Trial record 1 of 70 for:    "glycogen storage disease type IX" OR "Glycogen Storage Disease"
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Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by McGill University Health Center
Sponsor:
Information provided by (Responsible Party):
John Mitchell, McGill University Health Center
ClinicalTrials.gov Identifier:
NCT02054832
First received: January 31, 2014
Last updated: February 3, 2014
Last verified: February 2014
  Purpose

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.


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

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch

Resource links provided by NLM:


Further study details as provided by McGill University Health Center:

Primary Outcome Measures:
  • The aim of the present study is to determine if there is a change in quality of sleep perceived by GSD adults and children and their parents while starting a modified UCCS (Glycosade) to prevent nocturnal hypoglycaemia. [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
    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.

  • To determine if there is a change in quantity of sleep perceived by GSD adults and children and their parents while starting a modified UCCS (Glycosade) to prevent nocturnal hypoglycaemia. [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • To evaluate if there is a change in quality of life perceived by GSD adults and children and their parents with Glycosade. [ Time Frame: 1 month ] [ Designated as safety issue: No ]
    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.


Other Outcome Measures:
  • To describe the variability in glucose fluctuations with Glycosade using a CGM sensor. [ Time Frame: 1 week ] [ Designated as safety issue: No ]
    Glucose will be monitored with the aid of the CGM sensor for 5 to 7 days while starting Glycosade.

  • To establish if metabolic control is maintained using Glycosade. [ Time Frame: 24 hours ] [ Designated as safety issue: Yes ]
    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.

  • To evaluate the safety profile of Glycosade, based on the frequency of side effects, e.g. gastrointestinal side effects while receiving Glycosade. [ Time Frame: 1 month ] [ Designated as safety issue: Yes ]
    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.

  • To assess the acceptability/palatability of Glycosade. [ Time Frame: 1 month ] [ Designated as safety issue: No ]
    Patient report (or parent report on behalf of child) of palatability at 4 weeks after starting Glycosade compared to their previous regimen.


Estimated Enrollment: 20
Study Start Date: November 2013
Estimated Study Completion Date: July 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts
Glycosade
A prospective cohort design will be used to assess the impact on sleep and continue to monitor safety of 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

Ages Eligible for Study:   2 Years to 50 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02054832

Contacts
Contact: John J Mitchell, MD 514-412-4400 ext 22438 john.mitchell@muhc.mcgill.ca
Contact: Isabelle Rousseau-Nepton, M.D 514-412-4400 ext 22482 isabelle.rousseau-nepton@mail.mcgill.ca

Locations
Canada, Quebec
Montreal Children's hospital Recruiting
Montreal, Quebec, Canada, H3H1P3
Contact: John Mitchell, MD    514-412-4400 ext 22438    john.mitchell@muhc.mcgill.ca   
Contact: Isabelle Rousseau-Nepton, MD    514-412-4400 ext 22482    isabelle.rousseau-nepton@mail.mcgill.ca   
Principal Investigator: John Mitchell, MD         
Sponsors and Collaborators
John Mitchell
Investigators
Principal Investigator: John J Mitchell, MD Montreal Children's Hospital of the MUHC
  More Information

No publications provided

Responsible Party: John Mitchell, Division Head, Endocrinology, McGill University Health Center
ClinicalTrials.gov Identifier: NCT02054832     History of Changes
Other Study ID Numbers: 12-337-PED
Study First Received: January 31, 2014
Last Updated: February 3, 2014
Health Authority: Canada: Health Canada

Additional relevant MeSH terms:
Glycogen Storage Disease
Glycogen Storage Disease Type III
Glycogen Storage Disease Type I
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases

ClinicalTrials.gov processed this record on September 14, 2014