NCT05909735

Treatment of LSCD With DM

Completed Phase 1 Last updated 8 January 2026

What this trial tests

Phase 1 trial testing transplantation of a Descemet's Membrane corneal onlay, partial LSCD in Limbal Stem-cell Deficiency in 19 participants. Completed in 1 December 2025.

Timeline

30 November 2023

Primary endpoint
1 December 2025

1 December 2025

Quick facts

Lead sponsor	University of Minnesota
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	19
Start date	30 November 2023
Primary completion	1 December 2025
Estimated completion	1 December 2025
Sites	1 location across United States

Drugs / interventions tested

• transplantation of a Descemet's Membrane corneal onlay, partial LSCD
• transplantation of a Descemet's Membrane corneal onlay, total/near-total LSCD

Conditions studied

• Limbal Stem-cell Deficiency — all drugs for Limbal Stem-cell Deficiency →
• Congenital Aniridia — all drugs for Congenital Aniridia →

Sponsor

University of Minnesota

Who can join

18 and older, any sex, with Limbal Stem-cell Deficiency or Congenital Aniridia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Limbal Stem Cell Deficiency (LSCD) is a blinding disease that accounts for an estimated 15-20% of corneal blindness worldwide. Current treatments are limited. Traditional corneal transplantation with penetrating keratoplasty (PKP) is ineffective in treating these patients. Without a healthy population of limbal stem cells (LSC) to regenerate the corneal epithelium, standard corneal transplants will not re-epithelialize and will rapidly scar over or melt.

The limbal niche is the microenvironment surrounding the LSCs that is critical for maintaining their survival and proliferative potential under physiologic conditions. Extracellular signals from the microenvironment are critical to the normal function and maintenance of pluripotent stem cells. Identifying an effective niche replacement is thus an important focus of limbal stem cell research and critical for advancing treatments for LSCD.

Descemet's membrane (DM), an acellular, naturally occurring, basement membrane found on the posterior surface of the cornea, is a promising niche replacement. DM is routinely isolated and transplanted intraocularly with associated donor corneal endothelium for treatment of diseases like Fuchs' dystrophy and corneal bullous keratopathy that specifically affect DM and corneal endothelium. However, its application on the ocular surface has not been explored. DM is optically clear and highly resistant to collagenase digestion. This makes it very attractive as a long-term corneal on-lay and niche replacement on the surface of the eye. The anterior fetal banded layer of DM shares key compositional similarities with limbal basement membrane, which is a major component of the limbal niche. These similarities include limbus-specific extracellular matrix proteins such as collagen IV that is restricted to the α1, α2 subtypes, vitronectin, and BM40/SPARC. Of these, vitronectin and BM40/SPARC are known to promote proliferation of LSCs and induced pluripotent stem cells (iPSC) in culture.

Because of this, DM is a promising biological membrane for establishing a niche-like substrate on the corneal surface in patients with LSCD. The purpose of this pilot study is to investigate the clinical efficacy of using DM as a corneal on-lay to promote corneal re-epithelialization in partial LSCD.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Regenerative treatment of ophthalmic diseases with stem cells: Principles, progress, and challenges.
   Niu Y, Ji J, Yao K, Fu Q. · · 2024 · cited 25× · PMID 38586868 · DOI 10.1016/j.aopr.2024.02.001
2. Tackling visual impairment: emerging avenues in ophthalmology.
   Lin F, Su Y, Zhao C, Akter F, et al · · 2025 · cited 4× · PMID 40357281 · DOI 10.3389/fmed.2025.1567159