DEFERIPRONE ( DrugBank: Deferiprone )

6 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	4
6	パーキンソン病	15
18	脊髄小脳変性症（多系統萎縮症を除く。）	2
120	遺伝性ジストニア	11
122	脳表ヘモジデリン沈着症	2
233	ウォルフラム症候群	1

2. 筋萎縮性側索硬化症

臨床試験数 : 645 / 薬物数 : 589 - (DrugBank : 163) / 標的遺伝子数 : 150 - 標的パスウェイ数 : 225

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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	NCT03293069 (ClinicalTrials.gov)	January 1, 2019	21/9/2017	Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis	Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis: Multic ...	Amyotrophic Lateral Sclerosis	Drug: Deferiprone;Drug: Placebo Oral Tablet	University Hospital, Lille	Ministry of Health, France	Active, not recruiting	18 Years	75 Years	All	372	Phase 2/Phase 3	France
2	EUCTR2017-003763-35-FR (EUCTR)	17/05/2018	05/03/2018	conservative iron chelation by deferiprone for amyotrophic lateral sclerosis	Conservative Iron Chelation by Deferiproneas a disease-modifying Strategy for Amyotrophic Lateral Sc ...	De novo Amyotrophic Lateral Sclerosis patients;Therapeutic area: Diseases [C] - Nervous System Disea ...	Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	centre hospitalier universitaire de Lille	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	240	Phase 3	France
3	NCT02164253 (ClinicalTrials.gov)	September 2013	20/5/2014	Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients ...	Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferipronein Amyotrophic Latera ...	ALS (Amyotrophic Lateral Sclerosis);Iron Overload	Drug: Deferiprone	University Hospital, Lille	ApoPharma	Completed	18 Years	85 Years	All	23	Phase 2	France
4	NCT02880033 (ClinicalTrials.gov)	February 2011	3/8/2016	Oxidative Stress and Apoptosis of Energy Metabolism by Deferiprone From the Circulating Lymphocytes	Modulation of Oxidative Stress and Apoptosis of Energy Metabolism by DeferiproneFrom the Circulating ...	Parkinson's Disease;Amyotrophic Lateral Sclerosis;Oxidative Stress;Iron Overload	Drug: deferiprone;Drug: placebo	University Hospital, Lille	NULL	Completed	18 Years	80 Years	All	90	N/A	France

