2 completed, 2 recruiting, 1 active not recruiting, 1 available. 2 Phase 1, 2 Phase 2, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Recruiting 1 |
| Phase 2 | 2 | Active not recruiting 1, Completed 1 |
| Not applicable | 1 | Completed 1 |
Follow Pompe Disease Infantile-Onset: new trials by email Literature Review of Pompe Disease Infantile-Onset trials Research Report on Pompe Disease Infantile-Onset
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug GC301 - Pompe Disease Infantile-Onset | Phase 1 / Phase 2 | Active not recruiting | GeneCradle Inc | 16 | 2023-06-02 | 2025-07-03 |
| Registry of Patients Diagnosed With Lysosomal Storage Diseases There is no intervention - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | University of California, San Francisco | 250 | 2022-05-31 | 2026-04-08 | |
| MSOT in Pompe Disease Multispectral Optoacoustic Tomography - Pompe Disease, Pompe Disease (Late-onset) | Not applicable | Completed | University of Erlangen-Nürnberg Medical School | 20 | 2022-05-17 | 2025-03-12 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease. Cardiopulmonary exercise testing (CPET) - Pompe Disease Infantile-Onset | Unknown | Rambam Health Care Campus | 4 | 2018-06-24 | 2021-02-16 | |
| Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies Myozyme - Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II | Phase 2 | Completed | Genzyme, a Sanofi Company | 7 | 2003-04-01 | 2014-02-05 |
| Expanded Access for ATB200/AT2221 for the Treatment of IOPD ATB200, AT2221 - Pompe Disease Infantile-Onset | Available | Amicus Therapeutics | 2025-09-10 |
Gaucher Disease (2) · Glycogen Storage Disease Type II (2) · Mucopolysaccharidosis IV (2) · Mucopolysaccharidosis VI (2) · Mucopolysaccharidosis VII (2) · Wolman Disease (2) · Lysosomal Storage Diseases (1) · Metabolism, Inborn Errors (1) · Mucopolysaccharidoses (1) · Mucopolysaccharidosis I (1) · Mucopolysaccharidosis II (1) · Sudden Infant Death (1)
Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Carbohydrate Metabolism, Inborn Errors (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Lysosomal Storage Diseases, Nervous System (4) · Metabolic Diseases (4) · Nervous System Diseases (4) · Nutritional and Metabolic Diseases (4) · Lysosomal Storage Diseases (3) · Metabolism, Inborn Errors (3) · Cholesterol Ester Storage Disease (2) · Connective Tissue Diseases (2)
GAA protein, human (2) · Exercise Test (1) · GALNS protein, human (1) · Iduronidase (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
University of California, San Francisco (2) · Amicus Therapeutics (1) · GeneCradle Inc (1) · Genzyme, a Sanofi Company (1) · Rambam Health Care Campus (1) · University of Erlangen-Nürnberg Medical School (1)
United States (4) · China (1) · France (1) · Germany (1) · Israel (1) · Italy (1) · South Africa (1) · Taiwan (1)
Ferdinand Knieling, MD (1) · Lea Bentur (1) · Regina Trollmann, MD (1) · Tippi C MacKenzie, MD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 39245687, PMID 39891377, PMID 9918480