Clinical trial NCT02054832: Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch
Official title: A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Glycogen Storage Disease Type IA
Glycogen Storage Disease Type IB
Glycogen Storage Disease Type III
Glycogen Storage Disease Type 0
Lead sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
Sponsor class: OTHER
First posted: 2014-02-04
Enrollment: 11
Sex: ALL
Minimum age (years): 2
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Glycogen Storage Disease taking uncooked cornstarch to prevent hypoglycemia followed at the Montreal Children's Hospital and St-Luc Hospital.

Brief summary: The aim of the present study is to determine if there is a change in quality and quantity of sleep perceived by adults and children with GSD and their parents while starting a modified UCCS (Glycosade) to prevent nocturnal hypoglycemia. The investigators also aim to evaluate if there is a change in quality of life perceived by adults and children and their parents with Glycosade.

Detailed description: This is a prospective cohort study. Patients above 2 years old and their parents (for children only) will be enrolled during their usual follow-up. Parents will be asked to complete a quality of sleep questionnaire (as it pertains to both child and parent) relating to the past month on their current dietary regimen (standard UCCS) and a quality of life questionnaire (as it pertains to the child only). Parents will then complete a sleep diary (for both child and parent) and both child and parent will wear an actigraph that will record movements during sleep over a 1 week period. Adult GSD patients will complete their own questionnaires. Following this first assessment, they will be hospitalised over a 24 hour period as part of standard of care to start the modified UCCS, Glycosade, under supervision and with a continuous glucose monitoring (CGM) sensor. Following hospitalization, the family will return home. Glucose will be monitored with the aid of the CGM sensor for 5 to 7 days. The actigraphy and the sleep diary will be repeated after 2 weeks (for 1 week) while on Glycosade. One month after starting the modified UCCS, questionnaires on quality of sleep and quality of life will be repeated.

Eligibility criteria: Inclusion Criteria:

* Patients of ≥ 2 years old with a diagnostic of GSD 0, I, III, VI, IX or XI based on a liver biopsy, mutation in the appropriate gene or clinical evidence of GSD with a positive familial history
* Medical history of fasting hypoglycemia
* Currently taking standard UCCS
* With a stable condition
* Followed in GSD clinics at the Montreal Children's Hospital and the Hôpital St-Luc
* With informed consent obtained

Exclusion Criteria:

* Continuous overnight feeds

Primary outcomes:
measure: The aim of the present study is to determine if there is a change in quality and quantity of sleep perceived by GSD adults and children and their parents while starting a modified UCCS (Glycosade) to prevent nocturnal hypoglycaemia.
timeFrame: 2 weeks
Secondary outcomes:
measure: To evaluate if there is a change in quality of life perceived by GSD adults and children and their parents with Glycosade.
timeFrame: 1 month
Other outcomes:
measure: To describe the variability in glucose fluctuations with Glycosade using a CGM sensor.
timeFrame: 1 week
measure: To establish if metabolic control is maintained using Glycosade.
timeFrame: 24 hours
measure: To evaluate the safety profile of Glycosade, based on the frequency of side effects, e.g. gastrointestinal side effects while receiving Glycosade.
timeFrame: 1 month
measure: To assess the acceptability/palatability of Glycosade.
timeFrame: 1 month
Locations:
city: Montreal
country: Canada
facility: Montreal Children's hospital
state: Quebec
Study officials:
affiliation: Montreal Children's Hospital of the MUHC
name: John J Mitchell, MD
role: PRINCIPAL_INVESTIGATOR
References: