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 | 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 |
| 2 | 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 | ||
| 3 | NCT02185651 (ClinicalTrials.gov) | October 2016 | 1/7/2014 | A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction | A Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease;Hypersensitivity Reaction | Drug: Zavesca® Prescription | University of Florida | Amicus Therapeutics | Terminated | 18 Years | 65 Years | All | 2 | Phase 1 | United States |
| 4 | EUCTR2013-001768-48-AT (EUCTR) | 21/02/2016 | 12/01/2016 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Portugal;France;United States;Belgium;Austria;Netherlands;Germany;Japan;Italy;United Kingdom;Switzerland | ||
| 5 | EUCTR2013-001768-48-PT (EUCTR) | 30/01/2015 | 02/07/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | France;United States;Portugal;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;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 |
| 6 | EUCTR2013-001768-48-DE (EUCTR) | 23/09/2014 | 22/05/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Colombia;Germany;Japan;Italy;United Kingdom | ||
| 7 | EUCTR2013-001768-48-IT (EUCTR) | 22/09/2014 | 01/07/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 17.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: GILT-rhGAA Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;United Kingdom;Italy | ||
| 8 | EUCTR2013-001768-48-BE (EUCTR) | 08/09/2014 | 12/05/2014 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alpha Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Portugal;France;United States;Argentina;Belgium;Austria;Netherlands;Germany;Colombia;Japan;Italy;United Kingdom | ||
| 9 | NCT01924845 (ClinicalTrials.gov) | April 2014 | 13/8/2013 | BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects With Late-onset Pompe Disease | Late-onset Pompe Disease | Drug: BMN 701 | BioMarin Pharmaceutical | NULL | Terminated | 18 Years | N/A | All | 24 | Phase 3 | United States;Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom |
| 10 | EUCTR2013-001768-48-GB (EUCTR) | 27/11/2013 | 22/10/2013 | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 INN or Proposed INN: reveglucosidase alfa Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | Portugal;France;United States;Belgium;Austria;Denmark;Netherlands;Germany;Colombia;Japan;Italy;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 |
| 11 | 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 | ||
| 12 | EUCTR2011-001805-28-DE (EUCTR) | 14/02/2013 | 28/06/2012 | A long term study of the safety of BMN 701 in patients with POMPE disease | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | POMPE disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 21 | Phase 2 | United States;Australia;Germany;New Zealand;United Kingdom | ||
| 13 | 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 | ||
| 14 | EUCTR2010-023561-22-DE (EUCTR) | 04/07/2012 | 07/02/2012 | A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease | Pompe disease MedDRA version: 16.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 1;Phase 2 | France;United States;Netherlands;Germany;United Kingdom | ||
| 15 | EUCTR2011-001805-28-GB (EUCTR) | 03/07/2012 | 26/04/2012 | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | Pompe disease MedDRA version: 18.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;France;Australia;Germany;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 | 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 | ||
| 17 | NCT01380743 (ClinicalTrials.gov) | October 31, 2011 | 23/6/2011 | Drug-drug Interaction Study | An Open-Label, Multi-Center, International Study to Investigate Drug-Drug Interactions Between AT2220 and Alglucosidase Alfa in Patients With Pompe Disease | Pompe Disease | Drug: duvoglustat;Drug: rhGAA | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | All | 25 | Phase 2 | United States;Canada;France;United Kingdom |
| 18 | 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 | ||
| 19 | 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 | ||
| 20 | EUCTR2010-023561-22-GB (EUCTR) | 15/08/2011 | 11/04/2011 | A Study of the Safety and Tolerability of BMN 701 in Patients with Late-onset Pompe Disease | A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe Disease - A Phase 1/2 Study of BMN 701 in Patients with Late-onset Pompe Disease | Pompe disease MedDRA version: 13.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 701 Product Code: BMN 701 Other descriptive name: GILT-rhGAA | BioMarin Pharmaceutical Inc | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 1;Phase 2 | United States;Germany;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 |
| 21 | NCT01451879 (ClinicalTrials.gov) | October 2008 | 15/9/2011 | Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies | Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy | Pompe Disease | Drug: Rituximab;Drug: Miglustat | University of Florida | NULL | Completed | N/A | 65 Years | All | 11 | N/A | United States |
| 22 | 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 | |||
| 23 | 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 | |||
| 24 | 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 | |||
| 25 | 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 |
| 26 | 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 |
| 27 | EUCTR2005-002829-31-GB (EUCTR) | 31/08/2005 | 07/07/2005 | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Late-Onset Pompe Disease (Glycogen Storage Disease Type II) - mini LOTS | Glycogen Storage Disease type II (Pompe´s disease) | Genzyme Europe BV | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 2 | Belgium;United Kingdom | |||
| 28 | 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 |
| 29 | EUCTR2004-002168-59-IT (EUCTR) | 14/10/2004 | 03/01/2005 | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II) | Treatment for Type II Glycogenosis MedDRA version: 6.1;Level: PT;Classification code 10053185 | Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA INN or Proposed INN: alglucosidase alfa | GENZYME | NULL | Not Recruiting | Female: yes Male: yes | 20 | Italy | |||
| 30 | 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 |
| 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 | 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 |
| 32 | 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 |
| 33 | 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 |
| 34 | NCT00051935 (ClinicalTrials.gov) | January 2003 | 17/1/2003 | A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II | Open-Label, Pilot Study of the Safety, Pharmacokinetics and Pharmacodynamics of Recombinant Human Acid Alpha-Glucosidase (rhGAA) as Enzyme Replacement Therapy in Siblings With Glycogen Storage Disease Type II (GSD-II). | Glycogen Storage Disease Type II;Pompe Disease;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: Alglucosidase alfa | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 2 | Phase 2 | United States |
| 35 | NCT00025896 (ClinicalTrials.gov) | May 2001 | 31/10/2001 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | A Prospective Multinational, Multicenter, Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease | Pompe Disease;Glycogen Storage Disease Type II;Acid Maltase Deficiency Disease;Glycogenosis 2 | Drug: recombinant human acid alpha-glucosidase (rhGAA) | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 8 | Phase 2 | United States |
| 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 | EUCTR2015-000512-18-Outside-EU/EEA (EUCTR) | 02/03/2015 | A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease | A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 17.1;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lumizyme® INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: rhGAA | Genzyme Corporation Inc | NULL | NA | Female: yes Male: yes | 114 | Phase 4 | United States | |||
| 37 | 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 |