Filter Results
Conditions
Locations
Treatment Type
Trial Phase
Trial Status
Paid Participation
Clear All
18 Glycogen Storage Disease Trials
Power is an online platform that helps thousands of Glycogen Storage Disease patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.
ATB200/AT2221 for Pompe Disease
Columbus, OhioThis is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:18+
Sex:All
110 Participants Needed
Avalglucosidase Alfa for Pompe Disease
Cincinnati, OhioThis is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD.
Study details include:
* Study duration: Screening - up to 4 weeks;
* Primary Analysis Period (PAP) - 52 weeks;
* Extended Treatment Period (ETP) - 52 weeks;
* Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years.
* Treatment duration: Up to 4 years
* Visit frequency: every other week and potentially every week
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:0 - 12
Sex:All
Key Eligibility Criteria
Disqualifiers:Respiratory Insufficiency, Congenital Abnormality, Organic Disease, Others
17 Participants Needed
Cipaglucosidase Alfa + Miglustat for Pompe Disease
Cincinnati, OhioThis is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
No Placebo Group
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:< 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Invasive Ventilation, CRIM Negative, Others
Must Be Taking:ERT
36 Participants Needed
Cipaglucosidase Alfa + Miglustat for Pompe Disease
Cincinnati, OhioThis is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
No Placebo Group
Prior Safety Data
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:0 - 17
Sex:All
Key Eligibility Criteria
Disqualifiers:Pregnancy, Hypertrophic Cardiomyopathy, Others
Must Be Taking:ERT
22 Participants Needed
Antibody Levels and Biomarkers for Pompe Disease
Cincinnati, OhioPompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme breaks down a type of sugar called glycogen. Without this enzyme, there is a build-up of glycogen in the cells of the body. This causes muscle weakness and other symptoms. Pompe disease can happen at any age, but in late-onset Pompe disease, symptoms generally start from 12 months old onwards.
The standard treatment for people with Pompe disease is to receive regular infusions of the GAA enzyme. This is known as enzyme replacement therapy. However, people can build up antibodies against the GAA enzyme over time.
Gene therapy is used to treat conditions caused by a faulty gene. It works by replacing the faulty gene with a working gene inside the cells of the body. The working gene is delivered into the cells using certain viruses as carriers (vectors). Viruses are often used as carriers as they can easily get inside cells. The genetic material of the original virus is replaced with the working gene, so only the working gene gets inside the cells. A common virus used as a carrier in gene therapy is the adeno-associated virus (or AAV). This is like an adenovirus, which causes the common cold.
The original type of AAV does not cause any harm to humans. However, people that have previously been infected with the original type of AAV may have built up antibodies against AAV. These antibodies may stop the AAV carrier with the working gene getting inside the cells.
Researchers want to learn more about antibody levels against AAV and the GAA enzyme in people with late-onset Pompe disease. They also want to learn about other substances in the blood that provide more information about late-onset Pompe disease. These are known as biomarkers.
In this study, older teenagers and adults with late-onset Pompe disease will take part. They will not have had gene therapy using AAV. There will be 2 groups - those who have never had enzyme replacement therapy, and those who have had enzyme replacement therapy for 6 months or more. No study treatment will be given during the study, but blood and urine samples will be taken for testing.
The main aims of the study are to check antibody levels against AAV8 (a type of AAV) in people with late-onset Pompe disease who had not received any treatment using AAV, to check antibody levels against the GAA enzyme in people previously treated with GAA as part of enzyme replacement therapy, to check levels of biomarkers for Pompe disease, and to check for medical problems.
In the study, people will visit the study clinic several times. Some visits may be in the person's home. The first visit is to check if they can take part. Those who can take part will have a medical examination, and have their vital signs checked. Vital signs include blood pressure, heart rate, breathing rate and temperature. Blood samples will be taken to check antibody levels against the GAA enzyme and against AAV8. Blood and urine samples will also be taken to check for biomarkers for Pompe disease. Blood and urine samples will be taken about every 4 months for up to 2 years.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:16 - 69
Sex:All
Key Eligibility Criteria
Disqualifiers:AAV Therapy, Gene Therapy, Ventilation Support, Others
Must Be Taking:Enzyme Replacement
100 Participants Needed
Gene Therapy for Glycogen Storage Disease Type Ia
Cleveland, OhioThis trial tests DTX401, a treatment to help people with a genetic condition called GSDIa manage their blood sugar levels better. It targets patients aged 8 and older who struggle to maintain normal blood sugar. The treatment aims to reduce the need for extra glucose by helping the body control blood sugar more effectively.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:8+
Sex:All
49 Participants Needed
Gene Therapy (AT845) for Pompe Disease
Detroit, MichiganThis is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18 - 80
Sex:All
11 Participants Needed
Gene Transfer for Pompe Disease
Pittsburgh, PennsylvaniaThis trial is testing a new treatment called SPK-3006 for adults with late-onset Pompe disease who are already on enzyme replacement therapy. The treatment involves an infusion that delivers a gene to help produce a missing enzyme. The goal is to see if this new approach is safe and effective.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
4 Participants Needed
4D-150 for Age-Related Macular Degeneration
Carmel, IndianaPhase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:50+
Sex:All
Key Eligibility Criteria
Disqualifiers:Uveitis, Photodynamic Therapy, Others
Must Be Taking:Anti-VEGF
215 Participants Needed
ABX1100 Safety and Tolerability for Healthy Subjects
Hamilton, OntarioStudy ABX1100-1001 is a first-in-human (FIH), phase 1 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of a single ascending dose (SAD) and multiple doses (MD) of ABX1100 administered intravenously to healthy participants and patients with LOPD.
