256. 筋型糖原病
［臨床試験数：161，薬物数：114（DrugBank：23），標的遺伝子数：26，標的パスウェイ数：106］
Searched query = "Muscle glycogenosis", "Muscular glycogenosis", "Muscle glycogen storage disease", "Muscular glycogen storage disease", "Glycogen storage disease type 0", "GSD0", "Glycogen synthase deficiency", "Glycogen storage disease type II", "GSDII", "Pompe disease", "Alpha-1,4-glucosidase acid deficiency", "Glycogen storage disease type III", "GSDIII", "Cori disease", "Glycogen debranching enzyme deficiency", "Glycogen storage disease type IV", "GSDIV", "Andersen disease", "Glycogen-branching enzyme deficiency", "GBED", "Glycogen storage disease type V", "GSDV", "McArdle disease", "Muscle phosphorylase deficiency", "Muscular phosphorylase deficiency", "Glycogen storage disease type VII", "GSDVII", "Tarui disease", "Phosphofructokinase deficiency", "PFK deficiency", "Glycogen storage disease type IX", "GSDIX", "Phosphorylase kinase deficiency", "Phosphoglycerate kinase deficiency", "PGK deficiency", "Glycogen storage disease type X", "GSDX", "Phosphoglycerate mutase deficiency", "Glycogen storage diseass type XI", "GSDXI", "Kanno disease", "Lactate dehydrogenase deficiency", "Glycogen storage diseass type XII", "GSDXII", "Aldolase A deficiency", "Glycogen storage diseass type XIII", "GSDXIII", "Beta-enolase deficiency", "Glycogen storage diseass type XIV", "GSDXIV", "Phosphoglucomutase deficiency", "Glycogen storage diseass type XV", "GSDXV", "Glycogenin 1 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | EUCTR2018-000755-40-AT (EUCTR) | 25/09/2019 | 04/06/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 2 | EUCTR2018-000755-40-GR (EUCTR) | 28/08/2019 | 21/05/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 3 | EUCTR2018-000755-40-NL (EUCTR) | 28/08/2019 | 24/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: ALGLUCOSIDASE ALFA Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden | ||
| 4 | EUCTR2018-000755-40-SI (EUCTR) | 22/05/2019 | 03/04/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 5 | EUCTR2018-000755-40-DE (EUCTR) | 08/04/2019 | 29/11/2018 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 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 |
| 6 | EUCTR2018-000755-40-GB (EUCTR) | 18/03/2019 | 06/12/2018 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: N/A Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 7 | EUCTR2018-000755-40-BE (EUCTR) | 11/03/2019 | 03/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 8 | EUCTR2018-000755-40-SK (EUCTR) | 04/03/2019 | 08/01/2019 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: N/A Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 9 | EUCTR2018-000755-40-HU (EUCTR) | 28/02/2019 | 28/12/2018 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: ALGLUCOSIDASE ALFA Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Israel;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Romania;Bulgaria;Germany;Japan;New Zealand;Sweden | ||
| 10 | EUCTR2018-000755-40-DK (EUCTR) | 06/02/2019 | 30/11/2018 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 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 |
| 11 | EUCTR2018-000755-40-SE (EUCTR) | 30/01/2019 | 12/12/2018 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared withAlglucosidase Alfa/Placebo. | A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosidase Alfa/Placebo | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification code 10075702;Term: Pompe's disease late onset;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT2221 Product Code: AT2221 INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme INN or Proposed INN: ALGLUCOSIDASE ALFA | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Herzegovina;Korea, Republic of;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 12 | NCT03687333 (ClinicalTrials.gov) | December 4, 2018 | 24/9/2018 | 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 | Glycogen Storage Disease Type II | Drug: ALGLUCOSIDASE ALFA (MYOZYME) | Genzyme, a Sanofi Company | NULL | Active, not recruiting | N/A | 12 Months | All | 10 | Phase 4 | China |
| 13 | EUCTR2016-000942-77-PT (EUCTR) | 24/07/2018 | 12/04/2018 | 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 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 | 96 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Bulgaria;Germany;Japan;Sweden | ||
| 14 | 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 |
| 15 | 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 | ||
| 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 |
| 16 | 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 | ||
| 17 | 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 | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 18 | 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: 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 | Phase 3 | 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 | ||
| 19 | 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 | ||
| 20 | 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 | ||
| 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 |
| 21 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden | ||
| 22 | 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 | ||
| 23 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3;Phase 4 | United States;Taiwan;Canada;Turkey;Russian Federation;Germany;India | ||
| 24 | 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;China;India | ||
| 25 | 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 | ||
| 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 |
| 26 | NCT00567073 (ClinicalTrials.gov) | November 2011 | 1/12/2007 | Pompe Pregnancy Sub-Registry | A Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe Disease | Glycogen Storage Disease Type II (GSD-II);Pompe Disease (Late-onset);Glycogenesis 2 Acid Maltase Deficiency | Biological: alglucosidase alpha;Other: No Treatment | Genzyme, a Sanofi Company | NULL | Recruiting | N/A | N/A | Female | 20 | United States;Italy | |
| 27 | 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 | ||
| 28 | 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 | ||
| 29 | 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 |
| 30 | 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 |
| 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 |
