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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 21, 2020
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

Tracking Information
First Submitted Date  ICMJE February 21, 2019
First Posted Date  ICMJE May 21, 2019
Last Update Posted Date January 21, 2020
Estimated Study Start Date  ICMJE April 1, 2020
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 17, 2019)
  • 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.
  • 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.
  • Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination [ Time Frame: 12 months for all participants ]
    Feasibility assessed by successful cLSC manufacturing without contamination.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 17, 2019)
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Stem Cell Therapy for Limbal Stem Cell Deficiency
Official Title  ICMJE Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
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).
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Limbal Stem-cell Deficiency
Intervention  ICMJE
  • Biological: cLSC
    Transplantation of cLSC
  • Device: Scleral contact lens (SCL)
    Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface.
Study Arms  ICMJE
  • 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.
    Intervention: Biological: cLSC
  • Active Comparator: Scleral Contact Lens Device (SCL)
    Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface.
    Intervention: Device: Scleral contact lens (SCL)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 17, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2023
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clemence Bonnet, MD 310-794-7813 CBonnet@mednet.ucla.edu
Contact: Niree Dingizian NDingizian@mednet.ucla.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03957954
Other Study ID Numbers  ICMJE V001
R01EY021797 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sophie Deng, MD, PhD, University of California, Los Angeles
Study Sponsor  ICMJE University of California, Los Angeles
Collaborators  ICMJE
  • National Eye Institute (NEI)
  • California Institute for Regenerative Medicine (CIRM)
Investigators  ICMJE
Principal Investigator: Sophie Deng, MD, PhD Stein Eye Institute UCLA
PRS Account University of California, Los Angeles
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP