DM Corneal Onlay Transplant for Limbal Stem Cell Deficiency

Overseen By Meaghyn Kramer, BA

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Minnesota

Disqualifiers: Pregnancy, Prisoners, Consent incapacity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

Duration: Immediate post-operative period

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment for Limbal Stem Cell Deficiency (LSCD), a condition that can lead to blindness. The researchers focus on using Descemet's Membrane (DM) as a corneal onlay to aid healing and restore vision. The trial includes two groups: one for individuals with partial LSCD and another for those with total LSCD and recurring issues like frequent eye surface damage. Participants should have significant vision problems and meet specific criteria regarding the extent of eye involvement. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this innovative treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that this DM corneal onlay is safe for treating LSCD?

Research has shown that Descemet's Membrane (DM) is generally safe in similar eye procedures. In treatments like Descemet Membrane Endothelial Keratoplasty (DMEK), which use similar methods, DM is often well-tolerated. However, some patients in these procedures have experienced significant vision loss.

This study explores the use of DM as a corneal onlay, a novel approach for this specific condition. As an early-phase trial, it focuses on testing safety and determining the right dose, with close monitoring of any side effects or risks.

While DM appears promising, more research is needed to fully understand its safety for this new use. Participants will be closely monitored to ensure any adverse effects are promptly addressed. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Researchers are excited about the Descemet's Membrane corneal onlay because it offers a fresh approach to treating limbal stem cell deficiency (LSCD). Unlike the standard care, which often involves stem cell transplants or donor tissue, this treatment uses a corneal onlay. This onlay is a thin, transparent layer that can be placed directly onto the damaged cornea, potentially promoting healing and restoring vision. The direct application method of the corneal onlay could mean less invasive procedures and faster recovery times for patients, making it a promising alternative to current LSCD treatments.

What evidence suggests that the Descemet's Membrane corneal onlay could be an effective treatment for limbal stem cell deficiency?

Research suggests that Descemet's Membrane (DM) could be a promising treatment for limbal stem cell deficiency (LSCD). In this trial, participants will receive the DM corneal onlay transplant. DM, a natural part of the eye, shares important similarities with the area where stem cells grow, which is essential for healthy eye function. It contains proteins like vitronectin and BM40/SPARC that aid in stem cell growth and multiplication. These proteins play a crucial role in healing the eye's surface. Although no human clinical data exists yet, the similarities between DM and the stem cell area suggest it might help restore vision by promoting healing in LSCD patients. ^{1 2 3 4 6}

Who Is on the Research Team?

Stephen Kaufman, MD

Principal Investigator

University of Minnesota

Are You a Good Fit for This Trial?

This trial is for people with Limbal Stem Cell Deficiency (LSCD), a condition that can cause blindness, who have significant vision loss (20/100 or worse). It's suitable for those with partial LSCD affecting less than 75% of the corneal surface and those with more severe cases involving over 75%, experiencing frequent erosions or persistent defects despite treatment.

Inclusion Criteria

My eye condition affects less than 75% of the corneal surface and my vision is 20/100 or worse.

I have severe eye surface damage with frequent pain or vision worse than 20/100 despite treatment.

Exclusion Criteria

Pregnant women

Prisoners (vulnerable population)

I am unable to make medical decisions for myself.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Descemet's membrane as a corneal on-lay to promote corneal re-epithelialization in partial LSCD

Immediate post-operative period

1 visit (in-person) for the procedure

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of corneal neovascularization, epitheliopathy, graft retention, and visual improvement

24 weeks

Visits at week 1, month 1, month 3, and month 6

What Are the Treatments Tested in This Trial?

Interventions

• Descemet's Membrane corneal onlay

Trial Overview The study tests transplanting Descemet's Membrane (DM) onto the eye's surface as a potential new treatment. DM may serve as a replacement 'niche' to support limbal stem cells in patients with partial or near-total LSCD, aiming to promote healing and improve vision.

How Is the Trial Designed?

Group I: Visually significant partial LSCD Experimental Treatment 1 Intervention

Patient with visually significant partial LSCD, as defined by a best corrected visual acuity of 20/100 or less, and partial LSCD on slit lamp exam with at least 25% of the limbus intact or at least 25% of the corneal surface covered with corneal epithelium will be enrolled in the first arm.

Group II: Total/near-total LSCD with recurrent or persistent epithelial defects (PED) Experimental Treatment 1 Intervention

Patient with visually significant total LSCD, as defined by a best corrected visual acuity of 20/100 or less, and total LSCD on slit lamp exam with over 25% of the limbus intact or less than 25% of the corneal surface covered with corneal epithelium; and a history of a persistent epithelial defect that has persisted over 2 weeks despite maximal medical therapy, or a history of recurrent epithelial erosions that occur more frequently than once a month; will be enrolled in the second arm.

