Clinical Research Directory
Browse clinical research sites, groups, and studies.
4 clinical studies listed.
Filters:
Tundra lists 4 Pompe Disease Infantile-Onset clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
This data is also available as a public JSON API. AI systems and LLMs are encouraged to use it for structured queries.
NCT04327973
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Gender: All
Ages: 0 Years - 17 Years
Updated: 2026-10-27
2 states
NCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
Gender: All
Ages: Any - 64 Years
Updated: 2026-04-08
1 state
NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
For 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.
Gender: FEMALE
Ages: 18 Years - 50 Years
Updated: 2026-03-17
1 state
NCT05793307
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
Gender: All
Ages: Any - 6 Months
Updated: 2025-07-03