title: Ciliary neurotrophic factor for macular telangiectasia type 2: results from a phase 1 safety trial.
abstract: OBJECTIVE
  To evaluate the safety and tolerability of intraocular delivery of ciliary neurotrophic factor (CNTF) using an encapsulated cell implant for the treatment of macular telangiectasia type 2.
  
  
  METHODS
  An open-label safety trial conducted in 2 centers enrolling 7 participants with macular telangiectasia type 2.
  
  
  METHODS
  The participant's more severely affected eye (worse baseline visual acuity) received the high-dose implant of CNTF. Patients were followed for a period of 36 months. The primary safety outcome was a change in the parameters of the electroretinogram (ERG). Secondary efficacy outcomes were changes in visual acuity, en face measurements of the optical coherence tomography of the disruption in the ellipsoid zone, and microperimetry when compared with baseline.
  
  
  RESULTS
  The ERG findings demonstrated a reduction in the amplitude of the scotopic b-wave in 4 participants 3 months after implantation (month 3). All parameters returned to baseline values by month 12 and remained so at month 36 with no clinical impact on dark adaptation. There was no change in visual acuity compared with baseline. The area of the defect as measured functionally by microperimetry and structurally by the en face OCT imaging of the ellipsoid zone loss appeared unchanged from baseline.
  
  
  CONCLUSIONS
  The intraocular delivery of CNTF in the encapsulated cell implant appeared to be safe and well tolerated in eyes with macular telangiectasia type 2. Further evaluation in a randomized controlled clinical trial is warranted to test for efficacy.
authors.affiliation: National Eye Institute, National Institutes of Health, Bethesda, Maryland. Electronic address: echew@nei.nih.gov.
authors.forename: Emily Y
authors.initials: EY
authors.lastname: Chew
authors.affiliation: The EMMES Corporation, Rockville, Maryland.
authors.forename: Traci E
authors.initials: TE
authors.lastname: Clemons
authors.affiliation: National Institute of Health Research Biomedical Research Centre at Moorfields Eye Hospital National Health Service Foundation Trust and UCL Institute of Ophthalmology, London, United Kingdom.
authors.forename: Tunde
authors.initials: T
authors.lastname: Peto
authors.affiliation: Department of Research and Development, Moorfields Eye Hospital, London, United Kingdom; University College London, Institute of Ophthalmology, London, United Kingdom.
authors.forename: Ferenc B
authors.initials: FB
authors.lastname: Sallo
authors.affiliation: ATIS Consultants, Scarsdale, New York.
authors.forename: Avner
authors.initials: A
authors.lastname: Ingerman
authors.affiliation: Neurotech Pharmaceuticals, Cumberland, Rhode Island.
authors.forename: Weng
authors.initials: W
authors.lastname: Tao
authors.affiliation: Retina Associates of Cleveland, Cleveland, Ohio.
authors.forename: Lawrence
authors.initials: L
authors.lastname: Singerman
authors.affiliation: University of California Los Angeles, Los Angeles, California.
authors.forename: Steven D
authors.initials: SD
authors.lastname: Schwartz
authors.affiliation: Cleveland Clinic, Department of Ophthalmology, Cleveland Ohio; Louis Stokes Cleveland VA Medical Center, Cleveland, Ohio.
authors.forename: Neal S
authors.initials: NS
authors.lastname: Peachey
authors.affiliation: Department of Inherited Eye Disease, Moorfields Eye Hospital, London, United Kingdom.
authors.forename: Alan C
authors.initials: AC
authors.lastname: Bird
mesh_terms: D000368:Aged; D001323:Autoantibodies / Q000276:immunology; D020934:Ciliary Neurotrophic Factor / Q000008:administration & dosage* / Q000009:adverse effects / Q000276:immunology; D016503:Drug Delivery Systems; D004596:Electroretinography; D004797:Enzyme-Linked Immunosorbent Assay; D005260:Female; D005451:Fluorescein Angiography; D006801:Humans; D058449:Intravitreal Injections; D008297:Male; D008875:Middle Aged; D012160:Retina / Q000503:physiopathology*; D058456:Retinal Telangiectasis / Q000276:immunology / Q000503:physiopathology / Q000628:therapy*; D041623:Tomography, Optical Coherence; D014792:Visual Acuity / Q000502:physiology; D014794:Visual Fields / Q000502:physiology
chemical_list: D001323:Autoantibodies; D020934:Ciliary Neurotrophic Factor