Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I (GSD-DIET)

Insel Gruppe AG, University Hospital Bern

Status: Enrolling

Conditions: Glycogen Storage Disease Type I

Treatments: Dietary Supplement: Fructose and galactose; Dietary Supplement: Fructose

Study type: Interventional

Funder types: Other

Identifiers

NCT06852612

2024-02154

Details and patient eligibility

About

The present project will specifically assess metabolic effects of dietary interventions with controlled intake of fructose and fructose/galactose in GSDI, with the aim to provide evidence whether relaxed dietary restrictions of fructose and galactose may be justified in treatment recommendations at least for adults, which would considerably enlarge food choice in everyday life of the patients with an expected positive impact on the quality of life of patients with this rare disorder.

Full description

To assess relaxed restriction of fructose and fructose/galactose intake on secondary metabolic alterations in GSDI, (i) by looking at the traditional parameters for assessing metabolic control in clinical chemistry (lactate, triglycerides, uric acid), and (ii) by using a broad analytical approach relying on targeted metabolomics/lipidomics.

It is hypothesized that relaxed restrictions on the intake of fructose and/or galactose as part of the diet in everyday life may lead to an increase in blood lactate levels (=primary outcome), triglycerides, and uric acid to a certain degree compared to baseline. However, this increase is expected to remain within a range that is not clinically relevant for adult patients, especially when fructose/galactose intake is not excessive and stays within the usual daily allowances for healthy individuals, as planned in this study.

Enrollment: 20 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Genetically and/or enzymatically confirmed diagnosis of GSDI (GSDIa or GSDIb)
• Male or female ≥ 18y
• Restriction of fructose intake in usual dietary treatment
• Written informed consent

Exclusion criteria

• Non-compliance with routine dietary treatment
• Pregnancy or lactation
• Liver transplant
• Recurrent hospitalisations due to metabolic decompensation within the last 12 months
• Severe chronic kidney disease with glomerular filtration rate (GFR) < 30 ml/min
• For GSDIb: Severe, uncontrolled symptomatic inflammatory bowel disease

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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20 participants in 2 patient groups

Diet with additional fructose intake

Experimental group

Treatment:

Dietary Supplement: Fructose

Diet with additional fructose and galactose intake

Experimental group

Treatment:

Dietary Supplement: Fructose and galactose