Trial record 2 of 4 for:    fxtas

Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02603926
Recruitment Status : Enrolling by invitation
First Posted : November 13, 2015
Last Update Posted : June 8, 2017
Information provided by (Responsible Party):
Randi J. Hagerman, MD, University of California, Davis

Brief Summary:
The purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).

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)
Primary Purpose: Treatment
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

Arm Intervention/treatment
Experimental: Allopregnanolone
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.
Drug: Allopregnanolone

Primary Outcome Measures :
  1. 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).

Secondary Outcome Measures :
  1. 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.

  2. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

  • Fragile X premutation carrier status (55 to 200 CGG repeats in FMR1),
  • Diagnosis of FXTAS including an intention tremor and/or ataxia and/or deficits on the BDS-2 demonstrating executive function deficits.

Exclusion Criteria

  • other genetic problems in addition to the premutation
  • a history of significant brain trauma
  • significant substance abuse
  • inability to follow the protocol
  • liver or kidney disease
  • heart failure
  • active cancer
  • other serious systemic disease
  • current use of phenytoin

Information from the National Library of Medicine

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 identifier (NCT number): NCT02603926

United States, California
UC Davis MIND Institute
Sacramento, California, United States, 95817
Sponsors and Collaborators
Randi J. Hagerman, MD
Principal Investigator: Randi J Hagerman, MD UC Davis MIND Institute

Responsible Party: Randi J. Hagerman, MD, Medical Director, MIND Institute, University of California, Davis Identifier: NCT02603926     History of Changes
Other Study ID Numbers: 720668
First Posted: November 13, 2015    Key Record Dates
Last Update Posted: June 8, 2017
Last Verified: June 2017

Studies a U.S. FDA-regulated Drug Product: Yes

Additional relevant MeSH terms:
Cerebellar Ataxia
Fragile X Syndrome
Pathologic Processes
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Central Nervous System Depressants
Physiological Effects of Drugs