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Tbd    (DrugBank: -)

4 diseases
告示番号疾患名（ページ内リンク）臨床試験数
19ライソゾーム病3
86肺動脈性肺高血圧症2
276軟骨無形成症1
299嚢胞性線維症0

19. ライソゾーム病　［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 784 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2014-003480-37-IT
(EUCTR)		06/05/201507/01/2015An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 DiseaseA Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2).
MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)		BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes		22United States;Germany;United Kingdom;Japan;Italy
2EUCTR2012-005430-11-IT
(EUCTR)		14/05/201420/01/2014Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease Neuronal Ceroid Lipofuscinosis Type 2 (CLN2).
MedDRA version: 16.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Name: N/A
Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: recombinant human tripeptidyl peptidase-1		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		22Phase 1;Phase 2Germany;United Kingdom;Italy
3EUCTR2012-005430-11-DE
(EUCTR)		11/09/201310/04/2013Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients with Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2)
MedDRA version: 17.1;Level: LLT;Classification code 10052074;Term: Neuronal ceroid lipofuscinosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Product Name: N/A
Product Code: BMN 190
INN or Proposed INN: TBD
Other descriptive name: recombinant human tripeptidyl peptidase-1		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		22Phase 1;Phase 2Germany;United Kingdom

86. 肺動脈性肺高血圧症　［臨床試験数：1,083，薬物数：667（DrugBank：122），標的遺伝子数：98，標的パスウェイ数：185］
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.
2 / 1,083 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2016-001411-20-GB
(EUCTR)		12/01/201809/01/2017Long-term extension study of the safety and pharmacokinetics of QCC374 in PAH patientsLong-term, open label, multicenter, extension study to evaluate the safety and tolerability of QCC374 in patients with PAH Pulmonary arterial hypertension
MedDRA version: 20.0;Level: PT;Classification code 10064911;Term: Pulmonary arterial hypertension;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]		Product Code: QCC374 0.015 mg
INN or Proposed INN: TBD
Product Code: QCC374 0.06 mg
INN or Proposed INN: TBD		Novartis Pharma AGNULLNot RecruitingFemale: yes
Male: yes		38Phase 2United States;Taiwan;Germany;United Kingdom;Korea, Republic of
2EUCTR2016-001412-38-DE
(EUCTR)		27/07/201703/04/2017Safety, pharmacokinetics and efficacy study of QCC374 in PAH patientsA randomized, parallel-group, placebo-controlled subject and investigator blinded study to assess the safety, tolerability, pharmacokinetics and efficacy of QCC374 in the treatment of pulmonary arterial hypertension Pulmonary arterial hypertension
MedDRA version: 20.0;Level: PT;Classification code 10064911;Term: Pulmonary arterial hypertension;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]		Product Code: QCC374 0.015 mg
INN or Proposed INN: TBD
Product Code: QCC374 0.06 mg
INN or Proposed INN: TBD		Novartis Pharma AGNULLNot RecruitingFemale: yes
Male: yes		38Phase 2United States;Taiwan;Germany;United Kingdom;Korea, Republic of

276. 軟骨無形成症　［臨床試験数：37，薬物数：29（DrugBank：6），標的遺伝子数：4，標的パスウェイ数：25］
Searched query = "Achondroplasia"
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 / 37 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2013-004137-32-FR
(EUCTR)		18/06/2015A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia achondroplasia
MedDRA version: 18.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]		Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: TBD
Other descriptive name: MODIFIED RHCNP		BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes		24Phase 2United States;France;Australia;United Kingdom

299. 嚢胞性線維症　［臨床試験数：1,592，薬物数：1,539（DrugBank：255），標的遺伝子数：81，標的パスウェイ数：162］
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

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