Glycogen Storage Disease Type IANCT02054832
Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch
An observational study of Glycogen Storage Disease Type IA and Glycogen Storage Disease Type IB, run by John Mitchell at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 50 years
- Conditions the study is for
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB, Glycogen Storage Disease Type III
- What is being tested?
- Glycosade
- How many people
- 11 participants
- Where
- 1 siteCanada
- Study dates
- Nov 2013to Jul 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients with Glycogen Storage Disease taking uncooked cornstarch to prevent hypoglycemia followed at the Montreal Children's Hospital and St-Luc Hospital.
Who may be able to join
- 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
Who cannot join
- Continuous overnight feeds
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB, Glycogen Storage Disease Type III, Glycogen Storage Disease Type 0
- Being tested
- Glycosade
- Medical subject headings
- Hepatorenal form of glycogen storage disease, Glycogen Storage Disease IB, Glycogen Storage Disease Type III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlycosadeDietary supplement
| Study group | Role | What they receive |
|---|---|---|
| Glycosade | A prospective cohort design will be used to assess the impact on sleep and continue to monitor safety of Glycosade. Receives: Dietary Supplement: Glycosade |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
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.
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2014 (estimated)
- Study startNov 2013
- Primary completionJul 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IAStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- John Mitchell
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, John Mitchell, McGill University Health Centre/Research Institute of the McGill University Health Centre
- Investigators
- John J Mitchell, MD, Principal investigator, Montreal Children's Hospital of the MUHC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02054832
- Sponsor's protocol ID
- 12-337-PED
- Status last verified
- Sep 2015
- First submitted
- 31 Jan 2014
- Last updated
- 25 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Comparison of Quality of Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard and Modified Uncooked Cornstarch
- ClinicalTrials.gov ID
- NCT02054832
- Acronym / sponsor ID
- 12-337-PED
- Conditions
- Glycogen Storage Disease Type IA, Glycogen Storage Disease Type IB, Glycogen Storage Disease Type III, Glycogen Storage Disease Type 0
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- John MitchellUniversity, hospital or other
- Study start
- Nov 2013
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 4 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Glycosade | – | Dietary Supplement: GlycosadeA prospective cohort design will be used to assess the impact on sleep and continue to monitor safety of Glycosade. |
Who may qualify
- Ages
- 2 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with Glycogen Storage Disease taking uncooked cornstarch to prevent hypoglycemia followed at the Montreal Children's Hospital and St-Luc Hospital.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (1 listed)
- Continuous overnight feeds
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Glycosade | Dietary supplement | Glycosade |
Primary outcome
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.
Time frame: 2 weeks
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.
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John J Mitchell, MD principal investigator
Montreal Children's Hospital of the MUHC
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type IA and Glycogen Storage Disease Type IB, run by John Mitchell at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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. (time frame: 2 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2015.