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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:

 

MacTel Disease Progression Sub-Study:

LMRI is conducting a long-term study to better understand how MacTel Type 2 develops in patients over time, with the objective of establishing reliable ways to measure disease progression and identify new areas for treatment.

Although MacTel generally progresses slowly, the rate and characteristics of progression can vary from person to person. By following the same group of people with MacTel over several years, researchers can better understand how the disease changes over time and identify factors that may influence its progression.

During the study, researchers will collect a wide range of biological data, including measurements from blood samples, measurements of retinal changes seen in image scans, and other health indicators. Collecting different types of biological information together provides a more complete picture of what is happening in the body and may help identify markers that predict disease progression or lead to future treatments.

This study is open to people with a diagnosis of MacTel who previously participated in the MacTel Project Natural History Observation Registry (NHOR).  At least two years must have elapsed between their NHOR visit and their participation in the Disease Progression Study.

The following sites are participating in this study:

Centre for Eye Research Australia (Australia), Hôpital Lariboisière Service d’Ophtalmologie (France), Moorfields Eye Hospital (England), Save Sight Institute (Australia), St. Franziskus Hospital (Germany), University of Bonn (Germany), Oxford Eye Hospital (England) and University of Freiburg (Germany).

 

Tamoxifen Retinopathy Sub-Study:

LMRI is currently sponsoring a study that explores whether long-term use of tamoxifen, a common hormone therapy for breast cancer patients, may affect the body in ways that resemble Macular Telangiectasia Type 2 (MacTel).

Tamoxifen is widely used in the treatment of breast cancer and is typically prescribed for a minimum of five years. Although it is highly effective at slowing or preventing the growth of certain breast cancers, up to 12% of patients taking tamoxifen long term develop changes in the retina that can affect vision. These retinal changes are of interest to LMRI as they can look similar to retinal changes seen in people with MacTel. The underlying causes of these shared retinal changes are not yet understood.

Everyone has small molecules circulating in their blood. These are called serum metabolites. MacTel patients share some common characteristics in the levels of metabolites circulating in their blood. This study aims to determine if breast cancer patients taking tamoxifen have this same metabolic pattern that has been observed in MacTel patients. Researchers hope to better understand why tamoxifen-related retinal changes develop and what role these metabolites might play in eye disease. This research could improve our understanding of both conditions.

This study is currently enrolling at University of California, San Diego. Eligible participants will have a diagnosis of breast cancer and are either taking tamoxifen long-term or have never taken tamoxifen.

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 clinicaltrials.gov, Study ID NCT04907084

The study was conducted at several MacTel Registry sites.  See the clinicaltrials.gov page for details.

The SAFE study (Serine and Fenofibrate in Patients with MacTel Type 2 Study) has now closed. At this time, LMRI and its collaborating investigators are analyzing the results and preparing for publication. Updates will be posted as soon as findings are available to the public.

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 clinicaltrials.gov, study ID NCT03321916

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