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

NCT02054832 | Completed

• 1 other identifier: 12-337-PED
• Study Type: observational
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Glycogen Storage Disease Type IA; Glycogen Storage Disease Type IB; Glycogen Storage Disease Type III; Glycogen Storage Disease Type 0

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• To evaluate if there is a change in quality of life perceived by GSD adults and children and their parents with Glycosade.

  1 month

Other Outcomes (4)

• To describe the variability in glucose fluctuations with Glycosade using a CGM sensor.

  1 week

• To establish if metabolic control is maintained using Glycosade.

  24 hours

• To evaluate the safety profile of Glycosade, based on the frequency of side effects, e.g. gastrointestinal side effects while receiving Glycosade.

  1 month

Study Arms (1)

Glycosade

A prospective cohort design will be used to assess the impact on sleep and continue to monitor safety of Glycosade.

Dietary Supplement: Glycosade

Interventions

Glycosade DIETARY_SUPPLEMENT

Glycosade

Eligibility Criteria

• Age: 2 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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

You may not qualify if:

• Continuous overnight feeds

Sponsors & Collaborators

• John Mitchell: lead

Study Sites (1)

Montreal Children's hospital

Montreal, Quebec, H3H1P3, Canada

Location

MeSH Terms

Conditions

Hepatorenal form of glycogen storage disease; Glycogen Storage Disease IB; Glycogen Storage Disease Type III

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• John J Mitchell, MD

  Montreal Children's Hospital of the MUHC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Division Head, Endocrinology

Study Record Dates

• First Submitted: January 31, 2014
• First Posted: February 4, 2014
• Study Start: November 1, 2013
• Primary Completion: July 1, 2014
• Study Completion: July 1, 2014
• Last Updated: September 25, 2015

Record last verified: 2015-09

Locations

CA (1)