Augmentation of the Cholinergic System in Fragile X Syndrome: A Double-Blind Placebo-Controlled Randomized Study of Donepezil

Fragile X syndrome (FraX) is the most common known heritable cause of human intellectual disability. Though recent research has revealed much about the genetic and neurobiological bases of FraX, knowledge about specific and effective treatments for affected individuals is lacking. Based on information from both human and animal studies, one cause of intellectual disability in FraX may be related to deficits in a particular brain neurotransmitter system (the "cholinergic" system). Thus, the investigators propose to use a specific medication, donepezil, to augment cholinergic system in adolescents affected by FraX. If found to be effective, the knowledge generated by this research may also be relevant to other developmental disorders that share common disease pathways with FraX.

Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.

Investigator(s):

Intervention(s):

  • drug : donepezil hydrochloride
  • drug : sugar pill

Phase: Phase 2

Eligibility

Ages Eligible For Study:

12 Years - 29 Years

Inclusion Criteria

1. confirmed genetic diagnosis of fragile X syndrome 2. age >=12, <=29 3. Verbal IQ >= 50, <=75 4. Tanner pubertal stage >= 3

External Links

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Contact information

Primary Contact:

Mai K Manchanda, AB 650-704-9763

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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