N Acetyl L Leucine ( DrugBank: Leucine )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病3

19. ライソゾーム病


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
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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size
PhaseCountries
1EUCTR2021-005356-10-SK
(EUCTR)
15/03/202228/12/2021A pivotal study of N-Acetyl-L-Leucine on Niemann-Pick disease type CEffects 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 LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
53Phase 3United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerland
2EUCTR2018-004331-71-ES
(EUCTR)
10/06/201922/01/2019A 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 LtdNULLAuthorised-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): noUnited States;Slovakia;Spain;Germany;United Kingdom
3EUCTR2018-004406-25-ES
(EUCTR)
10/06/201912/04/2019A 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 LtdNULLAuthorised-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): noUnited States;Spain;Germany;United Kingdom