Clinical trials
Glycogen Storage Disease Type I clinical trials
Below are recruiting glycogen storage disease type i clinical trials, each written for real people, not researchers. We’re tracking 18 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06843330Recruiting
Lactate meter accuracy for children with GSD type Ia
This trial tests whether a simple finger-stick lactate meter gives accurate results for children and adults with a specific type of glycogen storage disease (GSD Ia). It aims to find a faster, easier way to monitor health during emergency visits or hospital stays.
Hartford, ConnecticutAges birth–60 years - NCT04808505RecruitingPhase 3
Study of enzyme therapy for children with infant-onset Pompe disease
This trial tests how well a new enzyme treatment (cipaglucosidase alfa with miglustat) works and how safe it is in children and babies with infant-onset Pompe disease. It may help by improving muscle function after earlier treatment or, for some babies, starting treatment when no prior enzyme therapy has been given.
Gainesville, FloridaAges Up to 17 years - NCT06735755RecruitingPhase 1/Phase 2
Study of BEAM-301 for glycogen storage disease type Ia
This study tests an experimental medicine called BEAM-301 for people with a specific form of glycogen storage disease type Ia (GSDIa). The goal is to see if it can help reduce dangerous low blood sugar episodes.
Orange, CaliforniaAges 18 years+ - NCT06396546Recruiting
Indian Children with Glycogen Storage Disease Registry
This study is creating a registry (a database) to better understand glycogen storage diseases in Indian children. If your child has a confirmed genetic diagnosis, joining helps doctors learn more about the condition and improve care.
Guindy, ChennaiAges Up to 18 years - NCT03564561Recruiting
Pompe disease study of walking and symptoms over time
This study follows people with Pompe disease to better understand how the disease progresses and how walking function changes. You may help researchers learn what symptoms and test results matter most, without necessarily receiving a new treatment.
Garches, Hauts-de-SeineAges 18–80 - NCT06616545Recruiting
French study on type 3 Glycogen storage disease
This study tracks the health of people with type 3 Glycogen storage disease. It may help doctors understand the disease better and plan better care.
ClamartAges Any age - NCT06636383Recruiting
A follow-up study for people with GSDIa who received DTX401
This study monitors the long-term health of people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401, either in a previous clinical trial or after it was approved. The goal is to gather more information about their condition over time.
Orange, CaliforniaAges 2 years+ - NCT06666413RecruitingPhase 4
Study of Nexviazyme in children with infantile Pompe disease
This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.
ShanghaiAges Up to 17 years - NCT06795152Recruiting
Rare GSD Natural History Study
This study follows people with rare types of glycogen storage disease (GSD) over time to learn more about how the disease progresses. It does not test a new treatment but will collect information to help future research.
Durham, North CarolinaAges birth–90 years - NCT06852612Recruiting
Dietary strategies for glycogen storage disease type I
This trial tests different diet strategies to help adults with GSDI (a metabolic condition also known as von Gierke disease) maintain stable blood sugars and avoid dangerous drops. It may help you learn how to better manage your condition through diet.
BernAges 18 years+ - NCT07136844Recruiting
Walking and arm movement study for nerve and muscle conditions
This study uses motion analysis to understand walking and arm function in adults with certain neurological or metabolic diseases. The goal is to find patterns that help doctors better manage these conditions.
LiègeAges 18 years+ - NCT07303140Recruiting
Liver stiffness in young people with glycogen storage disease type III
This study looks at liver stiffness (a sign of scarring, called fibrosis) in children and young adults with type III glycogen storage disease. Researchers want to see how common liver fibrosis is in this group and how it changes over time, using a non-invasive ultrasound-like test called elastography.
StrasbourgAges 1 month–21 years - NCT00231400Recruiting
Pompe disease registry for people with confirmed diagnosis
This trial is a registry, meaning it collects health information from people with confirmed Pompe disease. It may help researchers better understand the disease and improve future treatments.
Birmingham, AlabamaAges Any age - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT00567073Recruiting
Register pregnant people with Pompe disease for pregnancy data
This study keeps a special record of pregnancies in people with Pompe disease to learn more about pregnancy outcomes and newborn information. It does not test a drug by itself—it collects health information to improve future care.
Phoenix, ArizonaAges Any age - NCT02683512Recruiting
Study of GBE deficiency in GSD IV and APBD
This study follows people with GBE deficiency caused by GSD IV or APBD to better understand how the condition works over time. It may help researchers and clinicians plan future care and treatments.
Durham, North CarolinaAges birth–90 years - NCT07459582Recruiting
Home lactate meter and glucose meter accuracy in GSD
This study tests how well a home lactate meter and a standard blood sugar meter work for people with certain types of Glycogen Storage Disease (GSD Ia, Ib, or XI). If you or your child has one of these types, joining could help improve home monitoring.
Hartford, ConnecticutAges Up to 60 years - NCT07645898Recruiting
Glucose monitoring study of cornstarch in glycogen storage disease
This study uses a glucose monitor (a small sensor you wear) to measure how your blood sugar responds after eating raw cornstarch, a common treatment for glycogen storage disease type I. The goal is to understand how well cornstarch helps keep your blood sugar steady.
Naples, NapoliAges 18 years+
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Common questions
- Are there clinical trials for glycogen storage disease type i?
- Yes. Clin2 currently lists 18 recruiting glycogen storage disease type i studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a glycogen storage disease type i trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a glycogen storage disease type i trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.