| 31 | EUCTR2006-003644-31-DE (EUCTR) | 03/08/2007 | 11/07/2007 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | 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 Product Code: rhGAA, acid alpha glucosidase INN or Proposed INN: Alglucosidase alfa Other descriptive name: alglucosidase alpha | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 82 | Germany;Netherlands;France | |||
| 32 | EUCTR2006-003644-31-NL (EUCTR) | 01/08/2007 | 04/06/2007 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | 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 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 | 82 | Germany;Netherlands;France | |||
| 33 | 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 | |||
| 34 | 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 | |||||
| 35 | EUCTR2006-003644-31-FR (EUCTR) | 18/01/2007 | 11/12/2006 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | Glycogen Storage Disease type II (Pompe's disease) | Trade Name: Myozyme Product Name: Myozyme Product Code: rhGAA, acid alpha glucosidase INN or Proposed INN: Alglucosidase alfa Other descriptive name: alglucosidase alpha | Genzyme Europe BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Germany;Netherlands;France | |||
| 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 |
| 36 | NCT00268944 (ClinicalTrials.gov) | December 2005 | 22/12/2005 | Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support | Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | Both | 5 | Phase 3 | France |
| 37 | NCT00158600 (ClinicalTrials.gov) | September 2005 | 8/9/2005 | A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease | Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Efficacy and Pharmacokinetics of Myozyme in Patients With Late-Onset Pompe Disease. | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: alglucosidase alfa;Drug: Placebo | Genzyme, a Sanofi Company | NULL | Completed | 8 Years | N/A | All | 90 | Phase 3 | United States;France;Netherlands |
| 38 | EUCTR2005-001629-27-DE (EUCTR) | 03/08/2005 | 09/06/2005 | A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 | A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 | Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease. | Product Name: Myozyme INN or Proposed INN: Alglucosidase alfa Other descriptive name: n.a. | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 17 | Germany;Italy | |||
| 39 | EUCTR2005-001629-27-IT (EUCTR) | 21/06/2005 | 21/06/2005 | A Long-term Continuation Study of Patients With Infantile-Onset Pompe Disease WhoWere Previously Enrolled in Protocol AGLU01602 | A Long-term Continuation Study of Patients With Infantile-Onset Pompe Disease WhoWere Previously Enrolled in Protocol AGLU01602 | Pompe disease or glycogenosis type II MedDRA version: 6.1;Level: HLT;Classification code 10024579 | Product Name: Myozyme Product Code: NA Other descriptive name: NA | GENZYME | NULL | Not Recruiting | Female: yes Male: yes | Germany;Italy | ||||
| 40 | NCT00125879 (ClinicalTrials.gov) | June 2005 | 1/8/2005 | Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 | A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602 | Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 16 | Phase 2;Phase 3 | United States;France;Germany;Israel;Italy;Netherlands;Taiwan |
| 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 |
| 41 | NCT00250939 (ClinicalTrials.gov) | February 2005 | 8/11/2005 | A Study of rhGAA in Patients With Late-Onset Pompe Disease | Single-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease | Pompe Disease (Late-onset);Glycogen Storage Disease Type II (GSD-II);Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | 5 Years | 18 Years | Both | 5 | Phase 2 | Netherlands |
| 42 | NCT00074932 (ClinicalTrials.gov) | November 2004 | 23/12/2003 | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Late-onset Pompe Disease | Glycogen Storage Disease Type II;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 9 | N/A | United States |
| 43 | NCT00074919 (ClinicalTrials.gov) | December 2003 | 23/12/2003 | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease | Expanded Access Use of Myozyme (Alglucosidase Alfa) in Patients With Infantile-onset Pompe Disease | Glycogen Storage Disease Type II;Glycogenosis 2 | Biological: alglucosidase alfa | Genzyme, a Sanofi Company | NULL | Approved for marketing | N/A | N/A | Both | N/A | United States | |
| 44 | 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 |
| 45 | NCT00763932 (ClinicalTrials.gov) | April 2003 | 30/9/2008 | 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 | A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies | Pompe Disease Infantile-Onset;Glycogen Storage Disease Type II | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | Both | 7 | Phase 2 | United States;France;South Africa |
| 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 |
| 46 | NCT00765414 (ClinicalTrials.gov) | April 2003 | 2/10/2008 | Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies. | An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies | Pompe Disease Late-Onset;Glycogen Storage Disease Type II GSD II | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | Female | 1 | Phase 2 | United States |
| 47 | NCT00053573 (ClinicalTrials.gov) | February 2003 | 31/1/2003 | rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Biological: Myozyme | Genzyme, a Sanofi Company | NULL | Completed | 6 Months | 36 Months | Both | 20 | Phase 1;Phase 2 | United States;France;Israel;United Kingdom |
| 48 | EUCTR2013-002257-30-GB (EUCTR) | 21/10/2013 | A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients | AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID a-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE | Pompe disease MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;Netherlands;United Kingdom | ||||
| 49 | 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 | |||
| 50 | 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 | |||
| 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 |
| 51 | 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 | ||||
| 52 | 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 | |||
| 53 | 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 | ||||
| 54 | 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 |