19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
Showing 1 to 10 of 32 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 | NCT05058391 (ClinicalTrials.gov) | April 30, 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 | Not yet recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 2 | NCT04573023 (ClinicalTrials.gov) | February 14, 2022 | 17/9/2020 | A Phase ? Study of JR-141 in Patients With Mucopolysaccharidosis II | A Phase ? 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 | 50 | Phase 3 | United States |
| 3 | 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 | ||
| 4 | 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 |
| 5 | 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 |
| 6 | 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 ... | ||
| 7 | 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 |
| 8 | EUCTR2014-004143-13-ES (EUCTR) | 09/04/2015 | 09/02/2015 | An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and ... | 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 and cognitive impairment MedDRA version: 18.0;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] Hunter syndrome and cognitive impairment MedDRA version: 18.0;Level: PT;Classification code 10056889 ... | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive ... | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 42 | United States;Mexico;Canada;Argentina;Spain;Colombia;United Kingdom | |||
| 9 | NCT02262338 (ClinicalTrials.gov) | April 2015 | 2/10/2014 | Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Sy ... | A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome) A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduron ... | Mucopolysaccharidosis II | Drug: AGT-182 | ArmaGen, Inc | NULL | Completed | 18 Years | N/A | Male | 6 | Phase 1 | United States;Germany;Philippines;Netherlands |
| 10 | EUCTR2014-004143-13-GB (EUCTR) | 19/03/2015 | 30/01/2015 | An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and ... | 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 and cognitive impairment MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10056917;Term: Hunter's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] Hunter syndrome and cognitive impairment MedDRA version: 20.1;Level: PT;Classification code 10056889 ... | Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive name: idursulfase-IT Product Name: Idursulfase-IT Product Code: HGT-2310 INN or Proposed INN: IDURSULFASE Other descriptive ... | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 54 | Phase 2;Phase 3 | United States;France;Mexico;Canada;Argentina;Spain;Australia;Colombia;United Kingdom |