Open-Label Trial of Sulforaphane in Premutation Carriers With FXTAS
Completed · Not applicable
Conditions studied: Fragile X Associated Tremor/Ataxia Syndrome (Fxtas) (Diagnosis)
In brief
FXTAS is a rare genetic progressive neurodegenerative disorder, linked to a trinucleotide repeat expansion in the FMR1 gene. FXTAS is characterized by tremor and ataxia in addition to atrophy and white matter disease in the central nervous system (CNS). In addition to the major clinical observations of intention tremor and gait dysfunction, minor symptoms of parkinsonism, neuropathy, and cognitive decline also significantly impact individuals with FXTAS. The dietary supplement being tested in this study is called Sulforaphane. It is found in broccoli and similar cruciferous vegetables and may cause some gas and discomfort. This is not a study looking at clinical efficacy but instead a study of molecular outcome measures. Investigators want to get more information about how Sulforaphane affects specific biomolecular markers captured in blood. In this study, participants will be taking an increasing amount of the Sulphoraphane supplement pills (238mg/tablet), starting at 1 and increasing to 6, every morning at breakfast for 6 months. In addition, there will be a total of 3 visits (Initial, 3-month and 6-month) to the MIND Institute where participants will be evaluated. At each visit (3 total) participants will undergo a battery of medical and neurologic exams which make take 2-3 days to complete each time. Participants and/or their caregivers will also be asked to fill out questionnaires/surveys. At the initial visit and at 6 months, we will collect blood for analysis. Two MRI scans will be done, also at the initial visit and at 6 months.
Key facts
- Study ID
- NCT05233579
- Run by
- University of California, Davis
- People needed
- 15
- Starts
- 2021-06-25
- Expected to finish
- 2023-01-01
- Last updated by the study team
- 2023-07-12
Who can join
Age: 50 and older, up to 85. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of FXTAS
- Presence of a FMR1 premutation (55 to 200 CGG repeats)
You may not qualify if…
- Individuals with severe renal failure (GFR is <60 ml/min/1.73 m\^2)
- Significant and current reported substance abuse
- Individuals with substance use disorder (meets 6 or more symptoms of substance use disorder criteria)
- Any subject on hospice or on home oxygen
- Individuals who are pregnant
Where it is running
- UC Davis MIND Institute — Sacramento, California, United States
Full record on ClinicalTrials.gov
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