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6. パーキンソン病

臨床試験数 : 2,307 / 薬物数 : 2,007 - (DrugBank : 349) / 標的遺伝子数 : 188 - 標的パスウェイ数 : 199

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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	EUCTR2015-004344-19-DE (EUCTR)	07/02/2017	27/09/2016	An international trial of deferiprone in patients with Parkinson’s disease	A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of DeferiproneDelayed Release Tab ...	Parkinson’s disease MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's di ...	Product Name: Deferiprone 600 mg delayed release tablet INN or Proposed INN: DEFERIPRONE	ApoPharma	NULL	Not Recruiting			Female: yes Male: yes	140	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	France;Canada;Germany;United Kingdom
2	NCT02728843 (ClinicalTrials.gov)	October 12, 2016	31/3/2016	Study of Parkinson's Early Stage With Deferiprone	A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of DeferiproneDelayed Release Tab ...	Parkinson's Disease	Drug: Deferiprone;Drug: Placebo	ApoPharma	NULL	Completed	18 Years	80 Years	All	140	Phase 2	Canada;France;Germany;United Kingdom
3	EUCTR2015-003679-31-NL (EUCTR)	09/08/2016	24/05/2016	New therapeutic strategy in Parkinson’s disease	Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease - FAIRPARK II	De Novo Parkinson’s disease;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	Centre Hospitalier Régional et Universitaire de Lille	NULL	Not Recruiting			Female: yes Male: yes	338	Phase 2	Portugal;Czech Republic;Spain;Austria;Netherlands
4	EUCTR2015-004344-19-FR (EUCTR)	29/06/2016	20/04/2016	An international trial of deferiprone in patients with parkinson’s disease	A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of DeferiproneDelayed Release Tab ...	Parkinson’s disease MedDRA version: 19.0;Level: PT;Classification code 10061536;Term: Parkinson's di ...	Product Name: Deferiprone 600 mg delayed release tablet INN or Proposed INN: DEFERIPRONE	ApoPharma Inc.	NULL	Not Recruiting			Female: yes Male: yes	140	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	United States;France;Canada;Germany;Italy;United Kingdom
5	EUCTR2015-003679-31-CZ (EUCTR)	23/06/2016	28/01/2016	New therapeutic strategy in Parkinson’s disease	Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease - FAIRPARK II	De Novo Parkinson’s disease;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	Centre Hospitalier Régional et Universitaire de Lille	NULL	Not Recruiting			Female: yes Male: yes	338	Phase 2	Portugal;Czech Republic;Spain;Austria;Netherlands
6	EUCTR2015-003679-31-PT (EUCTR)	18/04/2016	28/01/2016	New therapeutic strategy in Parkinson’s disease	Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease - FAIRPARK II	De Novo Parkinson’s disease;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	Centre Hospitalier Régional et Universitaire de Lille	NULL	Not Recruiting			Female: yes Male: yes	338	Phase 2	Portugal;Czech Republic;Spain;Austria;Netherlands
7	EUCTR2015-003679-31-AT (EUCTR)	06/04/2016	11/01/2016	New therapeutic strategy in Parkinson’s disease	Conservative iron chelation as a disease-modifying strategy in Parkinson’s disease - FAIRPARK II	De Novo Parkinson’s disease;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	Centre Hospitalier Régional et Universitaire de Lille	NULL	Not Recruiting			Female: yes Male: yes	372	Phase 2	Portugal;France;Czech Republic;Spain;Austria;Netherlands;Germany;United Kingdom
8	EUCTR2015-004344-19-GB (EUCTR)	31/03/2016	16/02/2016	An international trial of deferiprone in patients with Parkinson’s disease	A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of DeferiproneDelayed Release Tab ...	Parkinson’s disease MedDRA version: 20.0;Level: PT;Classification code 10061536;Term: Parkinson's di ...	Product Name: Deferiprone 600 mg delayed release tablet INN or Proposed INN: DEFERIPRONE	ApoPharma	NULL	Not Recruiting			Female: yes Male: yes	140	Phase 2	France;Canada;Germany;United Kingdom
9	EUCTR2015-003679-31-ES (EUCTR)	21/03/2016	13/01/2016	New therapeutic strategy in Parkinson?s disease	Conservative iron chelation as a disease-modifying strategy in Parkinson?s disease: a multicentre, p ...	De Novo Parkinson?s disease MedDRA version: 18.1;Level: PT;Classification code 10061536;Term: Parkin ...	Trade Name: Ferriprox 500 mg Product Name: Deferiprone DR INN or Proposed INN: DEFERIPRONE	Centre Hospitalier Régional et Universitaire de Lille	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	338	Phase 3	Spain
10	NCT02655315 (ClinicalTrials.gov)	February 9, 2016	7/1/2016	Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease	Conservative Iron Chelation as a Disease-modifying Strategy in Parkinson's Disease. European Multice ...	Parkinson Disease	Drug: Deferiprone;Drug: Placebo	University Hospital, Lille	European Commission;ApoPharma	Completed	N/A	80 Years	All	372	Phase 2	Austria;Czechia;France;Germany;Netherlands;Portugal;Spain;United Kingdom;Czech Republic

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18. 脊髄小脳変性症（多系統萎縮症を除く。）

臨床試験数 : 76 / 薬物数 : 98 - (DrugBank : 31) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 65

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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	EUCTR2009-010865-22-IT (EUCTR)	06/05/2009	16/04/2009	An open-label, single treatment, safety and efficacy, long-term study of deferipronein subjects with ...	An open-label, single treatment, safety and efficacy, long-term study of deferipronein subjects with ...	patients affected by Friedreich Ataxia	INN or Proposed INN: Deferiprone	APOPHARMA INC., TORONTO, ONTARIO	NULL	Not Recruiting			Female: yes Male: yes	74		Belgium;France;Spain;Italy
2	EUCTR2007-003331-23-IT (EUCTR)	16/07/2008	10/07/2008	A study investigating the safety and tolerability of deferipronein patients with Friedreich's Ataxia ...	A study investigating the safety and tolerability of deferipronein patients with Friedreich's Ataxia ...	patients affected by Friedreich Ataxia MedDRA version: 9.1;Level: LLT;Classification code 10017374;T ...	Product Name: Deferiprone INN or Proposed INN: Deferiprone	APOPHARMA INC., TORONTO, ONTARIO	NULL	Not Recruiting			Female: yes Male: yes	80		United Kingdom;Belgium;France;Spain;Italy

