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 | EUCTR2016-000942-77-HU (EUCTR) | 29/01/2019 | 04/12/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Bulgaria;Germany;Japan;Sweden | ||
| 2 | EUCTR2016-000942-77-BG (EUCTR) | 23/10/2018 | 24/07/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 3 | EUCTR2016-000942-77-PT (EUCTR) | 24/07/2018 | 12/04/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 INN or Proposed INN: neoGAA Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 4 | EUCTR2010-022231-11-GB (EUCTR) | 07/11/2017 | 17/08/2017 | Pharmacokinetics of Alglucosidase Alfa in Patients with Pompe Disease | A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics of Alglucosidase Alfa in Patients with Pompe Disease - PAPAYA | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000011505 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | United States;Ukraine;Russian Federation;Bulgaria;Germany;United Kingdom;India | ||
| 5 | NCT02240407 (ClinicalTrials.gov) | October 17, 2017 | 11/9/2014 | Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease | Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Patients With Late-Onset Pompe Disease (LOPD) | Pompe Disease | Genetic: Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase;Drug: Rapamycin;Other: saline;Drug: Rituxan;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: Lidocaine;Drug: LMX 4 Topical Cream | University of Florida | NULL | Active, not recruiting | 18 Years | 50 Years | All | 2 | Phase 1 | United States |
| 6 | EUCTR2016-003475-21-GB (EUCTR) | 17/05/2017 | 03/01/2017 | A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase Alfa | An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Avalglucosidase Alfa (NeoGAA) Product Code: GZ402666 INN or Proposed INN: Avalglucosidase Alfa (NeoGAA) Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 34 | Phase 2 | United States;France;Taiwan;Germany;Japan;United Kingdom | ||
| 7 | EUCTR2016-000942-77-NL (EUCTR) | 21/03/2017 | 15/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 INN or Proposed INN: NEO-GAA Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 8 | EUCTR2016-000942-77-AT (EUCTR) | 06/02/2017 | 31/10/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 9 | EUCTR2016-000942-77-DE (EUCTR) | 18/11/2016 | 27/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 10 | EUCTR2016-000942-77-ES (EUCTR) | 20/10/2016 | 22/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 19.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 INN or Proposed INN: NeoGAA Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of | ||
| 11 | EUCTR2016-000942-77-BE (EUCTR) | 14/10/2016 | 01/07/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease - COMET | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Germany;Japan;Sweden | ||
| 12 | EUCTR2016-000942-77-CZ (EUCTR) | 05/10/2016 | 27/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 96 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 13 | EUCTR2016-000942-77-IT (EUCTR) | 26/08/2016 | 06/02/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease - COMET | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: a-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA) Trade Name: MYOZYME® INN or Proposed INN: ALGLUCOSIDASE ALFA | GENZYME CORPORATION | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 3 | Czech Republic;Poland;Belgium;Spain;Austria;Denmark;Germany;Netherlands;United Kingdom;Italy;Sweden | ||
| 14 | EUCTR2016-000942-77-DK (EUCTR) | 25/08/2016 | 02/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 19.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 15 | EUCTR2016-000942-77-SE (EUCTR) | 24/08/2016 | 08/06/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 16 | EUCTR2016-000942-77-GB (EUCTR) | 15/07/2016 | 04/05/2016 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients with Late-onset Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 17 | EUCTR2013-003321-28-DK (EUCTR) | 14/11/2014 | 18/09/2014 | Avalglucosidase Alfa Extension Study | An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Avalglucosidase Alfa Product Code: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 21 | Phase 2 | United States;France;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy | ||
| 18 | EUCTR2013-003321-28-DE (EUCTR) | 20/10/2014 | 25/09/2014 | Avalglucosidase Alfa Extension Study | An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Avalglucosidase Alfa Product Code: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 21 | Phase 2 | France;United States;Belgium;Denmark;Netherlands;Germany;United Kingdom;Italy | ||
