Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
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|ClinicalTrials.gov Identifier: NCT02603926|
Recruitment Status : Enrolling by invitation
First Posted : November 13, 2015
Last Update Posted : June 8, 2017
|Condition or disease||Intervention/treatment||Phase|
|Fragile X-associated Tremor/Ataxia Syndrome||Drug: Allopregnanolone||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone|
|Study Start Date :||October 2015|
|Actual Primary Completion Date :||December 2016|
|Estimated Study Completion Date :||December 2017|
Subjects will receive and intravenous infusion of Allopregnanolone at escalating doses of 2mg, 4mg, and 6mg once weekly over a three week period. The highest dose tolerated without sedation will be held stable for the remaining weekly infusions, for a total of 12 infusions.
- California Verbal Learning Test II [ Time Frame: 14 weeks ]California Verbal Learning Test II will be assessed pre- and post-treatment, measuring the total number of words remembered after 5 repeated trials(0-64) and then 20 minutes later (0-16).
- CATSYS tremor measurement scale [ Time Frame: 14 weeks ]The CATSYS system quantifies tremor by having a participant hold a tremor pen which is connected to a computer. Ataxia is quantified by measuring sway of patients standing with eyes open and closed on a force plate connected to a computer. Patients will be assessed pre- and post- treatment.
- Increase in hippocampal volume, as measured by structural MRI [ Time Frame: 14 weeks ]Patients will undergo a structural MRI pre-and post-treatment to measure hippocampal volume.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02603926
|United States, California|
|UC Davis MIND Institute|
|Sacramento, California, United States, 95817|
|Principal Investigator:||Randi J Hagerman, MD||UC Davis MIND Institute|