DDrare: Database of Drug Development for Rare Diseases

Sapropterin dihydrochloride (6r-bh4) (DrugBank: Sapropterin, 6R-BH4)

5 diseases

ID	Disease name (Link within this page)	Number of trials
51	Scleroderma	0
86	Pulmonary arterial hypertension	1
124	Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy	0
240	Phenylketonuria	0
299	Cystic fibrosis	0

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

0 / 466 trial found

86. Pulmonary arterial hypertension [ 1,083 clinical trials, 667 drugs, (DrugBank: 122 drugs), 98 drug target genes, 185 drug target pathways]

Searched query = "Pulmonary arterial hypertension", "PAH", "IPAH", "HPAH", "Eisenmenger 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 / 1,083 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	NCT00435331 (ClinicalTrials.gov)	March 2008	12/2/2007	6R-BH4 Pulmonary Arterial Hypertension Study	A Phase 1, Multicenter, Open-label, Dose-escalation Study to Evaluate the Safety and Efficacy of 6R-BH4 in Subjects With Pulmonary Arterial Hypertension	Pulmonary Arterial Hypertension	Drug: sapropterin dihydrochloride (6R-BH4)	Vanderbilt University	National Institutes of Health (NIH);BioMarin Pharmaceutical	Completed	18 Years	N/A	Both	14	Phase 1	United States

124. Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy [ 11 clinical trials, 13 drugs, (DrugBank: 4 drugs), 3 drug target genes, 11 drug target pathways]

Searched query = "Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy", "CADASIL", "Autosomal dominant cerebral artery disease with subcortical infarct and leukoencephalopathy", "Autosomal dominant cerebral artery disease"
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 / 11 trial found

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

0 / 125 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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 1,592 trial found