Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch

A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch

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.

Study Overview

• Status: Completed
• Conditions: Glycogen Storage Disease Type III; Glycogen Storage Disease Type IA; Glycogen Storage Disease Type IB; Glycogen Storage Disease Type 0
• Intervention / Treatment: Dietary supplement: 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.

• Study Type: Observational
• Enrollment (Actual): 11

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montreal, Quebec, Canada, H3H1P3
      • Montreal Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 46 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Glycosade; A prospective cohort design will be used to assess the impact on sleep and continue to monitor safety of Glycosade.	Dietary supplement: Glycosade

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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). Parents will also 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 prior to Glycosade. The actigraphy and the sleep diary will be repeated after 2 weeks (for 2 weeks) while on Glycosade. The quality of sleep questionnaire will be repeated after 1 month on Glycosade. Adult GSD patients will complete their own questionnaires.	2 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate if there is a change in quality of life perceived by GSD adults and children and their parents with Glycosade.	Parents (as it pertains to their child) and adult patients will be asked to complete quality of life questionnaire prior to Glycosade and 1 month after starting this new diet.	1 month

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
To describe the variability in glucose fluctuations with Glycosade using a CGM sensor.	Glucose will be monitored with the aid of the CGM sensor for 5 to 7 days while starting Glycosade.	1 week
To establish if metabolic control is maintained using Glycosade.	Metabolic control in hospital after starting Glycosade will be defined as the absence of deterioration of biochemical data at the end of fast compared to during the fast.	24 hours
To evaluate the safety profile of Glycosade, based on the frequency of side effects, e.g. gastrointestinal side effects while receiving Glycosade.	Subjects will be constantly monitored for adverse events throughout the study. They will be asked to keep a diary to document side effects while receiving Glycosade. They will also be asked at each visit whether they experienced any known or unknown side effects.	1 month
To assess the acceptability/palatability of Glycosade.	Patient report (or parent report on behalf of child) of palatability at 4 weeks after starting Glycosade compared to their previous regimen.	1 month

Collaborators and Investigators

• Sponsor: John Mitchell
• Investigators: Principal Investigator: John J Mitchell, MD, Montreal Children's Hospital of the MUHC

Study record dates

Study Major Dates

• Study Start: November 1, 2013
• Primary Completion (Actual): July 1, 2014
• Study Completion (Actual): July 1, 2014

Study Registration Dates

• First Submitted: January 31, 2014
• First Submitted That Met QC Criteria: February 3, 2014
• First Posted (Estimate): February 4, 2014

Study Record Updates

• Last Update Posted (Estimate): September 25, 2015
• Last Update Submitted That Met QC Criteria: September 23, 2015
• Last Verified: September 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Disease; Metabolic Diseases; Glycogen Storage Disease; Glycogen Storage Disease Type III
• Other Study ID Numbers: 12-337-PED