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 180 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 | 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 A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglusta ... | 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 An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immuno ... | 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 | 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 | |
| 3 | NCT04476550 (ClinicalTrials.gov) | September 1, 2022 | 26/2/2020 | Clinical Specimen Collection From Pompe Disease Patients | Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Pompe Disease Developing a Potential Cure for Pompe Disease: Clinical Specimen Collection From Individuals With Po ... | Pompe Disease | Drug: Filgrastim | Serhat Gumrukcu, MD PhD | Frida Therapeutics LLC | Recruiting | 3 Years | 30 Years | All | 12 | United States | |
| 4 | NCT05567627 (ClinicalTrials.gov) | August 1, 2022 | 25/9/2022 | Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) ... | Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy ... | Infantile-onset Pompe Disease | Biological: Genetic: GC301 | Seventh Medical Center of PLA General Hospital | GeneCradle Therapeutics, Inc | Recruiting | N/A | 6 Months | All | 6 | N/A | China |
| 5 | NCT05164055 (ClinicalTrials.gov) | July 11, 2022 | 16/11/2021 | Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) Avalglucosidase Alfa French 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 |
| 6 | EUCTR2019-001283-30-DK (EUCTR) | 03/11/2021 | 05/10/2021 | A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease (type II glycogen storage disease) A study of safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults ... | Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intra ... | Pompe Disease (also known as 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: Body processes [G] - Metabolic Phenomena [G03] Pompe Disease(also known as glycogen storage disease type II) MedDRA version: 20.1;Level: PT;Classif ... | Product Code: SPK-3006 INN or Proposed INN: SPK-3006 | Spark Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1;Phase 2 | France;United States;Spain;Denmark;Netherlands;Germany;United Kingdom;Italy | ||
| 7 | 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 | ||
| 8 | 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 |
| 9 | 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 | |
| 10 | NCT04532047 (ClinicalTrials.gov) | July 1, 2021 | 19/8/2020 | In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases | In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs ... | MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease Infantile-Onset;Wolman Disease MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe DiseaseInf ... | Drug: Aldurazyme (laronidase) | University of California, San Francisco | Duke University | Recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |