Ciliary neurotrophic factor for macular telangiectasia type 2: results from a phase 1 safety trial.
Details
Serval ID
serval:BIB_5CC7619927A3
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Ciliary neurotrophic factor for macular telangiectasia type 2: results from a phase 1 safety trial.
Journal
American journal of ophthalmology
Working group(s)
MacTel-CNTF Research Group
Contributor(s)
Schwartz S., Hubschman J.P., Kreiger A., McCannel T., Heilweil G., Udonetok J., Cupp D., Hosseini H., Wong R., Itty S., Hitchcock L., Ostrick R., Zelcer N., Kageyama J., Chun M., Shokouh B., Davila N., Almanzor R., Fash L., Bruce R., Nusinowitz S., Sanguinet J., Singerman L., Novak M., Zegarra H., Zakov Z., Pendergast S., Miller D., Coney J., Schartman J., Carson G.M., Peck J., James M., Rath S., Weiss D., Himmelman D., Stone L., Nitzche T., DuBois K., Pelton S., Tanner V., Holder G., Peto T., Sallo F.B., Leung I., Clemons T., Figueroa M., Rosenberg D., Musch D., Blumenkranz M., Flynn H., Bird A., Chew E., Friedlander M., Oswald Q., Schiffman R., Small R., Trombley J.
ISSN
1879-1891 (Electronic)
ISSN-L
0002-9394
Publication state
Published
Issued date
04/2015
Peer-reviewed
Oui
Volume
159
Number
4
Pages
659-666.e1
Language
english
Notes
Publication types: Clinical Trial, Phase I ; Journal Article ; Multicenter Study ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Publication Status: ppublish
Abstract
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.
An open-label safety trial conducted in 2 centers enrolling 7 participants with macular telangiectasia type 2.
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.
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.
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.
An open-label safety trial conducted in 2 centers enrolling 7 participants with macular telangiectasia type 2.
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.
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.
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.
Keywords
Aged, Autoantibodies/immunology, Ciliary Neurotrophic Factor/administration & dosage, Ciliary Neurotrophic Factor/adverse effects, Ciliary Neurotrophic Factor/immunology, Drug Delivery Systems, Electroretinography, Enzyme-Linked Immunosorbent Assay, Female, Fluorescein Angiography, Humans, Intravitreal Injections, Male, Middle Aged, Retina/physiopathology, Retinal Telangiectasis/immunology, Retinal Telangiectasis/physiopathology, Retinal Telangiectasis/therapy, Tomography, Optical Coherence, Visual Acuity/physiology, Visual Fields/physiology
Pubmed
Web of science
Create date
26/09/2024 19:19
Last modification date
27/09/2024 15:46
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