A clinical trial is a research study involving human volunteers, and is designed to answer specific health questions. Carefully conducted clinical trials are the safest and fastest way to find effective treatments, and new ways to improve health.
You can search for all studies that are currently enrolling participants at the University of Rochester by typing in keywords in the search box below. If you don’t find a study that interests you right now, consider signing up for our Volunteer Registry, which will notify you of future studies.
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Multi-Center Study of ManNAc for GNE Myopathy
Lead Researcher: Johanna Hamel
GNE myopathy is a rare genetic muscle disease characterized by progressive muscle
atrophy and
weakness. The disease is caused by mutations in the gene that encodes the enzyme that
initiates and regulates N-acetylneuraminic acid (Neu5Ac) biosynthesis and glycan sialylation.
Currently, there is no therapy available for this disease. N-Acetylmannosamine (ManNAc),
an
orphan drug in development for GNE myopathy, is an uncharged monosaccharide and the
first
committed precursor in Neu5Ac biosynthesis. In this randomized, double-blind,
placebo-controlled trial the efficacy and long-term safety of ManNAc will be evaluated
in
subjects with GNE myopathy.
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Staccato Alprazolam for Seizure Rescue
Lead Researcher: Thomas Wychowski
Epilepsy is a brain disorder characterized by recurrent seizures. It can occur as
a result of a brain injury, a structural brain lesion, as a part of many medical diseases,
or it may be something someone is born with. When seizures are not well controlled,
patients might experience seizure emergencies and have a need for acute treatment
in addition to their regular medicines. After patient's seizure types are evaluated
and approved, they will be trained on using the Staccato alprazolam device at home
for prolonged seizures. Study involves enrolling a study partner (family member, caregiver)
to participate in the study with the patient. Patients aged 12 and older with focal
or generalized epilepsy who have long seizures are eligible to participate.
View Study Details
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