120. 遺伝性ジストニア Hereditary dystonia Clinical trials / Disease details

臨床試験数 : 26 / 薬物数 : 19 - (DrugBank : 3) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 2

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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	NCT04184453 (ClinicalTrials.gov)	December 23, 2019	29/11/2019	Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasmin ...	Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasmin ...	Aceruloplasminemia	Drug: Deferiprone treated	First Affiliated Hospital of Fujian Medical University	NULL	Recruiting	18 Years	80 Years	All	5	Early Phase 1	China
2	NCT03726996 (ClinicalTrials.gov)	January 14, 2019	29/10/2018	Desipramine in Infantile Neuroaxonal Dystrophy (INAD).	Novel Off-label Use of Desipramine in Infantile Neuroaxonal Dystrophy: Targeting the Sphingolipid Me ...	Infantile Neuroaxonal Dystrophy	Drug: Desipramine	Duke University	NULL	Terminated	3 Years	17 Years	All	4	Phase 4	United States
3	NCT03570931 (ClinicalTrials.gov)	November 5, 2018	11/6/2018	A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy	A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neu ...	Infantile Neuroaxonal Dystrophy	Drug: RT001	Retrotope, Inc.	NULL	Active, not recruiting	18 Months	10 Years	All	19	Phase 2/Phase 3	United States
4	ChiCTR1900021076	2018-09-04	2019-01-27	A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegenerat ...	A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegenerat ...	pantothenate kinase-associated neurodegeneration	Experimental group:Standardized basal treatment + pantethine;Control group:Standardized basal treatm ...	Department of Pediatrics, Peking University First Hospital	NULL	Recruiting	0	16	Both	Experimental group:12;Control group:6;		China
5	NCT03857607 (ClinicalTrials.gov)	September 1, 2018	26/2/2019	Natural History Study of ATP1A3-related Disease	Natural History of ATP1A3-related Disease: a Deep Phenotyping-genotyping Project	ATP1A3-related Disease;Alternating Hemiplegia of Childhood;Rapid Onset Dystonia Parkinsonism;CAPOS	Genetic: Whole exome sequencing	Institute of Child Health	Great Ormond Street Hospital for Children NHS Foundation Trust;University College, London	Recruiting	6 Months	60 Years	All	100		United Kingdom
6	EUCTR2016-001955-29-FR (EUCTR)	29/01/2018	04/06/2018	Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associa ...	Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement ...	Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, th ...	Product Name: Fosmetpantotenate Product Code: RE-024 INN or Proposed INN: Fosmetpantotenate Other descr ...	Retrophin, Inc.	NULL	Not Recruiting			Female: yes Male: yes	82	Phase 3	Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain
7	EUCTR2016-001955-29-IT (EUCTR)	10/11/2017	05/11/2020	Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associa ...	Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement ...	Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, th ...	Product Name: Fosmetpantotenato Product Code: [RE-024] INN or Proposed INN: Fosmetpantotenato	RETROPHIN, INC	NULL	Not Recruiting			Female: yes Male: yes	82	Phase 3	United States;France;Czechia;Czech Republic;Canada;Spain;Poland;Norway;Germany;United Kingdom;Italy
8	EUCTR2016-001955-29-ES (EUCTR)	16/10/2017	30/03/2017	Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associa ...	Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement ...	Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, th ...	Product Name: Fosmetpantotenate Product Code: RE-024 INN or Proposed INN: Fosmetpantotenate	Retrophin, Inc.	NULL	Not Recruiting			Female: yes Male: yes	82	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	France;United States;Czech Republic;Canada;Poland;Spain;Norway;Germany;Italy;United Kingdom
9	EUCTR2016-001955-29-CZ (EUCTR)	06/10/2017	20/04/2017	Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associa ...	Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement ...	Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, th ...	Product Name: Fosmetpantotenate Product Code: RE-024 INN or Proposed INN: Fosmetpantotenate Other descr ...	Retrophin, Inc.	NULL	Not Recruiting			Female: yes Male: yes	82	Phase 3	Canada;Czech Republic;Norway;United States;Poland;Italy;United Kingdom;France;Germany;Spain
10	NCT03041116 (ClinicalTrials.gov)	July 17, 2017	2/12/2016	Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients	Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement ...	Pantothenate Kinase-Associated Neurodegeneration	Drug: fosmetpantotenate (RE-024);Drug: Placebo	Travere Therapeutics, Inc.	NULL	Active, not recruiting	6 Years	65 Years	All	82	Phase 3	United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain

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