Clinical Trial Involvement

Center for NeuroGenetics Trials       

There are many different types of clinical trials, each involving a different approach, from simply monitoring your disease symptoms over time (called natural history studies), to determining if new or repurposed drugs are safe to take (phase I trial), to testing if the treatment works (phase II trial) to finally seeing if the treatment is better than what is currently available (phase III trial).

At the Center for NeuroGenetics, our clinicians and researchers are involved in all aspects of clinical trials. Please refer to the list below when searching for active CNG clinical trials.

CNG Studies and Trials

DIsease Study title (click for more information)
C9orf72 ALS Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD.
Contact: Deborah Morrison,, (352) 273-5189
Primary Lateral Sclerosis Use of Dalfampridine in Primary Lateral Sclerosis
Contact: Jessica Magenheim,, (352) 273-5550
Friedreich’s Ataxia Biomarkers in Friedreich’s Ataxia
Contact: Samantha Norman,, (352) 273-8218
SCA Types 1, 2, 3 and 6 Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias
Contact: S.H. Subramony,, (352) 273-5000
Charcot-Marie-Tooth Disease Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
Contact: Amanda Cowsert,, (352) 733-2435


What is a Clinical Trial?

Clinical trials are a type of research that test ways to prevent, detect, or treat disease and are at the heart of all medical advances. Clinical trials are conducted in phases and are considered the only real way to prove whether a medical approach works and is safe for patients. Clinical trial participation, especially in the diseases we study at the CNG, can provide important advances in our knowledge and possible treatment of neurodegenerative diseases. 

Still curious about the clinical trial process? Click the links below to find more information on:

What are the four phases of clinical trials?
Why participate in a clinical trial?
Why are older and diverse participants important in clinical research?
What happens when a clinical trial or study ends?
Questions to ask before participating in a clinical trial.

What if I’m not eligible for an active CNG trial or study?

Feel free to use to find a trial or study that’s suitable for you. Trials and studies are a critical step in bringing life-saving treatments to those in need. Even if the trial is not with or through the CNG, we applaud every patient who makes the decision to contribute to science and the broader community!