19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | EUCTR2021-005356-10-SK (EUCTR) | 15/03/2022 | 28/12/2021 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Australia;Netherlands;Germany;United Kingdom;Switzerland | ||
| 2 | EUCTR2021-005356-10-DE (EUCTR) | 04/03/2022 | 29/12/2021 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study | Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Switzerland | ||
| 3 | EUCTR2018-004331-71-ES (EUCTR) | 10/06/2019 | 22/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease. | Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N Acetyl L Leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Slovakia;Spain;Germany;United Kingdom | ||
| 4 | EUCTR2018-004406-25-ES (EUCTR) | 10/06/2019 | 12/04/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). | Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study. | GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Spain;Germany;United Kingdom | ||
| 5 | EUCTR2018-004331-71-SK (EUCTR) | 04/06/2019 | 30/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease. | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational,multicenter, open-label, rater-blinded Phase II study. | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders 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] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Slovakia;Spain;Germany;United Kingdom | ||
| 6 | EUCTR2018-004406-25-DE (EUCTR) | 09/05/2019 | 23/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). | Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multicenter, open-label, rater-blinded Phase II study. | GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Spain;Germany;United Kingdom | ||
| 7 | EUCTR2018-004331-71-DE (EUCTR) | 08/05/2019 | 02/01/2019 | A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease. | Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 2 | United States;Slovakia;Spain;Germany;United Kingdom | ||
| 8 | EUCTR2021-005356-10-NL (EUCTR) | 18/01/2022 | A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type C | Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study | Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other descriptive name: 2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID | IntraBio Ltd | NULL | NA | Female: yes Male: yes | 53 | Phase 3 | United States;Czechia;Slovakia;Spain;Australia;Germany;Netherlands;United Kingdom;Switzerland |