ERT
( DrugBank: - / KEGG DRUG: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 35 |
19. ライソゾーム病
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 35 trials
| 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 | NCT05238324 (ClinicalTrials.gov) | March 202320230300 | 7/12/202120211207 | 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 |
| 2 | NCT05222906 (ClinicalTrials.gov) | April 18, 202220220418 | 20/1/202220220120 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher ... | A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT) A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Stu ... | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 40 | Phase 3 | United States;Argentina;Canada;China;France;Germany;Hungary;Japan |
| 3 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/202220220323 | 05/01/202220220105 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher ... | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator stu ... | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher ... | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Veng ... | Sanofi-aventis recherche & développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2 | Phase 3 | Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentin ... | ||
| 4 | NCT05152914 (ClinicalTrials.gov) | November 1, 202120211101 | 29/11/202120211129 | Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 | Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neur ... | Neuronal Ceroid Lipofuscinosis Type 2 | Drug: Cerliponase Alfa | David L Rogers, MD | NULL | Enrolling by invitation | 24 Months | 72 Months | All | 5 | Phase 1/Phase 2 | United States |
| 5 | NCT04532047 (ClinicalTrials.gov) | July 1, 202120210701 | 19/8/202020200819 | 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 Disease In ... | Drug: Aldurazyme (laronidase) | University of California, San Francisco | Duke University | Recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 6 | NCT04353466 (ClinicalTrials.gov) | January 1, 201720170101 | 13/4/202020200413 | Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs | An Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement in Patients With Gaucher Disease Currently Treated With Other ERTs An Open-label, Investigator Initiated Clinical Trial to Asses Impact of Elelyso on Bone Involvement ... | Gaucher Disease, Type 1 | Procedure: quantitative chemical shift imaging (QCSI);Drug: Elelyso | Shaare Zedek Medical Center | Pfizer | Completed | 18 Years | N/A | All | 30 | N/A | NULL |
| 7 | NCT02520934 (ClinicalTrials.gov) | July 201520150700 | 6/8/201520150806 | Miglustat on Gaucher Disease Type IIIB | Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease T ... | Gaucher Disease | Drug: Miglustat;Drug: ERT | National Taiwan University Hospital | Actelion | Active, not recruiting | 6 Years | N/A | All | 19 | N/A | Taiwan |
| 8 | NCT02969200 (ClinicalTrials.gov) | April 201520150400 | 2/11/201620161102 | Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance | Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard GFR 51Cr-EDTA Clearance Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard ... | Fabry Disease | Drug: Enzyme replacement therapy | Ulla Feldt-Rasmussen | NULL | Completed | N/A | N/A | All | 52 | Denmark | |
| 9 | NCT02930655 (ClinicalTrials.gov) | February 1, 201520150201 | 10/10/201620161010 | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease | A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement Therapy A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safe ... | Fabry Disease | Drug: Lucerastat;Drug: Enzyme replacement therapy (ERT) | Idorsia Pharmaceuticals Ltd. | NULL | Completed | 18 Years | N/A | All | 14 | Phase 1 | Germany |
| 10 | NCT02228460 (ClinicalTrials.gov) | November 201420141100 | 27/8/201420140827 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in ... | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy ... | Fabry Disease | Drug: GZ/SAR402671 | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 49 Years | Male | 11 | Phase 2 | United States;France;Poland;Russian Federation;United Kingdom;Czech Republic;Czechia |