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 39 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 | NCT02376751 (ClinicalTrials.gov) | August 2015 | 25/2/2015 | An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency | AN EXPANDED ACCESS PROTOCOL FOR SEBELIPASE ALFA FOR PATIENTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY | Lysosomal Acid Lipase Deficiency | Drug: sebelipase alfa | Alexion Pharmaceuticals | NULL | No longer available | 8 Months | N/A | Both | N/A | NULL | |
| 2 | 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 | ||
| 3 | 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 alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain 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 ... | ||
| 4 | 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-102in patients with lysosomal acid lipase ... | 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 ... | ||
| 5 | 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 alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain 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 ... | ||
| 6 | 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 | ||
| 7 | 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 alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain 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 ... | ||
| 8 | 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 alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain 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 ... | ||
| 9 | EUCTR2011-004287-30-BE (EUCTR) | 22/07/2014 | 26/05/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 17.1;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: 17.1;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;Croatia;Australia;Denmark;Netherlands;Germany;Japan United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croa ... | ||
| 10 | EUCTR2011-004287-30-HR (EUCTR) | 07/07/2014 | 03/09/2014 | Study of sebelipase alfa in a broad population of patients with Lysosomal Acid Lipase Deficiency (LALD). Study of sebelipase alfain a broad population of patients with Lysosomal Acid Lipase Deficiency (LAL ... | A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency A Multicenter, Open-Label Study of Sebelipase Alfain Patients with Lysosomal Acid Lipase Deficiency ... | Lysosomal Acid Lipase Deficiency (LALD) MedDRA version: 19.0;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: 19.0;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 ... | Alexion Pharmaceuitcals 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;Brazil;Croatia;Australia;Denmark;Netherlands;Germany;Japan United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Mexico;Canada;Belgium;Brazil;Croa ... |