Phase 2 Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel) (14-EI-0078, 17-EI-0136)

National Eye Institute

Investigators

Linked publications & trials

Abstract

We initiated this project at the NEI in fiscal year 2014 as a site in the phase 2 clinical trial (14-EI-0078) testing CNTF for macular telangiectasia (MacTel). We met the accrual goal at the NEI site and study analysis has been completed, with publication of the primary results. The trial met its primary endpoint, with eyes receiving sham treatment showing 31% greater progression of neurodegeneration than the CNTF-treated eyes using an en face OCT measure of change in area of outer retinal disruption, with difference in mean area of photoreceptor loss of 0.05 0.03 mm2 (p = 0.04) at 24 months. The phase 2 protocol has been terminated following completion. Participants in the phase 2 trial were enrolled in an extension study (17-EI-0136) to study effects of the CNTF implant at years 3, 4, 5, and 6. We opened a sub-study (19-EI-0045) to allow implantation of the CNTF-secreting device (NT-501; Renexus) in participants who received only the sham-surgery in the phase 2 trial, and implanted the device in two participants. Follow up in the extension study and sub-study are now complete (last study visit at the NIH site, 3/29/2021) and the multicenter study has recently been closed by the sponsor. In 2018, we were activated as a site in the phase 3 clinical trial (18-EI-0055) testing CNTF (NT-501; Renexus) for MacTel (listed as a separate project). This will be reported in the next few months. We achieved primary endpoint and the implant appears to be beneficial in reducing the progression of photoreceptor loss. This will be filed with the FDA soon.