Randomized Controlled Study of Donepezil in Fragile X Syndrome

Completed · Phase 2

Conditions studied: Fragile X Syndrome

In brief

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.

Key facts

Study ID

NCT01120626

Run by

Stanford University

People needed

45

Starts

2009-09-01

Expected to finish

2013-12-01

Last updated by the study team

2016-03-17

Who can join

Age: 12 and older, up to 29. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• Current or lifetime DSM-IV diagnosis of bipolar disorder, schizophrenia, schizoaffective disorder, or psychotic disorder, NOS based upon reported history
• 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
• Concomitant or anticipated use of other medications having prominent effects on the cholinergic system (e.g., bethanechol, benztropine, atropine, succinylcholine)
• 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)
• Medical illnesses where donepezil could worsen the condition such as asthma, cardiac conduction abnormalities, urinary obstruction or gastrointestinal disease with gastric bleeding
• Pregnancy or sexually active females not using a reliable method of contraception
• 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.)

Where it is running

• Stanford University School of Medicine — Stanford, California, United States

Full record on ClinicalTrials.gov

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