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N-acetyl-l-leucine    (DrugBank: Leucine, L-Leucine)

10 diseases
告示番号疾患名（ページ内リンク）臨床試験数
5進行性核上性麻痺0
6パーキンソン病0
7大脳皮質基底核変性症0
19ライソゾーム病9
96クローン病0
97潰瘍性大腸炎0
127前頭側頭葉変性症0
283後天性赤芽球癆0
284ダイアモンド・ブラックファン貧血0
299嚢胞性線維症0

5. 進行性核上性麻痺　［臨床試験数：82，薬物数：107（DrugBank：36），標的遺伝子数：60，標的パスウェイ数：90］
Searched query = "Progressive supranuclear palsy", "PSP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 82 trial found

6. パーキンソン病　［臨床試験数：2,123，薬物数：2,046（DrugBank：324），標的遺伝子数：183，標的パスウェイ数：198］
Searched query = "Parkinson disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 2,123 trial found

7. 大脳皮質基底核変性症　［臨床試験数：16，薬物数：33（DrugBank：11），標的遺伝子数：6，標的パスウェイ数：13］
Searched query = "Corticobasal degeneration", "Corticobasal syndrome", "CBD"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 16 trial found

19. ライソゾーム病　［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
9 / 784 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03759639
(ClinicalTrials.gov)		September 4, 201927/11/2018N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.Niemann-Pick Disease, Type CDrug: IB1001IntraBio IncNULLActive, not recruiting6 YearsN/AAll34Phase 2United States;Germany;Slovakia;Spain;United Kingdom
2EUCTR2018-004331-71-GB
(EUCTR)		20/06/201914/01/2019To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with Niemann-Pick Type C disease (NPC).Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational,multicenter, open-label, rater-blinded Phase II study. - IB1001-201Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients withNiemann-Pick Type C disease (NPC).
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] - Nervous System Diseases [C10]		Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: SUB195712
Other descriptive name: N-Acetyl-L-Leucine
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: N-Acetyl-L-Leucine		IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		39Phase 2United States;Slovakia;Spain;Germany;United Kingdom
3EUCTR2018-004406-25-GB
(EUCTR)		20/06/201906/02/2020To demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning and quality of life in patients with GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and SandhoffDisease): A multinational, multicenter, open-label, rater-blinded Phase II study - IB1001-202 To demonstrate that N-Acetyl-L-Leucine is effective in improvingsymptoms, functioning, and quality of life in patients GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).
MedDRA version: 20.0;Level: PT;Classification code 10043147;Term: Tay-Sachs disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 21.1;Classification code 10081314;Term: Sandhoff disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: SUB195712
Other descriptive name: N-Acetyl-L-Leucine
Product Name: N-Acetyl-L-Leucine
Product Code: IB1001
INN or Proposed INN: N-Acetyl-L-leucine
Other descriptive name: N-Acetyl-L-Leucine		IntraBio LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		39Phase 2United States;Spain;Germany;United Kingdom
4EUCTR2018-004406-25-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]		IntraBio LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		39Phase 2United States;Spain;Germany;United Kingdom
5EUCTR2018-004331-71-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]		IntraBio LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		39Phase 2United States;Slovakia;Spain;Germany;United Kingdom
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6NCT03759665
(ClinicalTrials.gov)		June 7, 201927/11/2018N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)Effects of N-Acetyl-L-Leucine on GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II StudyGM2 Gangliosidosis;Tay-Sachs Disease;Sandhoff DiseaseDrug: IB1001IntraBio IncNULLRecruiting6 YearsN/AAll39Phase 2United States;Germany;Spain;United Kingdom
7EUCTR2018-004331-71-SK
(EUCTR)		04/06/201930/01/2019A safety and efficacy study of N-Acetyl-L-Leucine on Niemann-Pick type C Disease. Effects of N-Acetyl-L-Leucine on Niemann-Pick 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]		IntraBio LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes		39Phase 2United States;Slovakia;Spain;Germany;United Kingdom
8EUCTR2018-004406-25-DE
(EUCTR)		09/05/201923/01/2019A 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
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		39Phase 2United States;Spain;Germany;United Kingdom
9EUCTR2018-004331-71-DE
(EUCTR)		08/05/201902/01/2019A 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
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		39Phase 2United States;Slovakia;Spain;Germany;United Kingdom

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96. クローン病　［臨床試験数：2,209，薬物数：1,276（DrugBank：240），標的遺伝子数：166，標的パスウェイ数：210］
Searched query = "Crohn disease", "Terminal ileitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 2,209 trial found

97. 潰瘍性大腸炎　［臨床試験数：2,269，薬物数：1,331（DrugBank：241），標的遺伝子数：114，標的パスウェイ数：181］
Searched query = "Ulcerative colitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 2,269 trial found

127. 前頭側頭葉変性症　［臨床試験数：74，薬物数：83（DrugBank：28），標的遺伝子数：29，標的パスウェイ数：99］
Searched query = "Frontotemporal lobar degeneration", "Frontotemporal dementia", "Semantic dementia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 74 trial found

283. 後天性赤芽球癆　［臨床試験数：17，薬物数：36（DrugBank：19），標的遺伝子数：16，標的パスウェイ数：91］
Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 17 trial found

284. ダイアモンド・ブラックファン貧血　［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］
Searched query = "Diamond-Blackfan anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 37 trial found

299. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］
Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 1,592 trial found

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