256. 筋型糖原病 Muscle glycogenosis Clinical trials / Disease details
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
Showing 1 to 10 of 98 diseases
| 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 | EUCTR2020-004686-39-NL (EUCTR) | 04/10/2021 | 04/05/2021 | Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) With Avalglucosidase Alfa Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and ... | An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD) - Baby-COMET An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharm ... | Glycogen storage disease type II MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: Glycogen storage disease type II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Glycogen storage disease type II MedDRA version: 20.1;Level: PT;Classification code 10053185;Term: G ... | Product Name: Avalglucosidase Alfa Product Code: GZ402666 INN or Proposed INN: ALGLUCOSIDASE ALFA Other descriptive name: RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN Product Name: Avalglucosidase Alfa Product Code: GZ402666 INN or Proposed INN: ALGLUCOSIDASE ALFA Other ... | Sanofi-Aventis Recherche & Développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | France;United States;Taiwan;Belgium;Germany;Netherlands;United Kingdom;Italy;China | ||
| 2 | NCT05017402 (ClinicalTrials.gov) | September 1, 2021 | 5/8/2021 | Higher Dose of Alglucosidase Alpha for Pompe Disease | Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study | Glycogen Storage Disease Type II | Drug: Alglucosidase Alfa | Taipei Veterans General Hospital, Taiwan | NULL | Not yet recruiting | N/A | 60 Years | All | 36 | NULL | |
| 3 | NCT04848779 (ClinicalTrials.gov) | June 10, 2021 | 29/3/2021 | A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD) A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase AlfaTreatment in Patien ... | A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients =6 Months of Age With Infantile-onset Pompe Disease (IOPD) A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase AlfaTreatment in Pa ... | Glycogen Storage Disease Type II | Drug: Alglucosidase alfa GZ419829 | Sanofi | NULL | Recruiting | 0 Days | 6 Months | All | 16 | Belgium;France;Germany;Italy;Netherlands;Taiwan;United Kingdom | |
| 4 | NCT04676373 (ClinicalTrials.gov) | March 10, 2021 | 15/12/2020 | Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Algluco ... | A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese ... | Pompe's Disease | Drug: ALGLUCOSIDASE ALFA | Genzyme, a Sanofi Company | NULL | Recruiting | 3 Years | N/A | All | 40 | Phase 4 | China |
| 5 | 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 study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with ... | 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 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-a ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | 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 Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 6 | JPRN-JapicCTI-194887 | 17/9/2019 | 26/07/2019 | ATB200/AT2221phase3 double blind randomized study | 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 A phase 3 double-blind randomized study to assess the efficacy and safety of intravenous ATB200 co-a ... | Pompe disease | Intervention name : Recombinant human acid alfa-glucosidase (ATB200) INN of the intervention : Recombinant human acid alfa-glucosidase Dosage And administration of the intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after AT2221(Miglustat) administration every 2 weeks Intervention name : Miglustat (AT2221) INN of the intervention : Miglustat Dosage And administration of the intervention : Subjects weighing >= 50 kg, 260mg (4 oral capsules of AT2221) 1hour prior to ATB200 infusion every 2 weeks. Subjects weighing >= 40 kg to < 50 kg, 195 mg (3 oral capsules) 1 hour prior to ATB200 infusion every 2 weeks. Control intervention name : Alglucosidase Alfa (Genetical Recombination) INN of the control intervention : Alglucosidase Alfa (Genetical Recombination) Dosage And administration of the control intervention : 20 mg/kg IV infusion over a 4-hour duration 1 hour after placebo administration every 2 weeks Control intervention name : Plecebo INN of the control intervention : - Dosage And administration of the control intervention : - Intervention name : Recombinant human acid alfa-glucosidase(ATB200) INN of the intervention : Recombi ... | Amicus Therapeutics, Inc./CMIC Co., Ltd. | NULL | complete | 18 | BOTH | 110 | Phase 3 | Japan, Asia except Japan, North America, South America, Europe, Oceania | |
| 7 | 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 study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with ... | 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 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-a ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | 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 Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 8 | 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 study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with ... | 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 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-a ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | 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 Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: Cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 9 | 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 study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with ... | 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 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-a ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | 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 Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Name: AT222 ... | 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 United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Bosnia and Her ... | ||
| 10 | EUCTR2018-000755-40-IT (EUCTR) | 17/05/2019 | 25/01/2021 | A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease compared with Alglucosidase Alfa/Placebo. A study investigating the efficacy and safety of intravenous (IV) ATB200 when Co-administrated with ... | 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 - AMICUS ATB200-03 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-a ... | 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] Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 20.0;Level: LLT;Classification c ... | Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: non applicabile Product Name: AT2221 Product Code: [AT2221] INN or Proposed INN: MIGLUSTAT Trade Name: Myozyme Product Name: Myozyme Product Code: [Myozyme] INN or Proposed INN: ALGLUCOSIDASI ALFA UMANA RICOMBINANTE Product Name: ATB200 Product Code: [ATB200] INN or Proposed INN: non applicabile Product Name: AT2221 Pr ... | AMICUS THERAPEUTICS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Korea, Democratic People's Republic of;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Australia;Denmark;Netherlands;Korea, Republi ... |