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Stem Cell Therapy for Limbal Stem Cell Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03957954
Recruitment Status : Recruiting
First Posted : May 21, 2019
Last Update Posted : January 11, 2023
Sponsor:
Collaborators:
National Eye Institute (NEI)
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Sophie Deng, MD, PhD, University of California, Los Angeles

Study Description
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Brief Summary:
This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events (safety).

Condition or disease Intervention/treatment Phase
Limbal Stem-cell Deficiency Biological: cLSC Device: Scleral contact lens (SCL) Phase 1

Detailed Description:
20 patients with severe to total Limbal Stem Cell Deficiency (LSCD) in one eye attributable to injury or ocular surgery. The first 5 eyes will receive the cLSCs to determine the feasibility and safety. Then the subsequent 15 eyes will be randomized into cLSC group and a scleral lens treatment (SCL) control group in a 2:1 ratio in blocks of 3 or 6 using a random number generator.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
Actual Study Start Date : September 30, 2020
Estimated Primary Completion Date : October 31, 2023
Estimated Study Completion Date : December 31, 2023
Arms and Interventions
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Arm Intervention/treatment
Experimental: Cultivated Limbal Stem-Cells (cLSC)
One dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter.
 Biological: cLSC
Transplantation of cLSC
Active Comparator: Scleral Contact Lens Device (SCL)
Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface.
 Device: Scleral contact lens (SCL)
Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface.


Outcome Measures
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Primary Outcome Measures :
  1. Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation [ Time Frame: 12 months for all participants ]
    Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.

  2. Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria [ Time Frame: 12 months for all participants ]
    Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.

  3. Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination [ Time Frame: 12 months for all participants ]
    Feasibility assessed by successful cLSC manufacturing without contamination.


Secondary Outcome Measures :
  1. Changes in the Area of Corneal Epithelial Defect [ Time Frame: At 6 months and 12 months in both cLSC and the control groups ]
    Evaluate changes in the area of corneal epithelial defect (ED) or lack of ED.

  2. Changes in the Clinical Score [ Time Frame: At 6 months and 12 months in both cLSC and the control groups ]
    Evaluate changes in the clinical score determined by slit lamp examination using fluorescein staining and confocal imaging.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Main inclusion Criteria:

  1. Male or female ≥18 years of age.
  2. Best corrected visual acuity in the affected eye of 20/200 or less.
  3. Documentation of a LSCD diagnosis and the central cornea is affected.
  4. Absence of lagophthalmos and eyelid abnormality
  5. Adequate forniceal depth is ≥ 5 mm.
  6. LSCD fails to resolve by surgical treatments of the ocular surface during the previous 6 months of screening visit.
  7. If the etiology of LSCD is due to chemical injury, a minimal interval of 1 year since the initial chemical injury is required.
  8. A Schirmer test result at 5 minute of ≥5 mm of wetting.
  9. Absence of active infectious keratitis in either eye at the Enrollment Visit.
  10. Have a life expectancy ≥ 2 years after enrollment.

Main exclusion Criteria:

  1. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
  2. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
  3. Exposure keratopathy or lagophthalmos of the study eye.
  4. Persistent severe ocular surface inflammation and/or meibomian gland dysfunction
  5. Chemical injury occurred less than 12 months ago.
  6. Presence of ocular surface tumor.
  7. Uncontrolled diabetes with last hemoglobin A1C (HgA1C) >8.5.
  8. Presence of known allergies to any of the cLSC components.
  9. Current participation in another simultaneous medical investigation or trial.
  10. Unable to be compliant with or complete the requirements of the study.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03957954


Contacts
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Contact: Clemence Bonnet, MD 310-794-7813 CBonnet@mednet.ucla.edu
Contact: Sofia Gudiel SGudiel@mednet.ucla.edu

Locations
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United States, California
University of California Recruiting
Los Angeles, California, United States, 90095
Contact: Clemence Bonnet, MD    310-794-7813      
Sponsors and Collaborators
University of California, Los Angeles
National Eye Institute (NEI)
California Institute for Regenerative Medicine (CIRM)
Investigators
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Principal Investigator: Sophie Deng, MD, PhD Stein Eye Institute UCLA
More Information
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Responsible Party: Sophie Deng, MD, PhD, Professor, University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT03957954    
Other Study ID Numbers: V001
R01EY021797 ( U.S. NIH Grant/Contract )
First Posted: May 21, 2019    Key Record Dates
Last Update Posted: January 11, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Additional relevant MeSH terms:
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Limbal Stem Cell Deficiency
Corneal Diseases
Eye Diseases