Glycogen Storage Disease Type INCT07739394
Diazoxide in the Treatment of Type 1 Glycogenosis
An observational study of Glycogen Storage Disease Type I, run by Central Hospital, Nancy, France.
Trial summary
In children with a glycogen storage disorder, one of the enzymes needed to convert glucose into glycogen, or to break down glycogen into glucose, is missing. There are many different types of glycogen storage disorders (also known as glycogenoses). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Glycogen Storage Disease Type I
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 30 Aug 2026to 15 Oct 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients with type I glycogen storage disease
Who may be able to join
- Patients followed at the CHRU de Nancy or CHU de Besançon for type I glycogen storage disease
- Person having received full information on the organization of the research and not having objected to the use of this data
- Parental consent for minors
Who cannot join
- Lack of data in medical records to meet the study's main objective
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
In children with a glycogen storage disorder, one of the enzymes needed to convert glucose into glycogen, or to break down glycogen into glucose, is missing. There are many different types of glycogen storage disorders (also known as glycogenoses).
Type 1 glycogenosis results in low blood sugar (hypoglycemia), increased lactate (a glucose metabolite produced by body tissues when oxygen supply is insufficient) and a bulky abdomen (glycogen accumulation induces liver enlargement). Low blood sugar leads to sweating, confusion, convulsions and coma. Type 1 glycogenosis manifests itself early in life.
Read more
In children, glycogen storage disorders can have other consequences, such as stunted growth linked to chronic acidosis, tend to increase uric acid levels (a breakdown product) which accumulate in the joints, leading to gout, and in the kidneys, leading to kidney stones.
The mainstay of treatment is frequent oral feeding with raw cornstarch or a lactose-free preparation with maltodextrin to maintain normal blood sugar levels. Nocturnal enteral feeding via gastrostomy is necessary during the first years of life.
These children tend to have greater insulin reactions (= hormone that brings sugar into the cells), resulting in a more rapid fall in blood sugar levels.
Diazoxide is a drug that inhibits pancreatic insulin secretion and prevents blood sugar levels from falling. It has been used successfully in some patients.
The main objective of this project is to describe the metabolic balance in children with type 1 glycogenosis treated with Diazoxide compared with children who did not receive Diazoxide treatment.
- Conditions
- Glycogen Storage Disease Type I
- Medical subject headings
- Glycogen Storage Disease Type I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| treatment with diazoxide | Not described | |
| No treatment with diazoxide | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
evolution of glycemia and lactic acid
Measured: From diagnosis to 10 years of follow-up (based on available data)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study start30 Aug 2026 (estimated)
- Primary completion30 Sep 2027 (estimated)
- Study completion15 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage Disease Type IStudies of the same condition, open and under way.
- More trials run by Central Hospital, Nancy, FranceThe 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
- Other
- Time perspective
- Retrospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Central Hospital, Nancy, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07739394
- Sponsor's protocol ID
- 2025PI065
- Status last verified
- Jul 2026
- First submitted
- 28 Jul 2026
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Gumus E, Ozen H. Glycogen storage diseases: An update. World J Gastroenterol. 2023 Jul 7;29(25):3932-3963. doi: 10.3748/wjg.v29.i25.3932.
PubMed 37476587
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Diazoxide in the Treatment of Type 1 Glycogenosis
- ClinicalTrials.gov ID
- NCT07739394
- Acronym / sponsor ID
- 2025PI065
- Conditions
- Glycogen Storage Disease Type I
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Central Hospital, Nancy, FranceUniversity, hospital or other
- Study start
- 30 Aug 2026 (estimated)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 15 Oct 2027 (estimated)
- First posted
- 31 Jul 2026 (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.
In children with a glycogen storage disorder, one of the enzymes needed to convert glucose into glycogen, or to break down glycogen into glucose, is missing. There are many different types of glycogen storage disorders (also known as glycogenoses).
Type 1 glycogenosis results in low blood sugar (hypoglycemia), increased lactate (a glucose metabolite produced by body tissues when oxygen supply is insufficient) and a bulky abdomen (glycogen accumulation induces liver enlargement). Low blood sugar leads to sweating, confusion, convulsions and coma. Type 1 glycogenosis manifests itself early in life.
In children, glycogen storage disorders can have other consequences, such as stunted growth linked to chronic acidosis, tend to increase uric acid levels (a breakdown product) which accumulate in the joints, leading to gout, and in the kidneys, leading to kidney stones.
The mainstay of treatment is frequent oral feeding with raw cornstarch or a lactose-free preparation with maltodextrin to maintain normal blood sugar levels. Nocturnal enteral feeding via gastrostomy is necessary during the first years of life.
These children tend to have greater insulin reactions (= hormone that brings sugar into the cells), resulting in a more rapid fall in blood sugar levels.
Diazoxide is a drug that inhibits pancreatic insulin secretion and prevents blood sugar levels from falling. It has been used successfully in some patients.
The main objective of this project is to describe the metabolic balance in children with type 1 glycogenosis treated with Diazoxide compared with children who did not receive Diazoxide treatment.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| treatment with diazoxide | – | – |
| No treatment with diazoxide | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with type I glycogen storage disease
Key inclusion criteria (3 listed)
- Patients followed at the CHRU de Nancy or CHU de Besançon for type I glycogen storage disease
- Person having received full information on the organization of the research and not having objected to the use of this data
- Parental consent for minors
Key exclusion criteria (1 listed)
- Lack of data in medical records to meet the study's main objective
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
evolution of glycemia and lactic acid
Time frame: From diagnosis to 10 years of follow-up (based on available data)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Results publication: PubMed 37476587Gumus E, Ozen H. Glycogen storage diseases: An update. World J Gastroenterol. 2023 Jul 7;29(25):3932-3963. doi: 10.3748/wjg.v29.i25.3932.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type I, run by Central Hospital, Nancy, France.
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 3 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: opening soon, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact François Feillet, Professor (0383154796, f.feillet@chru-nancy.Fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
evolution of glycemia and lactic acid (time frame: From diagnosis to 10 years of follow-up (based on available data)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07739394, which also lists 1 linked publication.
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 31 Jul 2026.