256. Muscle glycogenosis Clinical trials / Disease details
Clinical trials : 180 / Drugs : 133 - (DrugBank : 29) / Drug target genes : 25 - Drug target pathways : 105
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02525172 (ClinicalTrials.gov) | August 2015 | 10/8/2015 | Immune Modulation Therapy for Pompe Disease | Immune Modulation Therapy for ERT-naïve or ERT-treated Pompe Disease Patients | Pompe Disease | Drug: Rituximab;Drug: intravenous immune globulin;Drug: Bortezomib;Drug: Methotrexate | National Taiwan University Hospital | NULL | Recruiting | N/A | N/A | Both | 8 | Phase 4 | Taiwan |
| 2 | NCT00701129 (ClinicalTrials.gov) | October 2009 | 17/6/2008 | 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 | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II | Biological: Alglucosidase Alfa;Drug: Methotrexate;Drug: Rituximab | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | All | 4 | Phase 4 | United States;Israel |
| 3 | NCT01451879 (ClinicalTrials.gov) | October 2008 | 15/9/2011 | Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies | Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease | Drug: Rituximab;Drug: Miglustat | University of Florida | NULL | Completed | N/A | 65 Years | All | 11 | N/A | United States |
| 4 | EUCTR2015-000583-34-Outside-EU/EEA (EUCTR) | 15/04/2015 | Immune Tolerance Induction Study | An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients with Pompe Disease Who Have Previously Received Myozyme | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 17.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Alglucosidase alfa Product Code: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA Product Name: Cyclophosphamide INN or Proposed INN: CYCLOPHOSPHAMIDE MONOHYDRATE Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE Product Name: Rituximab INN or Proposed INN: RITUXIMAB Product Name: Methotrexate INN or Proposed INN: METHOTREXATE SODIUM Other descriptive name: METHOTREXATE SODIUM | Genzyme Corporation Inc | NULL | NA | Female: yes Male: yes | 9 | United States;Israel | ||||
| 5 | EUCTR2015-000584-14-Outside-EU/EEA (EUCTR) | 15/04/2015 | 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 | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naïve CRIM(-) Patients with Infantile-Onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 17.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA Product Name: Rituximab INN or Proposed INN: RITUXIMAB Product Name: Methotrexate INN or Proposed INN: METHOTREXATE SODIUM Other descriptive name: METHOTREXATE SODIUM | Genzyme Corporation Inc | NULL | NA | Female: yes Male: yes | 4 | United States |