Ongoing Clinical Trials for Glycogen Storage Disorder
Currently, there is 1 ongoing clinical trial for Glycogen Storage Disorder, specifically focused on Glycogen Storage Disease Type Ib. This trial is investigating the use of empagliflozin, a medication typically used for diabetes, to treat neutropenia (low white blood cell counts) in patients with this rare inherited condition. The trial is being conducted in Poland and aims to evaluate the safety and effectiveness of this treatment approach.
Clinical trial locations
Study on the Use of Empagliflozin for Treating Neutropenia in Patients with Glycogen Storage Disease Type Ib
This clinical trial is investigating a new treatment approach for Glycogen Storage Disease Type Ib, a very rare inherited condition that affects how the body stores and uses sugar. People with this condition often develop neutropenia, which means they have fewer neutrophils—a type of white blood cell that helps fight infections. This makes them more vulnerable to getting sick.
Investigational drug: The trial is testing empagliflozin, which is sold under the brand name Jardiance. This medication is typically used to treat type 2 diabetes by helping control blood sugar levels. In this study, researchers want to see if it can help increase neutrophil counts in patients with Glycogen Storage Disease Type Ib. Empagliflozin is taken orally as a 10 mg film-coated tablet.
Main inclusion criteria: The trial is open to both males and females who are at least 4 weeks old. Participants must have a confirmed diagnosis of Glycogenosis type Ib and must be experiencing neutropenia or have abnormal activity in certain blood cells called neutrophils. For children under 13 years old, a legal representative must provide consent, while children 13 years and older need to provide consent along with their legal representative. Adults must provide their own voluntary informed consent.
Main exclusion criteria: Patients cannot participate if they do not have a confirmed diagnosis of Glycogenosis type Ib or are not experiencing neutropenia. Pregnant or breastfeeding women are not eligible. People who are already participating in another clinical trial, have a history of allergic reactions to empagliflozin, have other medical conditions that might interfere with the study, or are unwilling or unable to follow the study procedures are also excluded.
Focus and goals: The primary goal of this trial is to evaluate how safe empagliflozin is for patients with Glycogen Storage Disease Type Ib and how well they tolerate the medication when used to treat neutropenia. The secondary goal is to assess whether the medication can effectively restore the number and function of neutrophils. The study involves regular monitoring visits to check for any side effects and to track changes in blood cell counts. The trial is expected to conclude by March 26, 2025.
Throughout the study, participants will undergo an initial assessment to confirm their eligibility, followed by regular follow-up visits. During these visits, safety data will be collected to monitor any adverse reactions. The study team will carefully evaluate both the safety of the treatment and its effectiveness in improving the immune function of patients with this rare condition.
Summary
Currently, there is only one ongoing clinical trial for Glycogen Storage Disorder, specifically targeting Type Ib patients with neutropenia. This trial represents an innovative approach by repurposing empagliflozin, a diabetes medication, for a rare genetic condition affecting immune function. The trial is being conducted in Poland and is open to participants as young as 4 weeks old, making it accessible to both children and adults affected by this condition. The focus on safety and tolerability reflects the cautious approach necessary when testing medications for rare diseases, particularly in pediatric populations. The expected completion date of March 2025 means results may become available relatively soon, potentially offering new treatment options for patients who currently have limited therapeutic choices.