Who Is Running the Clinical Trial?

University of Minnesota

Lead Sponsor

Trials

1,459

Recruited

1,623,000+

Published Research Related to This Trial

In a study of 100 Indian eyes undergoing Descemet Membrane Endothelial Keratoplasty (DMEK), the procedure demonstrated safety and effectiveness, with 57.6% of patients achieving a Best Spectacle Corrected Visual Acuity (BSCVA) of ≥20/25 after three months.

The mean endothelial cell loss was 26.92%, which is considered acceptable, and complications were relatively low, with only 8% experiencing partial graft detachment and one case of primary graft failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Outcomes of Descemet Membrane Endothelial Keratoplasty (DMEK) Using Surgeon's Prepared Donor DM-Roll in Consecutive 100 Indian Eyes. Basak, SK., Basak, S., Pradhan, VR.[2022]

The novel simple limbal epithelial transplantation (SLET) technique was evaluated in four patients with total unilateral limbal stem cell deficiency, showing promising results with a completely avascular corneal surface in two patients after 6 months.

No serious complications were reported from the surgery, indicating that SLET is a safe option for treating limbal stem cell deficiency, although one patient experienced issues with graft adherence.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of surgical outcomes of limbal transplantation using simple limbal epithelial transplantation technique in patients with total unilateral limbal deficiency. Queiroz, AG., Barbosa, MM., Santos, MS., et al.[2017]

In a study involving 10 human cadaver eyes and 1 patient, Descemet membrane endothelial keratoplasty (DMEK) successfully maintained the position of the transplanted donor Descemet membrane after a 30-minute air-fill, indicating effective surgical technique.

The patient achieved excellent visual acuity of 20/20 one week post-transplant, demonstrating that DMEK can provide rapid visual rehabilitation for corneal endothelial disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Descemet membrane endothelial keratoplasty (DMEK). Melles, GR., Ong, TS., Ververs, B., et al.[2022]

In a study involving 15 human eye bank eyes, a new technique for transplanting Descemet's membrane (DM) was developed, which successfully maintained low levels of astigmatism post-surgery, averaging only 1.0 D.

The transplanted DM retained an intact endothelial cell layer with minimal cell death (3.4%), indicating that the procedure is effective and preserves the health of the donor tissue.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transplantation of Descemet's membrane carrying viable endothelium through a small scleral incision. Melles, GR., Lander, F., Rietveld, FJ.[2022]

Decellularised human limbus (DHL) maintains essential structural and biochemical properties of the limbal niche, including key basement membrane proteins, making it a promising carrier for limbal epithelial stem cell (LESC) transplantation.

The decellularisation process effectively removed DNA and immune cells from the DHL, demonstrating its safety and lack of cytotoxicity, which is crucial for successful transplantation outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Generation and characterisation of decellularised human corneal limbus. Spaniol, K., Witt, J., Mertsch, S., et al.[2018]

Allogeneic limbal transplantation (ALT) was performed on 22 patients with limbal stem cell deficiency (LSCD), resulting in successful integration of the transplanted tissue into the cornea, as confirmed by anterior segment optical coherence tomography (AS-OCT).

Post-surgery, patients experienced a significant improvement in visual acuity, with scores improving from an average of 2.06 to 1.44 logMAR over a mean follow-up period of 19 months, indicating the efficacy of ALT in treating LSCD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Allogeneic Limbal Transplants Integrate into the Corneal Surface and Lead to an Improved Visual Acuity. Kesper, C., Heinzelmann, J., Viestenz, A., et al.[2023]

The deep anterior lamellar limbo-keratoplasty (DAL-LK) technique effectively managed bilateral limbal stem cell deficiency (LSCD) with corneal scarring in two patients, resulting in significant visual acuity improvements (20/80 and 20/60) over follow-up periods of 2.5 and 3 years, respectively.

DAL-LK offers a safer, single-stage solution for LSCD by simultaneously transplanting corneal stroma and limbal stem cells, preserving the host endothelium and eliminating the risk of rejection, which is a common issue with traditional methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Deep anterior lamellar limbo-keratoplasty for bilateral limbal stem cell deficiency with corneal scarring in chemical injury sequelae: Two case reports. Jain, N., Kate, A., Basu, S.[2023]

The use of a novel blunt instrument for preparing donor corneas in Descemet membrane endothelial keratoplasty improved visibility of the donor tissue without causing structural damage, as confirmed by transmission electron microscopy.

