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 26 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 | NCT02912793 (ClinicalTrials.gov) | March 20, 2017 | 19/8/2016 | Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients | A Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-ß-CD) in Patients With Niemann-Pick Disease Type C NPC-1 and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes A Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-ß-CD) in Patients With Nie ... | Niemann-Pick Disease, Type C1 | Drug: Hydroxypropyl-beta-cyclodextrin | Cyclo Therapeutics, Inc. | NULL | Completed | 2 Years | N/A | All | 12 | Phase 1/Phase 2 | Israel;Sweden;United Kingdom |
| 2 | EUCTR2015-005761-23-SE (EUCTR) | 19/01/2017 | 09/09/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Nieman ... | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-C ... | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Nieman ... | Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive name: hydroxypropyl-beta-cyclodextrin Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive na ... | Cyclo Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 11 | Phase 1;Phase 2 | United Kingdom;Italy;Sweden | ||
| 3 | EUCTR2015-005761-23-GB (EUCTR) | 26/09/2016 | 03/08/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Nieman ... | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-C ... | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Nieman ... | Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive name: hydroxypropyl-beta-cyclodextrin Product Name: Trappsol Cyclo INN or Proposed INN: hydroxypropyl-beta-cyclodextrin Other descriptive na ... | CTD Holdings, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | 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 - ... | Italy;United Kingdom;Sweden | ||
| 4 | EUCTR2014-000533-22-FI (EUCTR) | 23/06/2015 | 05/06/2015 | Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD). | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmac ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;Finland;Italy;United Kingdom | ||
| 5 | EUCTR2011-004287-30-NL (EUCTR) | 29/05/2015 | 01/04/2015 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LA ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Netherlands;Germany United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croa ... | ||
| 6 | EUCTR2011-002750-31-CZ (EUCTR) | 16/02/2015 | 22/03/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipas ... | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: ... | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other des ... | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Germany;Japan;Sweden United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Austra ... | ||
| 7 | EUCTR2011-004287-30-GB (EUCTR) | 19/01/2015 | 11/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LA ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 18.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 18.1;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Alexion Pharmaceuticals Inc | NULL | Not Recruiting | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Poland;Brazil;Croatia;Australia;Denmark;Netherlands;Germany;Japan United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Poland;Braz ... | ||
| 8 | EUCTR2014-000533-22-IT (EUCTR) | 18/09/2014 | 22/07/2014 | Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD). | A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmac ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 17.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 17.0;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;Finland;United Kingdom;Italy | ||
| 9 | EUCTR2011-004287-30-IT (EUCTR) | 18/09/2014 | 18/04/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LA ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 16.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 16.1;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Synageva BioPharma Corp. | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Poland;Croatia;Australia;Denmark;Netherlands;Germany;Japan United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Pola ... | ||
| 10 | EUCTR2011-004287-30-DE (EUCTR) | 12/08/2014 | 13/05/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LA ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.1;Level: HLT;Classification code 10024579 ... | Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha Product Name: Sebelipase Alfa Product Code: SBC-102 INN or Proposed INN: sebelipase alfa Other descript ... | Alexion Pharmaceuitcals Inc | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croatia;Australia;Denmark;Germany;Netherlands;Japan United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croa ... |