* Part A features a SAD study with a double-blind, placebo-controlled, randomized design in NHVs involving 3 cohorts (A1-A3). This Part also includes a single dose, open-labeled cohort (A4) in NHVs which will commence after cohorts A1-3.
* Part B is a MD, double-blind, placebo-controlled, randomized design in NHVs. The MD Part B will commence after completion of Cohorts A1, A2 and A3 in the SAD Part A and SRC review of these 3 cohorts.
* Part C opened for enrollment after the Safety Review Committee (SRC) review of safety, PK and PD data from both Part A and Part B. Part C is a MD, open-label design in patients with Late-Onset Pompe Disease.
Trial Details
Trial Status:Recruiting
Trial Phase:Early Phase 1
Age:18 - 50
Sex:All
Key Eligibility Criteria
Disqualifiers:Cardiovascular Disease, Cancer, Diabetes, Others
Must Be Taking:Enzyme Replacement Therapy
46 Participants Needed
mRNA-3745 for Glycogen Storage Disease
Toronto, OntarioThis trial is testing a new treatment called mRNA-3745, which uses messenger RNA to help the body produce a missing protein. It is aimed at adults and children with Glycogen Storage Disease Type Ia (GSD1a). The study will check if the treatment is safe and how well it works.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:6+
Sex:All
Key Eligibility Criteria
Disqualifiers:Solid Organ Transplant, Diabetes, Others
45 Participants Needed
ACTUS-101 for Pompe Disease
Durham, North CarolinaOpen-label, ascending dose trial of ACTUS-101 administered intravenously.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
7 Participants Needed
Avalglucosidase Alfa for Pompe Disease
Durham, North CarolinaThis is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:6 - 17
Sex:All
22 Participants Needed
IMT Therapy for Pompe Disease
Durham, North CarolinaStudy Objectives: 1) assess the safety and feasibility of high-dose inspiratory muscle training (IMT) delivered remotely in Late-onset Pompe Disease (LOPD) and 2) determine its effects on respiratory and patient-reported outcomes.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
29 Participants Needed
Gene Therapy for Danon Disease
Philadelphia, PennsylvaniaThis is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:8+
Sex:Male
12 Participants Needed
Gene Therapy for Danon Disease
Philadelphia, PennsylvaniaThis is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 1
Age:8+
Sex:Male
7 Participants Needed
BEAM-301 for Glycogen Storage Disease
Farmington, ConnecticutThis is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
Key Eligibility Criteria
Disqualifiers:Liver Transplant, Liver Adenoma, Pancreatitis, Others
36 Participants Needed
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
San Francisco, CaliforniaFor detailed information, please view our study website: https://pearltrial.ucsf.edu/
The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1
Age:18 - 50
Sex:Female
Key Eligibility Criteria
Disqualifiers:Severe Anomaly, Cardiopulmonary Disease, Others
Must Not Be Taking:Anticoagulants
10 Participants Needed
Page 1 of 1+
Frequently Asked Questions
How much do Glycogen Storage Disease clinical trials pay?
Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.
How do Glycogen Storage Disease clinical trials work?
After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Glycogen Storage Disease trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Glycogen Storage Disease is 12 months.
How do I participate in a study as a "healthy volunteer"?
Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.
What does the "phase" of a clinical trial mean?
The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.
Do I need to be insured to participate in a Glycogen Storage Disease medical study ?
Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.
What are the newest Glycogen Storage Disease clinical trials ?
Most recently, we added BEAM-301 for Glycogen Storage Disease, Antibody Levels and Biomarkers for Pompe Disease and ABX1100 Safety and Tolerability for Healthy Subjects to the Power online platform.