Melatonin Clinical Trial in Fragile X

With a $60,000 grant from FRAXA Research Foundation from 1998-1999, Dr. Randi Hagerman and her team at the University of California studied the effects of different compounds on individuals with fragile X syndrome, focusing specifically on melatonin. Results published.

$60,000 Grant
Randi Hagerman, MD
Principal Investigator

Karen Riley, PhD
Postdoctoral Fellow

University of California at Davis
1998-9 FRAXA Research Grant
$60,000 over 2 Years

Project Plan

by Karen Riley, 7/1/1998

There are few controlled studies of treatment of children and adults with fragile X syndrome. The FRAXA postdoctoral fellowship offers a unique opportunity to improve this situation. Dr. Karen Riley will act as a facilitator for collaborative research efforts with Drs. Loesch and Martin in Australia and with other centers in the US to improve and enhance treatment options for individuals and families affected by fragile X syndrome.

This project will focus on the primary and secondary benefits of pharmacological interventions. We will study the melatonin profile of children with fragile X syndrome and will carry out a controlled study of the efficacy of melatonin for sleep disturbances. Secondary benefits of improved sleep will also be explored and identified. These benefits could include decreases in outburst behavior, increased time on task, and improved social interactions. Sensory issues are also of paramount concern for individuals and families affected by fragile X. Dr. Riley will evaluate the impact of stimulants, clonidine, and serotonin agents on the electrodermal (sweat) responses to visual, auditory, tactile, olfactory, and vestibular stimuli in collaboration with Dr. Lucy Miller, O.T.R.

The study will also involve the development and organization of neuropsychological protocols to be utilized for controlled studies of a variety of psychopharmacological interventions with children and adults with fragile X. This team will strive to organize the participation of multiple centers, internationally and in the US, in the controlled trials of psychopharmacological interventions and in the exploration of secondary benefits. Drs. Riley and Hagerman report that they are eager to embark on this journey and are optimistic about the future findings.

Dr. Hagerman and colleagues have published results of their studies of melatonin:

J Clin Sleep Med. 2009 Apr 15;5(2):145-50. The efficacy of melatonin for sleep problems in children with autism, fragile X syndrome, or autism and fragile X syndrome

and Am J Med Genet. 2000 Dec 11;95(4):307-15. Melatonin profiles and sleep characteristics in boys with fragile X syndrome: a preliminary study