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 and Completed 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.

Serine Fenofibrate Study

The Serine and Fenofibrate study (SAFE study) is a Phase 2a study to investigate the effect of serine and/or fenofibrate on deoxysphingolipid levels in patient serum.

Study rationale:  Research by LMRI scientists and collaborators determined that low serine levels play a role in MacTel, leading to elevated levels of toxic lipids (deoxysphingolipids).  The SAFE study will determine if serine supplementation and/or fenofibrate administration has an effect on serine and/or deoxysphingolipid levels in patient serum.

In this study, patients are randomly assigned to one of six groups:  no treatment, low dose serine, high dose serine, fenofibrate, low dose serine and fenofibrate, high dose serine and fenofibrate.  This is a 10-week study.

This study is registered with, Study ID NCT04907084

The study was conducted at several MacTel Registry sites.  See the page for details.  This study is closed.

Laser Study

The Lowy Medical Research Institute sponsored 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 were randomized to either treatment or sham (placebo) procedure, in one eye.  The participants were followed for one year with a total of six visits.

The MacTel Laser Study is registered with, study ID NCT03321916

This study was conducted at the Medical College of Wisconsin. The Laser Study is completed.