Endothelial cell loss was lower in the group using the new instrument (63 cells/mm²) compared to the traditional method (116 cells/mm²), suggesting that this tool may help minimize tissue loss during graft preparation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Instrument to Enhance Visualization of Descemet Membrane During Graft Preparation for DMEK Surgery. Menzel-Severing, J., Salla, S., Plum, WJ., et al.[2017]

In a study of 40 patients undergoing Descemet's membrane endothelial keratoplasty (DMEK) for Fuchs' endothelial dystrophy, the learning curve showed that the last 13 procedures (33%) did not require rebubbling, indicating improved surgical proficiency over time.

After 3 months, 86% of the patients achieved a visual acuity of 0.5 or better, demonstrating that DMEK is a safe and effective procedure for restoring vision in patients with endothelial dysfunction.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The learning curve for Descemet membrane endothelial keratoplasty performed by two experienced corneal surgeons: a consecutive series of 40 cases. Wubbels, RJ., Remeijer, L., Engel, A., et al.[2020]

Descemetectomy, which involves the targeted removal of Descemet's membrane, is proposed as a new treatment option for endothelial diseases like Fuchs endothelial dystrophy, potentially reducing the need for corneal transplants.

This approach offers a promising alternative to transplantation, addressing the growing demand for human corneal transplants while providing a direct intervention for specific corneal conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Descemetectomy : An alternative to transplantation?] Bramann, EL., Schröder, K., Schrader, S., et al.[2018]

A novel limbal transplantation technique was successfully performed on six patients with limbal stem cell deficiency, resulting in a completely healed and stable corneal surface within 6 weeks, maintained for an average of 9.2 months.

Visual acuity significantly improved in 66.6% of the patients, demonstrating the technique's efficacy while requiring less donor tissue and avoiding the need for specialized cell expansion laboratories.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Simple limbal epithelial transplantation (SLET): a novel surgical technique for the treatment of unilateral limbal stem cell deficiency. Sangwan, VS., Basu, S., MacNeil, S., et al.[2022]

Descemet's membrane endothelial keratoplasty (DMEK) significantly improved visual acuity in patients with endothelial cell decompensation, with a final mean LogMAR visual acuity of 0.32 after surgery compared to 0.87 preoperatively, indicating effective visual rehabilitation.

Despite a notable complication rate of transplant dislocation in 31% of cases, the overall safety profile was strong, with no serious complications like elevated intraocular pressure or endophthalmitis reported, supporting DMEK as a gold standard treatment for endothelial diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical results after Descemet membrane endothelial keratoplasty]. Yoeruek, E., Bayyoud, T., Röck, D., et al.[2012]

In a study comparing two graft implantation techniques for Descemet membrane endothelial keratoplasty (DMEK) involving 40 patients, the technique using a DMEK injection cartridge resulted in a significantly longer insertion-to-attachment time and lower initial correct graft orientation compared to a standard lens injection cartridge.

Despite differences in insertion technique, both groups showed no significant differences in central corneal thickness, endothelial cell count, or best spectacle-corrected visual acuity after 12 months, indicating that while the technique may affect initial graft placement, it does not impact long-term outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Flushing Versus Pushing Technique for Graft Implantation in Descemet Membrane Endothelial Keratoplasty. Siebelmann, S., Janetzko, M., König, P., et al.[2021]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05909735?cond=%22Aniridia%22

Study Details | NCT05909735 | Treatment of LSCD With DM 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.

2. withpower.com withpower.com/trial/phase-1-limbal-stem-cell-deficiency-lscd-6-2023-82f47

DM Corneal Onlay Transplant for Limbal Stem Cell ... This Phase 1 medical study run by University of Minnesota is evaluating whether Descemet's Membrane corneal onlay will have tolerable side effects ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12081055/

Decellularized Descemet membrane anterior keratoplasty with ... Limbal stem cell deficiency (LSCD) occurs from deficiency or dysfunction of the LSCs located along the Palisades of Vogt. LSCs are responsible for generating ...

4. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/558855

Treatment of LSCD With DM - Carebox Connect Limbal Stem Cell Deficiency (LSCD) is a blinding disease that accounts for an estimated 15-20% of corneal blindness worldwide.

5. restoresight.org restoresight.org/wp-content/uploads/2025/10/2025-CEBF-Program-FINAL.pdf

FINAL PROGRAM Purpose: Descemet's Membrane Anterior Keratoplasty (DMAK) is a promising treatment for limbal stem cell deficiency and other ocular surface disorders. We ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38713471/

Decellularized Descemet Membrane Anterior Keratoplasty ... The purpose of this study was to describe the use of Descemet membrane anterior keratoplasty (DMAK) with modified allogeneic simple limbal epithelial ...