| 19 | EUCTR2013-003321-28-NL (EUCTR) | 25/09/2014 | 16/07/2014 | NeoGAA Extension Study | An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 INN or Proposed INN: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 2 | France;United States;Belgium;Denmark;Germany;Netherlands;United Kingdom;Italy | ||
| 20 | EUCTR2013-003321-28-BE (EUCTR) | 12/05/2014 | 17/02/2014 | Avalglucosidase Alfa Extension Study | An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa (NeoGAA, GZ402666) In Patients With Pompe Disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Avalglucosidase Alfa Product Code: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 21 | Phase 2 | France;United States;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom | ||
| 21 | EUCTR2013-003321-28-FR (EUCTR) | 19/03/2014 | 17/06/2015 | NeoGAA Extension Study | An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | United States;France;Belgium;Denmark;Netherlands;Germany;Italy;United Kingdom | |||
| 22 | EUCTR2011-005595-42-DE (EUCTR) | 17/04/2013 | 30/11/2012 | A study to compare alglucosidase alfa manufactured at the 160 L scale and the 4000 L scale in patients with infantile-onset Pompe disease who have never been treated. | A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve 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] | Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Corporation, a Sanofi company | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 4 | United States;Taiwan;Canada;Turkey;Russian Federation;Germany;India | ||
| 23 | EUCTR2010-022231-11-DE (EUCTR) | 13/12/2012 | 01/08/2012 | Pharmacokinetics of Alglucosidase Alfa in Patients Aged 8 Years of Age andOlder | A Phase 3/4 Prospective Study to Characterize the Pharmacokinetics ofAlglucosidase Alfa in Patients Aged 8 Years of Age and Older - PAPAYA | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 1;Phase 4 | United States;Poland;Ukraine;Russian Federation;Bulgaria;Germany;United Kingdom;China;India | ||
| 24 | EUCTR2010-020611-36-NL (EUCTR) | 13/01/2012 | 23/08/2011 | A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa. | A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 14.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme Product Name: Myozyme Product Code: not applicable INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 4 | United States;Denmark;Germany;Netherlands;United Kingdom | ||
| 25 | EUCTR2010-020611-36-GB (EUCTR) | 12/10/2011 | 26/05/2011 | A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa. | A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme Product Name: Myozyme Product Code: not applicable INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 4 | United States;Netherlands;Germany;United Kingdom | ||
| 26 | EUCTR2010-020611-36-DE (EUCTR) | 28/09/2011 | 30/06/2011 | A study to evaluate muscle tissue, blood, urine and imaging assessments in patients with Late-Onset Pompe Disease treated with alglucosidase alfa. | A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa - Exploratory Muscle Biopsy Assessment Study (EMBASSY) | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 14.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Myozyme Product Name: Myozyme Product Code: not applicable INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 4 | United States;Netherlands;Germany;United Kingdom | ||
| 27 | NCT00976352 (ClinicalTrials.gov) | September 2010 | 13/7/2009 | Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease | Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease | Pompe Disease | Drug: rAAV1-CMV-GAA (study agent) Administration;Other: RMST | University of Florida | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 2 Years | 18 Years | All | 9 | Phase 1/Phase 2 | United States |
| 28 | EUCTR2005-002759-42-DE (EUCTR) | 18/07/2007 | 24/07/2009 | A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTS | A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease - LOTS | Glycogen Storage Disease type II (Pompe's disease) MedDRA version: 9.1;Level: LLT;Classification code 10036143;Term: Pompe's disease | Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: Alglucosidase alfa Other descriptive name: alglucosidase alpha | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 90 | Germany | |||
| 29 | NCT00520143 (ClinicalTrials.gov) | July 2007 | 21/8/2007 | Alglucosidase Alfa Temporary Access Program | Alglucosidase Alfa Temporary Access Program | Glycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-Onset);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA]) | Genzyme, a Sanofi Company | NULL | Approved for marketing | 18 Years | N/A | Both | N/A | United States | |
