Non applicable

( DrugBank: - / KEGG DRUG: - )

2 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
10	シャルコー・マリー・トゥース病	1

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	5

10. シャルコー・マリー・トゥース病

臨床試験数 : 41 / 薬物数 : 46 - (DrugBank : 9) / 標的遺伝子数 : 11 - 標的パスウェイ数 : 15

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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	EUCTR2010-023097-40-FR (EUCTR)	29/11/2010	04/10/2010	A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX300 ...	A Phase II, Randomised, Placebo-Controlled Trial of the Safety, Efficacy, Pharmacodynamics of PTX300 ...	Charcot-Marie-Tooth disease (type 1A). MedDRA version: 12.1;Level: LLT;Classification code 10008414; ...	Product Name: non applicable Product Code: PXT3003 INN or Proposed INN: Baclofen INN or Proposed INN: N ...	Pharnext	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	80	Phase 2	France

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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

trials per page:

Showing 1 to 5 of 5 trials

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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	JPRN-jRCT2031210126 (jRCT2031210126)	27/07/2021	31/05/2021	Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II	Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II	mucopolysaccharidosis type II (MPS II)	[Post-marketing clinical study drug] Name - Post-marketing clinical study drug (code): IZCARGO for I.V ...	Yamamoto Tatsuyoshi	NULL	Recruiting	Not applicable	Not applicable	Both	20	Phase 4	Japan
2	EUCTR2019-002636-82-IT (EUCTR)	02/12/2019	30/07/2019	A clinical trial to evaluate the effect of OTL-200, a gene therapy treatment in patients with Late J ...	An open label, non-randomised trial to evaluate the safety and efficacy of a single infusion of OTL- ...	Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metac ...	Product Code: OTL-200 INN or Proposed INN: Not Applicable Other descriptive name: OTL-200 Dispersion f ...	Orchard Therapeutics (Europe) Limited	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	6	Phase 3	Italy
3	EUCTR2017-002806-10-NL (EUCTR)	29/11/2019	08/11/2018	An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, ...	An open, non-controlled, parallel, ascending multiple-dose, multicenter study to assess the safety, ...	Mucopolysaccharidosis Type IIIA or Sanfilippo Syndrome MedDRA version: 20.1;Level: LLT;Classificatio ...	Product Name: Chemically modified recombinant human sulfamidase Product Code: SOBI003 INN or Proposed ...	Swedish Orphan Biovitrum AB (publ)	NULL	Not Recruiting			Female: yes Male: yes	9	Phase 1	United States;Turkey;Germany;Netherlands
4	JPRN-jRCTs061190017 (jRCTs061190017)	21/11/2019	26/09/2019	Japan-Ambroxol Chaperone Study	2 cohort, Non-randomized, Multi-site Study to Evaluate the Efficacy and Safety of Chaperone Therapy ...	Neuronopathic Gaucher disease	Ambroxol will be given.	Narita Aya	NULL	Recruiting	Not applicable	Not applicable	Both	25	Phase 3	Japan
5	EUCTR2011-004410-42-FR (EUCTR)	26/11/2012	19/12/2012	Intracerebral Gene Therapy for MLD	A phase I/II, open labeled, monocentric study of direct intracranial administration of a replication ...	Early onset forms of MLD MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous ...	Product Name: AAVrh.10cuARSA Product Code: Non applicable	Inserm	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 1/2	France

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