DDrare: Database of Drug Development for Rare Diseases

Obeticholic acid (DrugBank: Obeticholic acid)

4 diseases

ID	Disease name (Link within this page)	Number of trials
20	Adrenoleukodystrophy	0
234	Peroxisomal disease (except Adrenoleukodystrophy)	0
265	Lipodystrophy	1
310	Congenital anomalies syndrome	0

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

234. Peroxisomal disease (except Adrenoleukodystrophy) [ 38 clinical trials, 37 drugs, (DrugBank: 12 drugs), 12 drug target genes, 42 drug target pathways]

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
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 / 38 trial found

265. Lipodystrophy [ 109 clinical trials, 164 drugs, (DrugBank: 59 drugs), 26 drug target genes, 94 drug target pathways]

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons 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 / 109 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	NCT02430077 (ClinicalTrials.gov)	June 2016	24/4/2015	Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients	Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients	Familial Partial Lipodystrophy	Drug: Obeticholic Acid;Drug: Placebo	University of Texas Southwestern Medical Center	NULL	Recruiting	18 Years	70 Years	All	20	Phase 2	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