19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | EUCTR2019-004909-27-NL (EUCTR) | 11/05/2021 | 03/09/2020 | STUDY TO EVALUATE THE SAFETY AND ESTABLISH A SAFE DOSE OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME | A PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME | Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II]) MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DNL310 Product Code: DNL310 Drug Substance INN or Proposed INN: Not applicable Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR | Denali Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 16 | Phase 1;Phase 2 | United States;Germany;Netherlands;United Kingdom;Italy | ||
| 2 | EUCTR2019-004909-27-GB (EUCTR) | 22/07/2020 | 01/05/2020 | STUDY TO EVALUATE THE SAFETY AND ESTABLISH A SAFE DOSE OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME | A PHASE 1/2, MULTICENTER, OPEN-LABEL STUDY TO DETERMINE THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DNL310 IN PEDIATRIC SUBJECTS WITH HUNTER SYNDROME | Hunter Syndrome (Mucopolysaccharidosis Type II [MPS II]) MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: DNL310 Product Code: DNL310 Drug Substance INN or Proposed INN: Not applicable Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR | Denali Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 16 | Phase 1;Phase 2 | United States;Netherlands;Germany;Italy;United Kingdom | ||
| 3 | NCT04251026 (ClinicalTrials.gov) | July 16, 2020 | 28/1/2020 | A Study of DNL310 in Pediatric Participants With Hunter Syndrome | A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants With Hunter Syndrome | Mucopolysaccharidosis II | Drug: DNL310 | Denali Therapeutics Inc. | NULL | Recruiting | 2 Years | 18 Years | Male | 30 | Phase 1/Phase 2 | United States;Netherlands |