Clinical Trials

A clinical trial is a research study done in humans to evaluate the effect of an intervention on a person’s health.  Part of LMRI’s mission is to develop new therapies to treat MacTel. To that end, the Lowy Medical Research Institute sponsors clinical trials and clinical studies to develop treatments for MacTel.  Although currently there are no approved treatments for MacTel, the Lowy Medical Research Institute is working to test new therapies with the hope to bring them to clinics through its clinical trials program.

Clinical trials participants must enroll in the MacTel Registry, and must meet eligibility criteria for the study.

Current Trials:

Phase 3 NT-501 Trial

CNTF-Secreting Cells for Treatment of MacTel

Neurotech Pharmaceuticals, with support from the Lowy Medical Research Institute, is testing the safety and efficacy of ciliary neurotrophic factor (CNTF) in patients with macular telangiectasia type 2 in a Phase 3 clinical trial.  This is a multi-center trial, with clinical sites in the United States, Europe, and Australia.

Ciliary neurotrophic factor is a therapeutic macromolecule that has been tested in numerous preclinical applications for its ability to decrease photoreceptor degeneration in disease models of the eye. Among the many growth factors, cytokines, and neurotrophic factors that have been tested for their ability to minimize photoreceptor loss, CNTF is among the most effective. However, CNTF degrades relatively quickly when injected into the eye. Therefore, it requires a special delivery device.

Neurotech developed an encapsulated cell technology implant (NT-501) to address this problem. The device is surgically implanted into the vitreous, which is the clear jelly-like substance that fills the area between the retina and the lens. The NT-501 device continuously delivers CNTF into the vitreous cavity of the eye. It results in controlled, long-term delivery of CNTF. From the vitreous, CNTF can diffuse to the retina, which is the part of the eye affected by MacTel.

Based on promising results from the Phase 1 and Phase 2 trials, LMRI and Neurotech are conducting a Phase 3 clinical trial. Participants enrolled in the Phase 3 trial will be followed for two years with a total of nine visits. This is a Treatment/Sham (placebo) study.

The Phase 1 and Phase 2 participants continue to be followed in a long-term extension study.

Enrollment in the Phase 3 trial is now closed.

Laser Study

The Lowy Medical Research Institute is sponsoring a pilot study to test subthreshold photothermal treatment in macular telangiectasia type 2. The purpose of this study, which employs a FDA approved laser system, is to induce a “healing” response in the treated portion of the macula. Participants will be randomized to either treatment or sham (placebo) procedure, in one eye.  The participants will be followed for one year with a total of six visits.

The MacTel Laser Study is registered with, study ID NCT03321916

This study is being conducted at the Medical College of Wisconsin. The Laser Study is currently enrolling.  If you are interested in participating in or would like more information about the Laser Study, contact LMRI.