Treatment Strategies for Children With Smith-Magenis Syndrome
Status:
Completed
Trial end date:
2018-05-22
Target enrollment:
Participant gender:
Summary
This study will examine the effect of bright light or melatonin treatment on sleep in
children with Smith-Magenis syndrome (SMS), a genetic disorder characterized by certain
physical, behavioral and developmental features. Patients have a disrupted sleep cycle
involving early waking, frequent daytime napping and frequent nighttime awakenings. Melatonin
is a hormone normally produced at night in healthy people. People with SMS produce high
levels of melatonin during the daytime and very low levels at night. This may affect their
behavior, mood, attention span and sleep patterns.
Healthy volunteers between 18 and 45 years of age and children with SMS who are between 3 and
16 years of age may be eligible for this study.
Healthy subjects are admitted to the NIH Clinical Center overnight. In the morning they take
one dose of time-release melatonin and have blood and saliva samples collected hourly from
7:00 AM to 6:00 PM.
Children with SMS participate in a 2-part study, as follows:
Part 1 Inpatient Trial
Pre-trial at-home phase: During the month before NIH inpatient admission, participants do the
following:
- Wear an actiwatch device or keep a daily sleep diary to monitor daytime alertness, mood
shifts and sleep patterns.
- Complete a behavior assessment survey related to the child s behaviors and sleep
patterns.
- Obtain frequent body temperature measurements.
- Collect several saliva samples over a 24-hour period.
NIH admission phase:
- Children are admitted to the NIH Clinical Center for 2-3 nights for bright light
treatment. They remain in their rooms for alternating periods of exposure to standard
dim room light and bright light, using a light box placed within 3 to 5 feet of the
child. An electroencephalogram (EEG) with additional electrodes to track eye movements
is used to monitor the child s attention. Between 8AM and 6PM serial blood samples are
collected to measure melatonin levels. A parent rates the child s mood and behavior
during the 2-day test period.
- Children are admitted to the NIH Clinical Center for 2-3 nights for melatonin treatment.
They take a single dose of melatonin or placebo tablet at bedtime. During the daytime,
EEG electrodes are placed to track eye movements. Between 7 PM and 7 AM serial blood
samples are collected to measure melatonin levels. A parent rates the child s behavior
and mood as described for the bright light study.
- Children may receive either or both of the bright light and melatonin treatments.
Part 2 Outpatient Trial
Children participate in a combined bright light with melatonin trial at home. They undergo
the same procedures outlined in the pre-trial at-home phase of Part 1 (actiwatch, behavior
assessments, body temperature measurements, saliva samples) over an 11-week period. If saliva
samples cannot be collected for melatonin testing, 24-hour urine samples may be collected
instead.