Cipaglucosidase Alfa ( DrugBank: Cipaglucosidase alfa )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 256 | 筋型糖原病 | 19 |
256. 筋型糖原病
臨床試験数 : 180 / 薬物数 : 133 - (DrugBank : 29) / 標的遺伝子数 : 25 - 標的パスウェイ数 : 105
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04808505 (ClinicalTrials.gov) | April 2023 | 10/3/2021 | A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years | Glycogen Storage Disease Type II Infantile Onset | Biological: Cipaglucosidase alfa;Drug: Miglustat | Amicus Therapeutics | NULL | Recruiting | N/A | 17 Years | All | 36 | Phase 3 | United States |
| 2 | EUCTR2019-000954-67-AT (EUCTR) | 19/06/2020 | 13/03/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;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 Other descriptive name: AT2221 65 mg Formulated Capsules | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | Australia;Netherlands;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark | ||
| 3 | EUCTR2019-000954-67-SI (EUCTR) | 03/06/2020 | 10/03/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | 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 | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 3 | United States;Taiwan;Slovenia;Greece;Spain;Austria;United Kingdom;Italy;France;Hungary;Canada;Argentina;Poland;Belgium;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Bosnia and Herzegovina;Korea, Republic of | ||
| 4 | EUCTR2019-000954-67-DE (EUCTR) | 17/04/2020 | 23/12/2019 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;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 | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | Germany;Japan;Sweden;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;United States;Taiwan;Slovakia;Greece;Spain;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of | ||
| 5 | EUCTR2019-000954-67-GB (EUCTR) | 08/04/2020 | 13/12/2019 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | 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 | 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 | ||
| 6 | EUCTR2019-000954-67-GR (EUCTR) | 02/04/2020 | 13/02/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;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 | 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;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 7 | EUCTR2019-000954-67-DK (EUCTR) | 16/03/2020 | 20/01/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | Adult Subjects With Late Onset Pompe Disease (LOPD) MedDRA version: 24.0;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 | 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;Korea, Republic of;Bosnia and Herzegovina;Slovenia;Austria;United Kingdom;Hungary;Canada;Argentina;Belgium;Poland;Romania;Bulgaria;Germany;Japan;Sweden | ||
| 8 | EUCTR2019-000954-67-FR (EUCTR) | 14/03/2020 | 20/01/2020 | A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease | A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | 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 | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | 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 | NCT03911505 (ClinicalTrials.gov) | February 13, 2020 | 9/4/2019 | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease | Pompe Disease (Late-onset) | Biological: Cipaglucosidase Alfa;Drug: Miglustat | Amicus Therapeutics | NULL | Recruiting | 0 Years | 17 Years | All | 22 | Phase 3 | United States;Australia;Canada;Japan;Taiwan |
| 10 | 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 | 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 | ||
| 11 | 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 | 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 | ||
| 12 | 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: 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 | 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 | ||
| 13 | 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 | 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 | ||
| 14 | 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 | 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 | ||
| 15 | 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 | 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 | ||
| 16 | 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 | Not Recruiting | 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 | ||
| 17 | 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 | 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 | ||
| 18 | EUCTR2015-004798-34-DE (EUCTR) | 30/08/2016 | 31/03/2016 | This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221. | AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE | Pompe Disease - acid maltase deficiency or glycogen storage disease type II. MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Code: AT2221 INN or Proposed INN: MIGLUSTAT | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Netherlands;Germany;United Kingdom | ||
| 19 | EUCTR2015-004798-34-GB (EUCTR) | 01/08/2016 | 04/01/2016 | This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the safety, tolerability, PK, PD and efficacy of intravenous (IV) ATB200 when co-administered with oral AT2221. | AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH POMPE DISEASE | Pompe Disease - acid maltase deficiency or glycogen storage disease type II. MedDRA version: 20.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ATB200 Product Code: ATB200 INN or Proposed INN: cipaglucosidase alfa Product Code: AT2221 INN or Proposed INN: MIGLUSTAT | Amicus Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Australia;Netherlands;Germany;United Kingdom |