TBD ( DrugBank: - )

5 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	3
90	網膜色素変性症	1
276	軟骨無形成症	1
299	嚢胞性線維症	1

19. ライソゾーム病

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2014-003480-37-IT (EUCTR)	06/05/2015	07/01/2015	An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients with CLN2 Disease	A 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.	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	22		United States;Germany;United Kingdom;Japan;Italy
2	EUCTR2012-005430-11-IT (EUCTR)	14/05/2014	20/01/2014	Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2	A 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.	NULL	Not Recruiting	Female: yes Male: yes	22	Phase 1;Phase 2	Germany;United Kingdom;Italy
3	EUCTR2012-005430-11-DE (EUCTR)	11/09/2013	10/04/2013	Phase 1/2 study to evaluate safety and efficacy of BMN 190 in patients with CLN2	A 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.	NULL	Not Recruiting	Female: yes Male: yes	22	Phase 1;Phase 2	Germany;United Kingdom

86. 肺動脈性肺高血圧症

臨床試験数 : 1,205 / 薬物数 : 684 - (DrugBank : 124) / 標的遺伝子数 : 100 - 標的パスウェイ数 : 193

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2016-001411-20-GB (EUCTR)	12/01/2018	09/01/2017	Long-term extension study of the safety and pharmacokinetics of QCC374 in PAH patients	Long-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 AG	NULL	Not Recruiting	Female: yes Male: yes	38	Phase 2	United States;Taiwan;Germany;United Kingdom;Korea, Republic of
2	EUCTR2016-001411-20-DE (EUCTR)	17/10/2017	07/08/2017	Long-term extension study of the safety and pharmacokinetics of QCC374 in PAH patients	Long-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 AG	NULL	Not Recruiting	Female: yes Male: yes	38	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no	United States;Taiwan;Germany;United Kingdom;Korea, Republic of
3	EUCTR2016-001412-38-DE (EUCTR)	27/07/2017	03/04/2017	Safety, pharmacokinetics and efficacy study of QCC374 in PAH patients	A 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 AG	NULL	Not Recruiting	Female: yes Male: yes	38	Phase 2	United States;Taiwan;Germany;United Kingdom;Korea, Republic of

90. 網膜色素変性症

臨床試験数 : 147 / 薬物数 : 176 - (DrugBank : 43) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 110

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	EUCTR2016-002696-10-SE (EUCTR)	09/05/2018	21/12/2017	Explore the safety and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene	An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene	retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]	Product Code: CPK850 INN or Proposed INN: TBD	Novartis Pharma AG	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	21	Phase 1;Phase 2	Sweden

276. 軟骨無形成症

臨床試験数 : 51 / 薬物数 : 34 - (DrugBank : 6) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 26

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	EUCTR2013-004137-32-FR (EUCTR)		18/06/2015	A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia	A 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.	NULL	Not Recruiting			Female: yes Male: yes	24	Phase 2	United States;France;Australia;United Kingdom

299. 嚢胞性線維症

臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174

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	EUCTR2019-000750-63-NL (EUCTR)	31/10/2019	05/08/2019	Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutation	A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation	Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: ABBV-2222 Product Code: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 INN or Proposed INN: Galicaftor Other descriptive name: ABBV-2222 Product Name: ABBV-3067 Product Code: ABBV-3067 INN or Proposed INN: TBD Other descriptive name: ABBV-3067	AbbVie Deutschland	NULL	Not Recruiting			Female: yes Male: yes	189	Phase 2	Serbia;United States;Slovakia;Spain;United Kingdom;France;Czech Republic;Hungary;Canada;Puerto Rico;Belgium;Poland;Netherlands

先頭へ