DDrare: Database of Drug Development for Rare Diseases

3h-cholesterol bound to albumin (DrugBank: Cholesterol)

6 diseases

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	0
90	Retinitis pigmentosa	0
160	Congenital ichthyosis	0
259	Lecithin-cholesterol acyltransferase deficiency	1
261	Tangier disease	1
310	Congenital anomalies syndrome	0

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

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

0 / 103 trial found

160. Congenital ichthyosis [ 32 clinical trials, 62 drugs, (DrugBank: 18 drugs), 15 drug target genes, 107 drug target pathways]

Searched query = "Congenital ichthyosis", "Keratinopathic ichthyosis", "Epidermolytic ichthyosis", "Harlequin ichthyosis", "Congenital Ichthyosiform Erythroderma", "Foliate ichthyosis", "Ichthyosis syndrome", "Netherton syndrome", "Sjogren-Larsson syndrome", "Sjögren-Larsson syndrome", "Keratitis-ichtyosis-deafness syndrome", "Dorfman-Chanarin syndrome", "Neutral lipid storage disease", "NLSD", "Multiple sulfatase deficiency", "Austin disease", "Recessive X-linked ichthyosis", "RXLI", "X-linked recessive ichthyosis", "Ichthyosis, brittle hair, impaired intelligence, decreased fertility and short stature", "IBID", "Trichothiodystrophy", "Follicular ichthyosis", "Congenital hemidysplasia, ichthyosiform erythroderma or nevus, and limb defects syndrome", "CHILD syndrome", "Conradi-Hunermann-Happle syndrome", "Conradi-Hünermann-Happle syndrome", "CHHS"
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 / 32 trial found

259. Lecithin-cholesterol acyltransferase deficiency [ 1 clinical trial, 2 drugs, (DrugBank: 1 drug), 0 drug target gene, 0 drug target pathway]

Searched query = "Lecithin-cholesterol acyltransferase deficiency", "LCAT deficiency", "Fish-eye 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 / 1 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	NCT01782027 (ClinicalTrials.gov)	October 2012	30/1/2013	Mendelian Reverse Cholesterol Transport Study	A Validation Study Evaluating the Use of 3H-Cholesterol Bound to Albumin as a Method to Assess Reverse Cholesterol Transport in Subjects With Monogenic Diseases Affecting HDL Metabolism	Cholesterol, HDL;Lipid Metabolism, Inborn Errors;Tangier Disease;LCAT Deficiency;Cholesteryl Ester Transfer Protein (CETP) Deficiency	Drug: 3H-cholesterol bound to albumin	University of Pennsylvania	NULL	Suspended	18 Years	75 Years	All	30	N/A	United States

261. Tangier disease [ 1 clinical trial, 2 drugs, (DrugBank: 1 drug), 0 drug target gene, 0 drug target pathway]

Searched query = "Tangier 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 / 1 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	NCT01782027 (ClinicalTrials.gov)	October 2012	30/1/2013	Mendelian Reverse Cholesterol Transport Study	A Validation Study Evaluating the Use of 3H-Cholesterol Bound to Albumin as a Method to Assess Reverse Cholesterol Transport in Subjects With Monogenic Diseases Affecting HDL Metabolism	Cholesterol, HDL;Lipid Metabolism, Inborn Errors;Tangier Disease;LCAT Deficiency;Cholesteryl Ester Transfer Protein (CETP) Deficiency	Drug: 3H-cholesterol bound to albumin	University of Pennsylvania	NULL	Suspended	18 Years	75 Years	All	30	N/A	United States

310. Congenital anomalies syndrome [ 10 clinical trials, 18 drugs, (DrugBank: 10 drugs), 3 drug target genes, 7 drug target pathways]

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

0 / 10 trial found