Glycogen storage disease type II
Information
- Disease name
- Glycogen storage disease type II
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04093349 | Active, not recruiting | Phase 1/Phase 2 | A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) | October 1, 2020 | April 2032 |
| NCT05164055 | Active, not recruiting | Phase 4 | Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) | July 11, 2022 | December 31, 2024 |
| NCT00074919 | Approved for marketing | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease | December 2003 | February 2007 | |
| NCT00053573 | Completed | Phase 1/Phase 2 | rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) | February 2003 | November 2006 |
| NCT00059280 | Completed | Phase 2/Phase 3 | A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease | April 2003 | September 2005 |
| NCT00074932 | Completed | N/A | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease | November 2004 | December 2006 |
| NCT00077662 | Completed | A Prospective, Observational Study in Patients With Late-Onset Pompe Disease | March 2004 | January 2006 | |
| NCT00125879 | Completed | Phase 2/Phase 3 | Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 | June 2005 | December 2006 |
| NCT00001331 | Completed | Genetic and Family Studies of Inherited Muscle Diseases | May 1993 | March 2002 | |
| NCT00515398 | Completed | A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease | August 2007 | January 2008 | |
| NCT00701129 | Completed | Phase 4 | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease | October 2009 | March 2013 |
| NCT00763932 | Completed | Phase 2 | 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 | April 2003 | July 2006 |
| NCT02363153 | Completed | N/A | Diet and Exercise in Pompe Disease | November 6, 2017 | May 12, 2021 |
| NCT02801539 | Completed | N/A | Respiratory Muscle Training in L-Onset Pompe Disease (LOPD) | February 2, 2017 | April 17, 2019 |
| NCT03687333 | Completed | Phase 4 | Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment | December 4, 2018 | December 30, 2020 |
| NCT00025896 | Completed | Phase 2 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | May 2001 | September 2002 |
| NCT00051935 | Completed | Phase 2 | A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II | January 2003 | October 2003 |
| NCT06130228 | Not yet recruiting | Phase 2 | Nutritional Therapy in Late-onset Pompe Disease | April 1, 2024 | April 1, 2025 |
| NCT05017402 | Not yet recruiting | Higher Dose of Alglucosidase Alpha for Pompe Disease | September 1, 2021 | December 31, 2026 | |
| NCT00231400 | Recruiting | Pompe Disease Registry Protocol | September 15, 2004 | January 31, 2034 | |
| NCT05951790 | Recruiting | N/A | Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease | March 1, 2023 | April 1, 2024 |
| NCT04848779 | Recruiting | A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) | June 10, 2021 | March 30, 2026 | |
| NCT04910776 | Recruiting | Phase 3 | Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa | September 1, 2021 | August 28, 2026 |
| NCT02761421 | Unknown status | Effect of Motor Development, Motor Function and Electrophysiologic Findings of IOPD Under ERT | January 2016 | December 2016 | |
| NCT02635269 | Unknown status | N/A | Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy | January 2016 | December 2020 |
| NCT02385162 | Withdrawn | Biomarker for Glycogen Storage Diseases (BioGlycogen) | August 20, 2018 | February 28, 2021 | |
| NCT01656590 | Withdrawn | Phase 2 | High Protein and Exercise Therapy Plus Nocturnal Enteral Feeding in Juvenile-onset Pompe Disease | October 2012 | August 2013 |
- MeSH unique ID (MeSH (Medical Subject Headings))
- D006009