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 34 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 | NCT05734521 (ClinicalTrials.gov) | October 26, 2022 | 3/2/2023 | Avalglucosidase Alfa Pregnancy Study | A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviaz ... | Pompe Disease;Pregnancy | Biological: avalglucosidase alfa-NGPT (GZ402666) IV;Biological: avalglucosidase alfa-NGPT (GZ402666) | Sanofi | NULL | Recruiting | N/A | N/A | Female | 100 | United States | |
| 2 | NCT05164055 (ClinicalTrials.gov) | July 11, 2022 | 16/11/2021 | Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Avalglucosidase AlfaFrench Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidas ... | A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Development Studies in France A French Multicenter Phase 4 Open Label Extension Study of Long Term Safety and Efficacy in Patients ... | Glycogen Storage Disease Type II | Drug: Avalglucosidase alfa (GZ402666) | Genzyme, a Sanofi Company | NULL | Recruiting | 6 Months | N/A | All | 18 | Phase 4 | France |
| 3 | 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 | ||
| 4 | NCT04910776 (ClinicalTrials.gov) | September 1, 2021 | 25/5/2021 | Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalgluco ... | An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD) An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharm ... | Glycogen Storage Disease Type II | Drug: avalglucosidase alfa | Genzyme, a Sanofi Company | NULL | Recruiting | 0 Days | 12 Months | All | 18 | Phase 3 | United States;Belgium;China;Germany;Italy;Netherlands;Taiwan;United Kingdom |
| 5 | EUCTR2020-004686-39-IT (EUCTR) | 27/04/2021 | 07/06/2021 | Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) WithAvalglucosidase Alfa(Baby-COMET) 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 inTreatment-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: avalglucosidasi alfa Other descriptive name: RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN Product Name: Avalglucosidase Alfa Product Code: [GZ402666] INN or Proposed INN: avalglucosidasi alfa O ... | SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 3 | France;United States;Taiwan;Belgium;Netherlands;Germany;United Kingdom;China;Italy | ||
| 6 | EUCTR2016-000942-77-HU (EUCTR) | 29/01/2019 | 04/12/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAAand Alglucosidase Alf ... | 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 A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and sa ... | 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] Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification cod ... | 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 Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase co ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Poland;Brazil;Belgium;Bulgaria;Germany;Japan;Sweden Portugal;United States;Taiwan;Spain;Russian Federation;Colombia;Switzerland;Italy;France;Australia;D ... | ||
| 7 | EUCTR2016-000942-77-BG (EUCTR) | 23/10/2018 | 24/07/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAAand Alglucosidase Alf ... | 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 A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and sa ... | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification cod ... | 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 Product Name: neoGAA Product Code: GZ402666 Other descriptive name: Recombinant human a-glucosidase co ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 96 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Australia;D ... | ||
| 8 | EUCTR2016-000942-77-PT (EUCTR) | 24/07/2018 | 12/04/2018 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfaand Alg ... | 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 A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Sa ... | Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification code 10036143;Term: Pompe's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 24.0;Level: LLT;Classification cod ... | Product Name: neoGAA or avalglucosidase alfa Product Code: GZ402666 INN or Proposed INN: neoGAA Other descriptive name: Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA) Trade Name: MYOZYME® Product Name: Myozyme® INN or Proposed INN: ALGLUCOSIDASE ALFA Product Name: neoGAAor avalglucosidase alfa Product Code: GZ402666 INN or Proposed INN: neoGAA Other de ... | Genzyme Corporation | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Japan;Sweden United States;Portugal;Taiwan;Spain;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Aus ... | ||
| 9 | NCT03019406 (ClinicalTrials.gov) | October 12, 2017 | 20/12/2016 | 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 A Study to Assess Safety and Efficacy of Avalglucosidase AlfaAdministered Every Other Week in Pediat ... | 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 An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Ef ... | Glycogen Storage Disease Type II-Pompe's Disease | Drug: avalglucosidase alfa GZ402666;Drug: alglucosidase alfa GZ419829 | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 6 Months | 17 Years | All | 22 | Phase 2 | United States;France;Japan;Taiwan;United Kingdom |
| 10 | 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 A Study to Assess Safety and Efficacy of Avalglucosidase AlfaAdministered Every Other Week in Pediat ... | 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 An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Ef ... | 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] Pompe disease (acid alpha-glucosidase deficiency) MedDRA version: 20.0;Level: LLT;Classification cod ... | 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 Product Name: Avalglucosidase Alfa(NeoGAA) Product Code: GZ402666 INN or Proposed INN: Avalglucosidase ... | 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 |