DDrare: Database of Drug Development for Rare Diseases

N-acetylcysteine (nac) (DrugBank: Acetylcysteine, N-acetylcysteine)

11 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
6	パーキンソン病	1
13	多発性硬化症／視神経脊髄炎	1
19	ライソゾーム病	0
34	神経線維腫症	1
84	サルコイドーシス	0
85	特発性間質性肺炎	1
88	慢性血栓塞栓性肺高血圧症	0
90	網膜色素変性症	2
254	ポルフィリン症	0
296	胆道閉鎖症	0
310	先天異常症候群	1

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 2,123 trial found

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	NCT03146130 (ClinicalTrials.gov)	July 5, 2018	3/5/2017	Study of the Efficacy of N-acetylcysteine (NAC) on Impulse Control Disorders	Study of the Efficacy of N-acetylcysteine (NAC) on Impulse Control Disorders (TCI) Induced by Dopaminergic Treatments in Parkinson's Disease	Impulse Control Disorder;Parkinson	Biological: Variation of behaviors of Parkinson's disease	Centre Hospitalier Universitaire, Amiens	NULL	Unknown status	18 Years	N/A	All	70	Phase 3	France

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
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.

1 / 3,050 trial found

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	NCT00203099 (ClinicalTrials.gov)	December 2004	13/9/2005	Safety and Efficacy Study of Copaxone Administered in Combination With N-Acetylcysteine	A Pilot, Multicenter, Open-label, One-group Study to Explore the Efficacy, Tolerability and Safety of the Combination of Glatiramer Acetate (GA) and N-Acetylcysteine (NAC) in Subjects With Relapsing Remitting Multiple Sclerosis (RR-MS)	Relapse Remitting Multiple Sclerosis	Drug: Glatiramer Acetate, N-Acetylcysteine	Teva Pharmaceutical Industries	NULL	Completed	18 Years	50 Years	Both	18	Phase 2	Canada

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 784 trial found

34. 神経線維腫症　［臨床試験数：120，薬物数：182（DrugBank：72），標的遺伝子数：84，標的パスウェイ数：194］

Searched query = "Neurofibromatosis", "NF1", "von Recklinghausen disease", "NF2"
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.

1 / 120 trial found

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	NCT04481035 (ClinicalTrials.gov)	January 15, 2019	14/4/2020	Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1	Antioxidant Therapy With N-acetylcysteine for Learning and Motor Behavior in Children With Neurofibromatosis Type 1	Neurofibromatosis 1	Drug: N-acetylcysteine (NAC);Other: Placebo	Children's Hospital Medical Center, Cincinnati	NULL	Active, not recruiting	8 Years	16 Years	All	5	Phase 2	United States

84. サルコイドーシス　［臨床試験数：143，薬物数：221（DrugBank：79），標的遺伝子数：82，標的パスウェイ数：165］

Searched query = "Sarcoidosis"
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 / 143 trial found

85. 特発性間質性肺炎　［臨床試験数：514，薬物数：377（DrugBank：108），標的遺伝子数：97，標的パスウェイ数：204］

Searched query = "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP"
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.

1 / 514 trial found

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	NCT00639496 (ClinicalTrials.gov)	March 2000	13/3/2008	Study of the Effects of High-dose N-acetylcysteine (NAC) in Idiopathic Pulmonary Fibrosis (IPF)	Idiopathic Pulmonary Fibrosis International Group Exploring NAC I Annual Study of the Effects of High-dose N-acetylcysteine (NAC) in Idiopathic Pulmonary Fibrosis (IPF)	Pulmonary Fibrosis	Drug: n-acetylcysteine;Drug: placebo	Zambon SpA	NULL	Completed	18 Years	75 Years	Both	184	Phase 3	Belgium;France;Germany;Italy;Netherlands;Spain;United Kingdom

88. 慢性血栓塞栓性肺高血圧症　［臨床試験数：145，薬物数：112（DrugBank：22），標的遺伝子数：13，標的パスウェイ数：52］

Searched query = "Chronic thromboembolic pulmonary hypertension", "CTEPH", "Idiopathic chronic pulmonary thromboembolism"
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 / 145 trial found

90. 網膜色素変性症　［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］

Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
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.

2 / 103 trials found

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	NCT03999021 (ClinicalTrials.gov)	June 24, 2019	24/6/2019	FIGHT-RP 1 Extension Study	A Phase 1 Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis Pigmentosa	Retinitis Pigmentosa	Drug: NAC effervescent tablets	Johns Hopkins University	NULL	Active, not recruiting	18 Years	N/A	All	30	Phase 1	United States
2	NCT03063021 (ClinicalTrials.gov)	February 15, 2017	16/2/2017	The FIGHT-RP1 Study	A Phase 1 Open Label Dose Ranging Study to Assess the Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis Pigmentosa (FIGHT-RP1 Study)	Retinitis Pigmentosa	Drug: N-Acetyl Cysteine (NAC)	Johns Hopkins University	NULL	Completed	18 Years	N/A	All	30	Phase 1	United States

254. ポルフィリン症　［臨床試験数：59，薬物数：52（DrugBank：17），標的遺伝子数：17，標的パスウェイ数：33］

Searched query = "Porphyria", "CEP", "Erythropoietic protoporphyria", "EPP", "X-linked dominant protoporphyria", "XLDP", "PCT", "HEP", "AIP", "ADP", "VP", "Hereditary coproporphyria", "HCP"
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 / 59 trial found

296. 胆道閉鎖症　［臨床試験数：53，薬物数：56（DrugBank：31），標的遺伝子数：43，標的パスウェイ数：50］

Searched query = "Biliary atresia"
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 / 53 trial found

310. 先天異常症候群　［臨床試験数：10，薬物数：18（DrugBank：10），標的遺伝子数：3，標的パスウェイ数：7］

Searched query = "Congenital anomalies syndrome", "Trisomy 1q", "9q34 deletion syndrome", "Cornelia de Lange syndrome", "CdLS", "Smith-Lemli-Opitz syndrome", "SLO syndrome"
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.

1 / 10 trial found

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	NCT04381897 (ClinicalTrials.gov)	December 1, 2020	6/5/2020	Use of N-Acetylcysteine in the Treatment of Repetitive and Self-Injurious Behaviors in Cornelia de Lange Syndrome	Use of N-Acetylcysteine (NAC) in the Treatment of Repetitive Behaviors (RB) and Self-Injurious Behaviors (SIB) in Cornelia de Lange Syndrome: A Randomized Double-Blind Placebo-Controlled Pilot Study	Cornelia de Lange Syndrome	Drug: N-acetyl cysteine;Other: Placebo	Johns Hopkins University	Cornelia de Lange Syndrome Foundation	Not yet recruiting	13 Years	35 Years	All	10	Phase 2	United States

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