256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 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 | NCT02898753 (ClinicalTrials.gov) | June 21, 2017 | 8/9/2016 | VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease | A Three-Month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 Versus Myozyme®/Lumizyme® in Patients With Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment With VAL-1221 in All Patients | Pompe Disease | Drug: VAL-1221;Drug: RhGAA | Valerion Therapeutics, LLC | NULL | Terminated | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United States;United Kingdom |
| 5 | EUCTR2016-004578-16-GB (EUCTR) | 19/06/2017 | 29/03/2017 | A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 in Ambulatory and Ventilator-free Patients with Pompe Disease | A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patients with Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment with VAL-1221 in all Patients | Late-Onset GSD-II (Pompe Disease) MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10045253;Term: Type II glycogen storage disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: VAL-1221 Product Code: VAL-1221 INN or Proposed INN: VAL-1221 Trade Name: Myozyme Product Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Valerion Therapeutics, LLC | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;United Kingdom | ||
| 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 | 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 |
| 18 | NCT00701701 (ClinicalTrials.gov) | December 14, 2008 | 17/6/2008 | 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;Glycogen Storage Disease Type II (GSD-II);Glycogenesis 2 Acid Maltase Deficiency | Biological: Myozyme® (alglucosidase alfa) | Genzyme, a Sanofi Company | NULL | Terminated | 1 Month | N/A | All | 4 | Phase 4 | United States;Israel;Canada |
| 19 | 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 | |||||
| 20 | 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 | |||
| 21 | 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 | ||||
| 22 | 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 | ||||
| 23 | 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 | |||
| 24 | 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 | ||||
| 25 | EUCTR2021-004047-25-Outside-EU/EEA (EUCTR) | 27/07/2021 | Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients with Infantile-Onset Pompe Disease with One Year Alglucosidase Alfa Treatment - APOLLO-IOPD | Infantile-Onset Pompe Disease 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: MYOZYME® Product Name: Alglucosidase Alfa Product Code: GZ419829 INN or Proposed INN: ALGLUCOSIDASE ALFA | Sanofi(China) Investment Co Ltd, Shanghai Branch | NULL | NA | Female: yes Male: yes | Phase 4 | China | ||||
| 26 | 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 |