Glycogen Storage Disease Type IIINCT05196165
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
An observational study of Glycogen Storage Disease Type III, run by Ultragenyx Pharmaceutical Inc at 4 sites in 2 countries.
Trial summary
The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Glycogen Storage Disease Type III
- How many people
- 14 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 20 May 2022to 2 Mar 2023
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study aims to enroll at least 5 participants from each of the following age groups: 1-5 years, 6-12 years, 12-17 years, and ≥ 18 years.
Who may be able to join
- Diagnosis of GSD III, confirmed by amylo-alpha-1,6-glucosidase 4-alpha-glucanotransferase (AGL) sequencing or glycogen debranching enzyme (GDE) enzymatic testing
Who cannot join
- Participant is unwilling to remain blinded to continuous glucose monitor (CGM) data for the first 13 weeks of the study, or the Investigator determines that blinding would compromise subject safety
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation or pose undue risk
- Use of any IP or investigational medical device within 30 days or 5.5 half-lives, whichever is longer, prior to screening, or during the study
- Note: Other criteria may apply per protocol
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States3 sites
Baylor College of Medicine
Houston, Texas, 77030
University of Texas
Houston, Texas, 77030
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Netherlands1 site
University of Groningen Beatrix Children's Hospital
Groningen, 9700RB
About this study
Official title: Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over With GSD III
The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III).
- Conditions
- Glycogen Storage Disease Type III
- Being tested
- No Intervention
- Medical subject headings
- Glycogen Storage Disease Type III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
No investigational product (IP) will be administered in this study
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Hypoglycemic Events During the 26-week Observation Period
Measured: Week 26
Secondary outcomes (7)
Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores
Measured: Up to Week 26
Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores
Measured: Up to Week 26
Number of Times the Participant can Rise from a Seated to a Standing Position in a 30-second Period, as Measured by the Site-to-Stand (STS) Test
Measured: Up to Week 26
Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)
Measured: Up to Week 26
Ankle Dorsiflexion, Measured Bilaterally Using Goniometry
Measured: Up to Week 26
Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)
Measured: Up to Week 26
GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score
Measured: Up to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2022
- Study start20 May 2022
- Primary completion2 Mar 2023
- Study completion2 Mar 2023
Why it stopped: Sponsor decision not related to safety concerns
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 IIIStudies of the same condition, open and under way.
- More trials run by Ultragenyx Pharmaceutical IncThe sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Ultragenyx Pharmaceutical Inc
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05196165
- Sponsor's protocol ID
- UX053-CL002
- Status last verified
- Sep 2023
- First submitted
- 10 Dec 2021
- Last updated
- 28 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over With GSD III
- ClinicalTrials.gov ID
- NCT05196165
- Acronym / sponsor ID
- UX053-CL002
- Conditions
- Glycogen Storage Disease Type III
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sponsor decision not related to safety concerns
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Ultragenyx Pharmaceutical IncIndustry (pharma, biotech, devices)
- Study start
- 20 May 2022 (actual)
- Primary completion
- 2 Mar 2023 (actual)
- Study completion
- 2 Mar 2023 (actual)
- First posted
- 19 Jan 2022 (actual)
- 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 primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III).
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study aims to enroll at least 5 participants from each of the following age groups: 1-5 years, 6-12 years, 12-17 years, and ≥ 18 years.
Key inclusion criteria (1 listed)
- Diagnosis of GSD III, confirmed by amylo-alpha-1,6-glucosidase 4-alpha-glucanotransferase (AGL) sequencing or glycogen debranching enzyme (GDE) enzymatic testing
Key exclusion criteria (4 listed)
- Participant is unwilling to remain blinded to continuous glucose monitor (CGM) data for the first 13 weeks of the study, or the Investigator determines that blinding would compromise subject safety
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation or pose undue risk
- Use of any IP or investigational medical device within 30 days or 5.5 half-lives, whichever is longer, prior to screening, or during the study
- Note: Other criteria may apply per protocol
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 |
|---|---|---|
| No InterventionNo investigational product (IP) will be administered in this study | Other | – |
Primary outcome
Number of Hypoglycemic Events During the 26-week Observation Period
Time frame: Week 26
7 secondary outcomes
Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores
Time frame: Up to Week 26
Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores
Time frame: Up to Week 26
Number of Times the Participant can Rise from a Seated to a Standing Position in a 30-second Period, as Measured by the Site-to-Stand (STS) Test
Time frame: Up to Week 26
Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)
Time frame: Up to Week 26
Ankle Dorsiflexion, Measured Bilaterally Using Goniometry
Time frame: Up to Week 26
Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)
Time frame: Up to Week 26
GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score
Time frame: Up to Week 26
Results and safety
The study was stopped early (reason given: Sponsor decision not related to safety concerns). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
- Patient Advocacy at Ultragenyx: Glycogen Storage Disease Type III (GSDIII)Link provided by the sponsor.
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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.
Medical Director study director
Ultragenyx Pharmaceutical Inc
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type III, run by Ultragenyx Pharmaceutical Inc at 4 sites in 2 countries.
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 1 inclusion and 4 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: stopped early, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, Netherlands. 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 was stopped early (reason given: Sponsor decision not related to safety concerns). Results are not available in the source record.
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?
Number of Hypoglycemic Events During the 26-week Observation Period (time frame: Week 26). 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 28 Sep 2023.