Diazoxide in the Treatment of Type 1 Glycogenosis

Starting soon

Conditions studied: Glycogen Storage Disease Type I

In brief

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.

Key facts

Study ID

NCT07739394

Run by

Central Hospital, Nancy, France

People needed

15

Starts

2026-08-30

Expected to finish

2027-10-15

Last updated by the study team

2026-07-31

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Lack of data in medical records to meet the study's main objective

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.