suggesting that DT-168 is likely to be safe for humans?

Research has shown that DT-168, an eye drop treatment for Fuchs' dystrophy, was safe in earlier studies. Tests with healthy volunteers reported no serious side effects or eye problems. This suggests the treatment is generally safe for people. Currently, DT-168 is being tested in patients awaiting corneal transplants. This ongoing study aims to further confirm its safety and tolerability.12345

Why do researchers think this study treatment might be promising?

DT-168 is unique because it introduces a new approach to treating Fuchs' Dystrophy compared to traditional options like corneal transplants and Descemet membrane endothelial keratoplasty (DMEK). Unlike these surgical methods, which replace damaged corneal tissue, DT-168 is an experimental drug that aims to enhance cellular function and potentially restore the health of existing corneal cells. Researchers are excited about DT-168 because it targets the underlying cellular dysfunction, offering a less invasive and more targeted treatment option that could improve patient outcomes and reduce the need for complex surgeries.

What evidence suggests that DT-168 might be an effective treatment for Fuchs' dystrophy?

Research has shown that DT-168, the investigational treatment in this trial, has potential in early studies for treating Fuchs' endothelial corneal dystrophy. In lab tests, DT-168 reduced harmful cell clusters to levels similar to those in healthy cells, suggesting the drug might help restore the function of the corneal endothelium, a layer of cells on the eye's surface. Previous human trials demonstrated that DT-168 is well-tolerated and does not cause serious side effects. These findings offer hope for its effectiveness in treating Fuchs' dystrophy.12346

Are You a Good Fit for This Trial?

This trial is for adults over 30 with Fuchs endothelial corneal dystrophy (FECD) who are scheduled for keratoplasty. They must have a positive TCF4 allele and be able to consent. Pregnant or breastfeeding women, contact lens users, and those on topical steroids or other studies can't join.

Inclusion Criteria

I have a confirmed genetic mutation related to TCF4.
I am scheduled for a cornea transplant due to Fuchs' dystrophy.
I can sign and understand the consent form.
See 2 more

Exclusion Criteria

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
I am pregnant, planning to become pregnant, or am breastfeeding.
See 7 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive DT-168 Ophthalmic Solution and are assessed for pharmacodynamics, safety, and tolerability

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • DT-168
Trial Overview The study tests DT-168 Ophthalmic Solution in patients with FECD before they undergo keratoplasty. It aims to assess how the drug works in the body, its safety, and if it's easy to tolerate without causing discomfort.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: ActiveExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Design Therapeutics, Inc.

Lead Sponsor

Trials
4
Recruited
130+

Citations

News Release DetailsDesign plans to conduct a Phase 2 biomarker trial of DT-168 to evaluate safety, tolerability, and corneal endothelium biomarkers in patients with FECD.
Promising Phase 1 Results for DT-168 in Fuchs ...The trial in healthy volunteers showed the drug was well-tolerated with no serious or ocular adverse events.
Design Therapeutics to Present Phase 1 Data for Fuchs ...We believe DT-168, formulated as an eye drop, has the potential to restore endothelial function, representing a significant advancement in the ...
Genetic Therapy of Fuchs Endothelial Corneal DystrophyDT-168 significantly reduced nuclear foci to levels seen in cells cultured from healthy individuals.
Design reports positive outcomes from trial of DT-168 eye ...Design Therapeutics has reported positive outcomes from the double-masked, randomised Phase I trial of DT-168 in healthy volunteers.
Design Therapeutics To Reveal Phase 1 Safety Data For ...DT-168 is a GeneTAC small-molecule therapy formulated as an eye drop for the treatment of Fuchs endothelial corneal dystrophy or FECD, a ...
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