Clinical Research Overview
Overview of MacTel Clinical Research
The Lowy Medical Research Institute, through the MacTel Project, places a major focus on the power of clinical studies to advance the understanding of MacTel. This focus has been largely successful to date; it has redefined the disease and has brought a therapy to clinical trials.
The MacTel Project’s Natural History Observation Study played a major role in improving the diagnosis of MacTel and the understanding of the disease. The Natural History Study enrolled more than 400 individuals with MacTel from around the world. Participants had annual clinic visits for five years. At these visits, their health and MacTel progression were recorded. This detailed clinical history, from very early to more advanced stages of disease, has led to new disease insights.
The Lowy Medical Research Institute also sponsors a Natural History Observation Registry. The registry involves an initial clinic visit at the time of enrollment, and an annual telephone survey. Participants in the registry are providing valuable clinical information about the progression of their disease. They are also connected to the opportunity to participate in laboratory research and clinical trials.
One of the objectives of The Lowy Medical Research Institute is to bring new therapies to the clinic to treat MacTel. To that end, LMRI is sponsoring Phase 1 and Phase 2 clinical trials to test the safety and efficacy of ciliary neurotrophic factor (CNTF) in patients with MacTel. These trials are ongoing.