19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 10 of 899 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 |
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1 | NCT05710367 (ClinicalTrials.gov) | August 2023 | 10/1/2023 | Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients | Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients; A Prospective, Randomized, Double-Blind, Placebo- Controlled Study. Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry DiseasePatients; A ... | Fabry Disease | Drug: Dapagliflozin 10mg Tab;Drug: Placebo | Albina Nowak, MD | NULL | Not yet recruiting | 18 Years | 70 Years | All | 46 | Phase 2 | NULL |
2 | NCT05698901 (ClinicalTrials.gov) | March 31, 2023 | 16/1/2023 | Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease | Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease | Fabry Disease | Drug: Agalsidase beta | Mackay Memorial Hospital | NULL | Not yet recruiting | 18 Years | N/A | All | 150 | NULL | |
3 | NCT05238324 (ClinicalTrials.gov) | March 2023 | 7/12/2021 | Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II | A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Treated Adults With Mucopolysaccharidosis Type II (MPS II) (juMPStart Trial) A Phase 1 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-203 in ERT-Tre ... | Mucopolysaccharidosis II | Biological: Genetic HMI-203 | Homology Medicines, Inc | NULL | Recruiting | 18 Years | 45 Years | Male | 9 | Phase 1 | United States;Canada |
4 | NCT05710692 (ClinicalTrials.gov) | March 2023 | 13/12/2022 | Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Diseas ... | A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE) A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Effic ... | Fabry Disease | Drug: PRX-102 1 mg/kg every 2 weeks;Drug: PRX-102 2 mg/kg every 4 weeks | Chiesi Farmaceutici S.p.A. | NULL | Not yet recruiting | 18 Years | 60 Years | All | 18 | Phase 2/Phase 3 | NULL |
5 | NCT05594992 (ClinicalTrials.gov) | February 22, 2023 | 6/10/2022 | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male Subjects An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis ... | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male Subjects An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis ... | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Enrolling by invitation | N/A | N/A | Male | 80 | Phase 3 | United States |
6 | NCT05739643 (ClinicalTrials.gov) | February 3, 2023 | 13/2/2023 | Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated in the Past With HSCT Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe DiseaseTreated in the Past With ... | A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previously Received Hematopoietic Stem Cell Transplantation (REKLAIM) A Phase 1b Clinical Study of Intravenous AAVrh10 Vector Expressing GALC in Krabbe Subjects Who Previ ... | Krabbe Disease | Biological: FBX-101 | Forge Biologics, Inc | NULL | Recruiting | N/A | N/A | All | 12 | Phase 1/Phase 2 | United States |
7 | NCT05665166 (ClinicalTrials.gov) | February 2023 | 7/12/2022 | Autologous Ex-vivo Gene Modified HSCT in MPSII | A Phase I/II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11B Lentiviral Vector Encoding Human IDS Tagged With ApoEII in Patients With Neuronopathic Mucopolysaccharidosis Type II (nMPS II, Hunters Syndrome) A Phase I/II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11B Lent ... | Mucopolysaccharidosis II | Genetic: Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII Genetic: Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEI ... | University of Manchester | Manchester University NHS Foundation Trust;AVROBIO;CTI Clinical Trial and Consulting Services;Great Ormond Street Hospital for Children NHS Foundation Trust Manchester University NHS Foundation Trust;AVROBIO;CTI Clinical Trial and Consulting Services;Great ... | Not yet recruiting | 3 Months | 12 Months | Male | 5 | Phase 1/Phase 2 | NULL |
8 | NCT05682144 (ClinicalTrials.gov) | January 15, 2023 | 12/12/2022 | ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I | A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With ... | Mucopolysaccharidosis IH/S;Mucopolysaccharidosis IS | Biological: Autologous Plasmablasts (B cells) | Immusoft Corporation | NULL | Not yet recruiting | 18 Years | N/A | All | 2 | Phase 1 | United States |
9 | NCT05529992 (ClinicalTrials.gov) | January 3, 2023 | 2/9/2022 | A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher ... | A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucer ... | Gaucher Disease | Drug: Velaglucerase Alfa | Takeda | NULL | Recruiting | 2 Years | N/A | All | 20 | Phase 3 | China |
10 | EUCTR2021-002550-82-NL (EUCTR) | 22/12/2022 | 30/07/2022 | Effectiveness of ambroxol for treating children and adults with Gaucher disease type 3 | Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series - ATTACK-GD3 | Gaucher disease type 3 MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metabolic and nutritional disorders congenital;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Gaucher diseasetype 3 MedDRA version: 20.0;Level: HLGT;Classification code 10027424;Term: Metabolic ... | Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL Hydrochloride Other descriptive name: Mucoangin Product Name: Ambroxol hydrochloride INN or Proposed INN: AMBROXOL HYDROCHLORIDE Other descriptive name: AMBROXOL HYDROCHLORIDE PH. EUR. Product Name: Ambroxolhydrochloride INN or Proposed INN: AMBROXOLHydrochloride Other descriptive name: ... | Amsterdam UMC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 4 | Phase 3 | Netherlands |