2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID

( DrugBank: - / KEGG DRUG: - )

2 diseases

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	9

告示番号	疾患名（ページ内リンク）	臨床試験数
65	原発性免疫不全症候群	1

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

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2021-005356-10-NL (EUCTR)	08/04/2022	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 ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	53	Phase 3	United States;Czechia;Slovakia;Spain;Australia;Germany;Netherlands;United Kingdom;Switzerland
2	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 ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	53	Phase 3	United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerla ...
3	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 ...	Niemann-Pick Disease type C (NPC);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neona ...	Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	53	Phase 3	United States;Czechia;Czech Republic;Slovakia;Spain;Australia;Netherlands;Germany;United Kingdom;Swi ...
4	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, o ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N Acetyl L Leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	39	Phase 2	United States;Slovakia;Spain;Germany;United Kingdom
5	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 Dise ...	Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinationa ...	GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) MedDRA version: 20.0;Level: SOC;Classification c ...	Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N Acetyl L leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	39	Phase 2	United States;Spain;Germany;United Kingdom
6	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-labe ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	39	Phase 2	United States;Slovakia;Spain;Germany;United Kingdom
7	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 Dise ...	Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinationa ...	GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) MedDRA version: 20.0;Level: SOC;Classification c ...	Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-Leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	39	Phase 2	United States;Spain;Germany;United Kingdom
8	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, o ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	39	Phase 2	United States;Slovakia;Spain;Germany;United Kingdom
9	EUCTR2021-005356-10-CZ (EUCTR)		15/07/2022	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 ...	Niemann-Pick Disease type C (NPC) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: ...	Product Name: N-acetyl-L-leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L Leucine Other des ...	IntraBio Ltd	NULL	NA	Female: yes Male: yes	53	Phase 3	United States;Czechia;Czech Republic;Slovakia;Australia;Germany;Netherlands;United Kingdom;Switzerla ...

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65. 原発性免疫不全症候群

臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217

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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	EUCTR2018-004407-39-DE (EUCTR)	26/06/2020	05/02/2020	A safety and efficacy study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia.	Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A multinational, multicenter, open-lab ...	Ataxia-Telangiectasia (A-T) MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Conge ...	Product Name: N-Acetyl-L-Leucine Product Code: IB1001 INN or Proposed INN: N-Acetyl-L-leucine Other des ...	IntraBio Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	39	Phase 2	United States;Spain;Germany;United Kingdom

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