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 38 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 | 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 Study to determine effectiveness and safety of DNL310 vs Idursulfasein Pediatric Participants With N ... | 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 A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL ... | Mucopolysaccharidosis Type II [MPS II];Therapeutic area: Diseases [C] - Nervous System Diseases [C10] 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 Product Name: DNL310 Product Code: DNL310 INN or Proposed INN: DNL310 Other descriptive name: IDURONATE ... | 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 Colombia;Argentina;United States;Czechia;United Kingdom;Spain;Canada;Czech Republic;Sweden;Netherlan ... | ||
| 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 Study to Determine the Efficacy and Safety of DNL310 vs Idursulfasein Pediatric Participants With ... | 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 A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL ... | 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 | NCT05058391 (ClinicalTrials.gov) | April 21, 2022 | 24/9/2021 | A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India | A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II) A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Sa ... | Hunter Syndrome | Biological: Elaprase | Shire | NULL | Recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 4 | NCT04573023 (ClinicalTrials.gov) | February 14, 2022 | 17/9/2020 | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141;Drug: Idursulfase;Drug: JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd. | NULL | Recruiting | N/A | N/A | Male | 80 | Phase 3 | United States;Brazil;France;Germany;Spain |
| 5 | JPRN-jRCT2031220250 | 11/03/2020 | 07/08/2022 | Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II | Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II - CKK001 | Mucopolysaccharidosis type II;D016532 | [Investigative drug, dose, and administration method] - Investigative drug: Vial formulation filled with idursulfase beta for intracerebroventricular administration (Concentration 15 +/- 1.5 mg/mL, liquid volume 1.25 mL) - Dose and administration method: An implantable cerebrospinal fluid reservoir is attached to the subject's head, and idursulfase beta 30 mg is administered to the ventricles once every 4 weeks. [Investigative drug, dose, and administration method] - Investigative drug: Vial formulation filled w ... | Yanagi Komei | NULL | Complete | >= 6month old | < 15age old | Male | 6 | Phase 3 | Japan |
| 6 | EUCTR2018-000192-33-GB (EUCTR) | 17/08/2018 | 09/04/2018 | A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in patients with Mucopolysaccharidosis II (MPS II) A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in ... | A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II) A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Toler ... | Mucopolysaccharidosis type II (MPS II) MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Mucopolysaccharidosis type II (MPS II) MedDRA version: 20.1;Level: PT;Classification code 10056889;T ... | Product Name: rAAV2/6 Left ZFN Vector Product Code: SB-47171 INN or Proposed INN: Not yet assigned Other descriptive name: Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector Product Name: rAAV2/6 Right ZFN Vector Product Code: SB-47898 INN or Proposed INN: Not yet assigned Other descriptive name: Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector Product Name: rAAV 2/6 hIDS DONOR Product Code: hIDS DONOR INN or Proposed INN: Not yet assigned Other descriptive name: Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector Product Name: rAAV2/6 Left ZFN Vector Product Code: SB-47171 INN or Proposed INN: Not yet assigned Othe ... | Sangamo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 32 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ... | United States;United Kingdom | ||
| 7 | NCT02663024 (ClinicalTrials.gov) | December 2016 | 17/1/2016 | Study of Idursulfase-beta (GC1111) in Hunter Syndrome | Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Idursulfase-beta (GC1111) in Hunter Syndrome (Mucopolysaccharidosis II) Patients Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokin ... | Mucopolysaccharidosis II | Biological: idursulfase beta;Biological: idursulfase | Green Cross Corporation | NULL | Not yet recruiting | 5 Years | 35 Years | Male | 20 | Phase 2 | NULL |
| 8 | JPRN-JMA-IIA00350 | 29/07/2016 | 04/06/2018 | Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II | Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II | Patients at the age between 6 months and 15 years with severe type mucopolysaccharidosis type II intravenously administered isursulfase at least for 24 weeks Patients at the age between 6 months and 15 years with severe type mucopolysaccharidosis type II int ... | Intervention type:DRUG. Intervention1:BHP001, Dose form:INJECTION, Route of administration:OTHER. | Torayuki Okuyama | Haruo Shintaku | Completed | >=6 MONTHS | <15 YEARS | Male | 6 | Phase 1-2 | Japan |
| 9 | EUCTR2014-004804-31-DE (EUCTR) | 12/04/2016 | 06/08/2015 | A long-term study on changes in height and weight of children with MPS II receiving Elaprase and who started the treatment before the age of 6 years. A long-term study on changes in height and weight of children with MPS II receiving Elaprase and who ... | A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients with MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6 Years of Age A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and We ... | Hunter syndrome (Mucopolysaccharidosis II, [MPS II]);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Hunter syndrome (Mucopolysaccharidosis II, [MPS II]);Therapeutic area: Diseases [C] - Congenital, He ... | Trade Name: Elaprase INN or Proposed INN: IDURSULFASE | Shire Human Genetic Therapies, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 4 | United States;Serbia;Philippines;Saudi Arabia;Malaysia;Thailand;Costa Rica;Oman;Dominican Republic;Germany;Vietnam United States;Serbia;Philippines;Saudi Arabia;Malaysia;Thailand;Costa Rica;Oman;Dominican Republic;G ... | ||
| 10 | NCT02412787 (ClinicalTrials.gov) | October 28, 2015 | 1/4/2015 | Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 Study of Long Term Safety and Clinical Outcomes of IdursulfaseIT and Elaprase Treatment in Pediatric ... | An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of In ... | Hunter Syndrome | Drug: Idursulfase-IT;Drug: Elaprase | Shire | NULL | Active, not recruiting | N/A | 18 Years | Male | 49 | Phase 2/Phase 3 | United States;Australia;Canada;France;Mexico;Spain;United Kingdom;Argentina;Colombia |