| 30 | EUCTR2007-001375-11-NL (EUCTR) | 16/05/2007 | 24/05/2007 | Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable | Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable | Pompe disease (glycogen storage disease type II) is a genetic, lysosomal storage disorder with a frequency of 1 in 40.000 newborns. The disease is caused by deficiency of alpha-glucosidase, a lysosomal hydrolase involved in the degradation of glycogen. | Trade Name: Myozyme Product Name: Myozyme Product Code: MYOZYME® | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Netherlands | |||||
| 31 | EUCTR2005-002829-31-GB (EUCTR) | 31/08/2005 | 07/07/2005 | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Glycogen Storage Disease type II (Pompe´s disease) | Product Name: Myozyme Product Code: rhGAA, acid alpha glucosidase INN or Proposed INN: Alglucosidase alfa Other descriptive name: alglucosidase alpha | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Belgium;United Kingdom | ||
| 32 | EUCTR2004-002168-59-IT (EUCTR) | 14/10/2004 | 03/01/2005 | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | Treatment for Type II Glycogenosis MedDRA version: 6.1;Level: PT;Classification code 10053185 | Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa | GENZYME | NULL | Not Recruiting | Female: yes Male: yes | 20 | Italy | |||
| 33 | NCT00059280 (ClinicalTrials.gov) | April 2003 | 22/4/2003 | A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease | An Open-label, Multicenter, Multinational Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease | Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | N/A | 26 Weeks | Both | 16 | Phase 2/Phase 3 | United States;France;Israel;Taiwan;United Kingdom |
| 34 | NCT00051935 (ClinicalTrials.gov) | January 2003 | 17/1/2003 | A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II | Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II). | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: Alglucosidase alfa | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 2 | Phase 2 | United States |
| 35 | NCT00025896 (ClinicalTrials.gov) | May 2001 | 31/10/2001 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: recombinant human acid alpha-glucosidase (rhGAA) | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 8 | Phase 2 | United States |
| 36 | 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 | ||||
| 37 | EUCTR2016-000942-77-PL (EUCTR) | 23/11/2017 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of Avalglucosidase Alfa (neoGAA ,GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | NA | Female: yes Male: yes | 100 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | |||
| 38 | 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 | ||||
| 39 | EUCTR2016-003475-21-DE (EUCTR) | 09/03/2017 | A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase Alfa | An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 INN or Proposed INN: - Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | Not Recruiting | Female: yes Male: yes | 34 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Taiwan;Germany;Japan;United Kingdom | |||
| 40 | EUCTR2015-000582-31-Outside-EU/EEA (EUCTR) | 15/04/2015 | An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen | An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen | 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] | Trade Name: Myozyme 50 mg powder for concentrate for solution for infusion INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation Inc | NULL | NA | Female: yes Male: yes | 14 | United States;Australia;Canada | ||||
| 41 | EUCTR2005-002829-31-BE (EUCTR) | 09/02/2006 | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Glycogen Storage Disease type II (Pompe´s disease) | Product Name: Myozyme Product Code: rhGAA, acid alpha glucosidase INN or Proposed INN: Alglucosidase alfa Other descriptive name: alglucosidase alpha | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 2 | Belgium;United Kingdom | |||
| 42 | EUCTR2016-003475-21-FR (EUCTR) | 02/06/2017 | A Study to Assess Safety and Efficacy of NeoGAA Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated with Alglucosidase Alfa | An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of NeoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: neoGAA Product Code: GZ402666 INN or Proposed INN: - Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA | Genzyme Corporation | NULL | NA | Female: yes Male: yes | 34 | Phase 2 | United States;France;Taiwan;Germany;Japan;United Kingdom | |||
| 43 | EUCTR2015-000512-18-Outside-EU/EEA (EUCTR) | 02/03/2015 | A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease | A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale | 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: Lumizyme® INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Corporation Inc | NULL | NA | Female: yes Male: yes | 114 | Phase 4 | United States |