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
| 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 | EUCTR2021-005200-35-CZ (EUCTR) | 05/08/2022 | 24/05/2022 | Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR Trade Name: Idursulfase (Elaprase) Product Name: idursulfase (ELAPRASE) INN or Proposed INN: Idursulfase Other descriptive name: Purified form of the lysosomal enzyme iduronate-2-sulfatase | Denali Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 2;Phase 3 | Colombia;Argentina;United States;Czechia;United Kingdom;Spain;Canada;Czech Republic;Sweden;Netherlands;Turkey;Belgium;Brazil;Mexico;Italy;Australia;France;Germany | ||
| 2 | NCT05371613 (ClinicalTrials.gov) | July 21, 2022 | 9/5/2022 | A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II | Mucopolysaccharidosis II | Drug: DNL310;Drug: Idursulfase | Denali Therapeutics Inc. | NULL | Recruiting | 2 Years | 16 Years | All | 54 | Phase 2/Phase 3 | United States;Belgium;Canada;Czechia;France;Germany;Netherlands;Spain;Sweden;United Kingdom |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | N/A | 18 Years | Male | 45 | Phase 1/Phase 2 | United States;Netherlands |
| 6 | EUCTR2021-005200-35-FR (EUCTR) | 20/05/2022 | Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR Trade Name: Idursulfase (Elaprase) Product Name: idursulfase (ELAPRASE) INN or Proposed INN: Idursulfase Other descriptive name: Purified form of the lysosomal enzyme iduronate-2-sulfatase | Denali Therapeutics Inc. | NULL | NA | Female: yes Male: yes | 54 | Phase 2;Phase 3 | United States;Czechia;Spain;Turkey;Colombia;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Argentina;Belgium;Brazil;Australia;Netherlands;Germany;Sweden | |||
| 7 | EUCTR2021-005200-35-DE (EUCTR) | 27/04/2022 | Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR Trade Name: Idursulfase (Elaprase) Product Name: idursulfase (ELAPRASE) INN or Proposed INN: Idursulfase Other descriptive name: Purified form of the lysosomal enzyme iduronate-2-sulfatase | Denali Therapeutics Inc. | NULL | NA | Female: yes Male: yes | 54 | Phase 2;Phase 3 | United States;Czechia;Spain;Turkey;Colombia;United Kingdom;Italy;France;Czech Republic;Mexico;Canada;Argentina;Brazil;Belgium;Australia;Netherlands;Germany;Sweden | |||
| 8 | EUCTR2021-005200-35-NL (EUCTR) | 11/05/2022 | Study to determine effectiveness and safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Hunter Syndrome | A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR Trade Name: Idursulfase (Elaprase) Product Name: idursulfase (ELAPRASE) INN or Proposed INN: Idursulfase Other descriptive name: Purified form of the lysosomal enzyme iduronate-2-sulfatase | Denali Therapeutics Inc. | NULL | NA | Female: yes Male: yes | 54 | Phase 2;Phase 3 | United States;Czechia;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Belgium;Brazil;Australia;Netherlands;Germany;Sweden |