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Sapropterindihydrochloride    (DrugBank: -)

5 diseases
IDDisease name (Link within this page)Number of trials
51Scleroderma0
86Pulmonary arterial hypertension0
124Cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy0
240Phenylketonuria2
299Cystic fibrosis0

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.
0 / 1,083 trial found

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 125 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2009-018168-81-DK
(EUCTR)		16/04/201003/03/2010ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDUREENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 14.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 10010331 - Congenital, familial and genetic disorders		Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE		Merck Serono NorwayNULLNot RecruitingFemale: yes
Male: yes		70Phase 4Denmark
2EUCTR2009-018168-81-NO
(EUCTR)		10/03/2010ENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDUREENDURE: A Phase IV, prospective, open-label, uncontrolled, multi-centre cohort trial to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 28 days - ENDURE Subjects diagnosed with Phenylketonuria (PKU) (classic or mild PKU, or mild hyperphenylalaninaemia (HPA)).
MedDRA version: 12.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU)		Trade Name: Kuvan
INN or Proposed INN: Sapropterindihydrochloride
Other descriptive name: SAPROPTERIN HYDROCHLORIDE		Merck Serono NorwayNULLNAFemale: yes
Male: yes		150Phase 4Denmark;Norway

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