Clinical Trial: A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Prospective, Randomized, Double-Masked, Vehicle Controlled, Paired-Eye Phase 1/2 Clinical Study To Evaluate the Safety, Tolerability and Efficacy of Elamipretide Topical Ophthalmic Solution in Subje

Brief Summary: This is a Phase 1/2, prospective, randomized, double-masked, vehicle controlled, paired-eye study in approximately 16 subjects to evaluate safety, tolerability and efficacy of elamipretide 1.0% topical ophthalmic solution in patients with FCED presenting with mild to moderate corneal edema.

Detailed Summary:
Sponsor: Stealth BioTherapeutics Inc.

Current Primary Outcome:

  • The incidence and severity of systemic and ocular adverse events (AEs) [ Time Frame: Baseline to Week 16 (follow-up visit) ]
  • Change from Baseline in findings from slit lamp examinations (SLE) [ Time Frame: Baseline to Week 16 (follow-up visit) ]
  • Change from Baseline in intraocular pressure (IOP) using Goldmann applanation tonometry [ Time Frame: Baseline to Week 16 (follow-up visit) ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change from Baseline in central corneal thickness [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale scale) [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell count [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell morphology: hexagonality using specular microscopy [ Time Frame: Baseline to Week 12 ]
    Qualitative assessment of appearance of the cells
  • Change from Baseline in endothelial cell morphology: cell density (cells/mm2) using specular microscopy [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell morphology: coefficient of variation (CV%) using specular microscopy [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in corneal area affected by microcysts (mm2) [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in corneal bullae [ Time Frame: Baseline to Week 12 ]
    Descriptive assessment made by Investigator
  • Change from Baseline in severity of corneal stromal folds [ Time Frame: Baseline to Week 12 ]
    Descriptive assessment made by Investigator; severity is not assessed using a scale.
  • Change from Baseline in Contrast Sensitivity using VectorVision's CSV-1000 instrument [ Time Frame: Baseline and Week 12 ]


Original Secondary Outcome:

  • Change from Baseline in central corneal thickness [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale scale) [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell count [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell morphology: hexagonality using specular microscopy [ Time Frame: Baseline to Week 12 ]
    Qualitative assessment of appearance of the cells
  • Change from Baseline in endothelial cell morphology: cell density (cells/mm2) using specular microscopy [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in endothelial cell morphology: coefficient of variation (CV%) using specular microscopy [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in corneal area affected by microcysts (mm2) [ Time Frame: Baseline to Week 12 ]
  • Change from Baseline in corneal bullae [ Time Frame: Baseline to Week 12 ]
    Descriptive assessment made by Investigator
  • Change from Baseline in severity of corneal stromal folds [ Time Frame: Baseline to Week 12 ]
    Descriptive assessment made by Investigator
  • Change from Baseline in Contrast Sensitivity using VectorVision's CSV-1000 instrument [ Time Frame: Baseline and Week 12 ]


Information By: Stealth BioTherapeutics Inc.

Dates:
Date Received: December 20, 2015
Date Started: December 2015
Date Completion: November 2016
Last Updated: October 12, 2016
Last Verified: October 2016