©2022 Stanford Medicine
Randomized Controlled Study of Donepezil in Fragile X Syndrome
Not Recruiting
Trial ID: NCT01120626
Purpose
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.
Official Title
Augmentation of the Cholinergic System in Fragile X Syndrome: A Double-Blind Placebo-Controlled Randomized Study of Donepezil
Stanford Investigator(s)
Antonio Hardan, M.D.
Professor of Psychiatry and Behavioral Sciences
Eligibility
Inclusion Criteria:
1. confirmed genetic diagnosis of fragile X syndrome
2. age >=12, <=29
3. Verbal IQ >= 50, <=75
4. Tanner pubertal stage >= 3
Exclusion Criteria:
1. Current or lifetime DSM-IV diagnosis of bipolar disorder, schizophrenia,
schizoaffective disorder, or psychotic disorder, NOS based upon reported history
2. Poorly controlled seizure disorder or taking more than one anticonvulsant (subjects
cannot be prescribed carbamazepine, phenytoin, or phenobarbital due to potential
interaction effects with donepezil). The investigators will permit one anticonvulsant
as monotherapy for seizures if the seizure disorder is well controlled with no
evidence of break through seizures within the past year
3. Concomitant or anticipated use of other medications having prominent effects on the
cholinergic system (e.g., bethanechol, benztropine, atropine, succinylcholine)
4. Medications or nutritional supplements that have the potential to significantly alter
donepezil levels, clinical effects or adverse reactions (antifungal agents,
corticosteroids, erythromycin, beta-blockers, calcium channel blockers, NSAIDs, gingko
biloba, St. John's wort)
5. Medical illnesses where donepezil could worsen the condition such as asthma, cardiac
conduction abnormalities, urinary obstruction or gastrointestinal disease with gastric
bleeding
6. Pregnancy or sexually active females not using a reliable method of contraception
7. If considering participation in brain MRI part of the study, then any
contraindications for MRI (e.g., orthodontia, metal in or on the body, etc.)
Intervention(s):
drug: sugar pill
drug: donepezil
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Mai K Manchanda, AB
650-704-9763