DDrare: Database of Drug Development for Rare Diseases

Procedure: liver biopsy (DrugBank: -)

3 diseases

ID	Disease name (Link within this page)	Number of trials
171	Wilson disease	1
231	Alpha-1-antitrypsin deficiency	1
240	Phenylketonuria	1

171. Wilson disease [ 50 clinical trials, 59 drugs, (DrugBank: 17 drugs), 3 drug target genes, 28 drug target pathways]

Searched query = "Wilson 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 / 50 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	NCT02702765 (ClinicalTrials.gov)	February 2016	26/2/2016	sCD163 and sMR in Wilsons Disease - Associations With Disease Severity and Fibrosis	Macrophages and the Macrophage Activation Markers sCD163 and Mannose Receptor (sMR) in Patients With Wilsons Disease - Associations With Liver Disease Severity and Fibrosis	Wilsons Disease	Procedure: Fibroscan;Procedure: Ultrasound;Drug: Galactose;Procedure: Liver biopsy;Procedure: Functional hepatic nitrogen clearance	University of Aarhus	Aarhus University Hospital	Active, not recruiting	18 Years	100 Years	All	33	N/A	Denmark

231. Alpha-1-antitrypsin deficiency [ 83 clinical trials, 89 drugs, (DrugBank: 16 drugs), 35 drug target genes, 46 drug target pathways]

Searched query = "Alpha-1-antitrypsin deficiency", "AATD"
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 / 83 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	NCT01810458 (ClinicalTrials.gov)	October 2013	6/3/2013	Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)	Clinical Predictors and Epigenetic Markers for Liver Fibrosis in Alpha-1 Antitrypsin Deficiency	Liver Fibrosis;Alpha-1 Antitrypsin Deficiency;AAT Deficiency;AATD	Device: Abdominal ultrasound;Procedure: History and physical;Procedure: Intravenous catheter;Procedure: Blood draw;Other: Liver questionnaire;Procedure: Liver Biopsy;Drug: Midazolam;Drug: Fentanyl;Drug: Lidocaine;Drug: Acetaminophen;Drug: Lorazepam;Drug: Oxycodone/Acetaminophen;Drug: Ondansetron	University of Florida	NULL	Completed	18 Years	70 Years	All	109		United States

240. Phenylketonuria [ 125 clinical trials, 95 drugs, (DrugBank: 11 drugs), 1 drug target gene, 5 drug target pathways]

Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
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 / 125 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	NCT01465100 (ClinicalTrials.gov)	February 2021	20/10/2011	Liver Cell Transplant for Phenylketonuria	Hepatocyte Transplantation for Phenylketonuria	Phenylketonuria	Radiation: Preparative Radiation Therapy;Procedure: Hepatocyte Transplant;Drug: Immunosuppression;Other: Liver Evaluation;Behavioral: Neuro-psychological Assessment;Diagnostic Test: Whole body Phe oxidation testing;Procedure: Liver Biopsy	Ira Fox	NULL	Recruiting	14 Years	55 Years	All	10	Phase 1;Phase 2	United States