DDrare: Database of Drug Development for Rare Diseases

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

17 diseases

ID	Disease name (Link within this page)	Number of trials
6	Parkinson disease	1
13	Multiple sclerosis/Neuromyelitis optica	1
19	Lysosomal storage disease	0
20	Adrenoleukodystrophy	0
34	Neurofibromatosis	1
49	Systemic lupus erythematosus	1
51	Scleroderma	1
58	Hypertrophic cardiomyopathy	0
85	Idiopathic interstitial pneumonia	1
90	Retinitis pigmentosa	2
94	Primary sclerosing cholangitis	0
111	Congenital myopathy	0
164	Oculocutaneous albinism	0
193	Prader-Willi syndrome	0
233	Wolfram syndrome	0
298	Hereditary pancreatitis	0
299	Cystic fibrosis	1

6. Parkinson disease [ 2,123 clinical trials, 2,046 drugs, (DrugBank: 324 drugs), 183 drug target genes, 198 drug target pathways]

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. Multiple sclerosis/Neuromyelitis optica [ 3,050 clinical trials, 2,147 drugs, (DrugBank: 348 drugs), 244 drug target genes, 228 drug target pathways]

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. Lysosomal storage disease [ 784 clinical trials, 673 drugs, (DrugBank: 101 drugs), 68 drug target genes, 184 drug target pathways]

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

20. Adrenoleukodystrophy [ 49 clinical trials, 86 drugs, (DrugBank: 29 drugs), 18 drug target genes, 112 drug target pathways]

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
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 / 49 trial found

34. Neurofibromatosis [ 120 clinical trials, 182 drugs, (DrugBank: 72 drugs), 84 drug target genes, 194 drug target pathways]

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

49. Systemic lupus erythematosus [ 827 clinical trials, 638 drugs, (DrugBank: 168 drugs), 108 drug target genes, 191 drug target pathways]

Searched query = "Systemic lupus erythematosus", "SLE"
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 / 827 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	NCT00775476 (ClinicalTrials.gov)	October 2020	17/10/2008	Treatment of Systemic Lupus Erythematosus (SLE) With N-acetylcysteine	Treatment of Systemic Lupus Erythematosus (SLE) With N-acetylcysteine (NAC) (SNAC)	Systemic Lupus Erythematosus	Drug: N-acetylcysteine;Drug: Placebo	State University of New York - Upstate Medical University	NULL	Not yet recruiting	18 Years	N/A	All	290	Phase 2	United States

51. Scleroderma [ 466 clinical trials, 536 drugs, (DrugBank: 142 drugs), 110 drug target genes, 210 drug target pathways]

Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
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 / 466 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	NCT00428883 (ClinicalTrials.gov)	January 2007	29/1/2007	High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis	Rare Disease With Microvascular Involvement: High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis	Scleroderma, Diffuse	Drug: N-acetylcysteine (NAC)	Università Politecnica delle Marche	NULL	Recruiting	18 Years	80 Years	Both	45	Phase 2;Phase 3	Italy

58. Hypertrophic cardiomyopathy [ 92 clinical trials, 126 drugs, (DrugBank: 35 drugs), 40 drug target genes, 141 drug target pathways]

Searched query = "Hypertrophic cardiomyopathy"
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 / 92 trial found

85. Idiopathic interstitial pneumonia [ 514 clinical trials, 377 drugs, (DrugBank: 108 drugs), 97 drug target genes, 204 drug target pathways]

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

90. Retinitis pigmentosa [ 103 clinical trials, 158 drugs, (DrugBank: 42 drugs), 52 drug target genes, 107 drug target pathways]

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

94. Primary sclerosing cholangitis [ 134 clinical trials, 105 drugs, (DrugBank: 37 drugs), 18 drug target genes, 131 drug target pathways]

Searched query = "Primary sclerosing cholangitis", "PSC"
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 / 134 trial found

111. Congenital myopathy [ 7 clinical trials, 13 drugs, (DrugBank: 4 drugs), 1 drug target gene, 8 drug target pathways]

Searched query = "Congenital myopathy", "Nemaline myopathy", "Central core disease", "Minicore myopathy", "Myotubular myopathy", "Centronuclear myopathy", "Congenital fiber-type disproportion myopathy"
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 / 7 trial found

164. Oculocutaneous albinism [ 15 clinical trials, 57 drugs, (DrugBank: 34 drugs), 34 drug target genes, 136 drug target pathways]

Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli syndrome"
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 / 15 trial found

193. Prader-Willi syndrome [ 95 clinical trials, 104 drugs, (DrugBank: 27 drugs), 50 drug target genes, 63 drug target pathways]

Searched query = "Prader-Willi syndrome"
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 / 95 trial found

233. Wolfram syndrome [ 6 clinical trials, 12 drugs, (DrugBank: 7 drugs), 11 drug target genes, 40 drug target pathways]

Searched query = "Wolfram syndrome", "Diabetes Insipidus, Diabetes mellitus, optic atrophy, and deafness syndrome", "DIDMOAD syndrome"
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 / 6 trial found

298. Hereditary pancreatitis [ 93 clinical trials, 168 drugs, (DrugBank: 51 drugs), 48 drug target genes, 134 drug target pathways]

Searched query = "Hereditary pancreatitis", "Chronic pancreatitis"
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 / 93 trial found

299. Cystic fibrosis [ 1,592 clinical trials, 1,539 drugs, (DrugBank: 255 drugs), 81 drug target genes, 162 drug target pathways]

Searched query = "Cystic fibrosis"
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 / 1,592 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	NCT00809094 (ClinicalTrials.gov)	November 2008	15/12/2008	NAC Phase IIB: A Multi-Center, Phase IIB, Randomized, Placebo-controlled, Double-Blind Study Of The Effects Of N-Acetylcysteine On Redox Changes and Lung Inflammation In Cystic Fibrosis Patients	A Multi-Center, Phase IIB, Randomized, Placebo-controlled, Double-Blind Study Of The Effects Of N-Acetylcysteine On Redox Changes and Lung Inflammation In Cystic Fibrosis Patients	Cystic Fibrosis	Drug: N-acetylcysteine (NAC);Drug: Placebo	Stanford University	Cystic Fibrosis Foundation Therapeutics	Completed	7 Years	N/A	All	70	Phase 2	United States