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120. 遺伝性ジストニア

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 DeferiproneTablets in Aceruloplasmina ...	Clinical Curative Effect Evaluation Study of Treatment of Oral DeferiproneTablets in Aceruloplasmina ...	Aceruloplasminemia	Drug: Deferiprone treated	First Affiliated Hospital of Fujian Medical University	NULL	Recruiting	18 Years	80 Years	All	5	Early Phase 1	China
2	NCT02635841 (ClinicalTrials.gov)	June 16, 2016	17/12/2015	Compassionate Use of Deferiprone in Patients With PKAN	The Compassionate Use of Deferipronein Patients With Pantothenate Kinase-Associated Neurodegeneratio ...	Pantothenate Kinase-Associated Neurodegeneration	Drug: Deferiprone	Chiesi Canada Corp	NULL	No longer available	7 Years	N/A	All			NULL
3	EUCTR2014-001427-79-GB (EUCTR)	30/12/2015	05/11/2015	An international open label trial of deferipronein patients with iron storage brain disorders - an e ...	Long-term Safety and Efficacy Study of Deferipronein Patients with Pantothenate Kinase-Associated Ne ...	Pantothenate Kinase-Associated Neurodegeneration (PKAN) MedDRA version: 18.1;Level: PT;Classificatio ...	Trade Name: Ferriprox Product Name: Deferiprone80 mg/mL oral solution INN or Proposed INN: Deferiprone ...	ApoPharma Inc	NULL	Not Recruiting			Female: yes Male: yes	89	Phase 3	United States;Germany;Italy;United Kingdom
4	EUCTR2014-001427-79-DE (EUCTR)	27/03/2015	27/03/2015	An 18-month extension of an international trial of deferipronein patients with iron storage brain di ...	Long-term Safety and Efficacy Study of Deferipronein Patients with Pantothenate Kinase-Associated Ne ...	Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous Sys ...	Product Name: deferiprone 80 mg/mL oral solution INN or Proposed INN: DEFERIPRONE	ApoPharma Inc.	NULL	Not Recruiting			Female: yes Male: yes	90	Phase 3	United States;Germany;Italy;United Kingdom
5	NCT02174848 (ClinicalTrials.gov)	June 2014	5/6/2014	Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration	Long-term Safety and Efficacy Study of Deferipronein Patients With Pantothenate Kinase-Associated Ne ...	Pantothenate Kinase-Associated Neurodegeneration	Drug: Deferiprone oral solution	ApoPharma	NULL	Completed	5 Years	N/A	All	68	Phase 3	United States;Germany;Italy;United Kingdom
6	EUCTR2012-000845-11-IT (EUCTR)	24/10/2013	31/07/2013	An international trial of deferiprone in patients with iron storage brain disorders	A randomized, double-blind, placebo-controlled trial of deferipronein patients with pantothenate kin ...	Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous Sys ...	Product Name: Deferiprone80 mg/mL oral solution INN or Proposed INN: Deferiprone Other descriptive nam ...	ApoPharma Inc.	NULL	Not Recruiting			Female: yes Male: yes	90	Phase 3	United States;Poland;Germany;United Kingdom;Italy
7	EUCTR2012-000845-11-GB (EUCTR)	24/10/2013	19/09/2013	An international trial of deferiprone in patients with iron storage brain disorders	A randomized, double-blind, placebo-controlled trial of deferipronein patients with pantothenate kin ...	Pantothenate Kinase-Associated Neurodegeneration (PKAN) MedDRA version: 16.0;Level: PT;Classificatio ...	Trade Name: Ferriprox Product Name: Deferiprone80 mg/mL oral solution (Ferriprox) INN or Proposed INN: ...	ApoPharma Inc	NULL	Not Recruiting			Female: yes Male: yes	90	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Poland;Germany;Italy;United Kingdom
8	NCT01741532 (ClinicalTrials.gov)	December 13, 2012	3/12/2012	Efficacy and Safety Study of Deferipronein Patients With Pantothenate Kinase-associated Neurodegener ...	A Randomized, Double-blind, Placebo-controlled Trial of Deferipronein Patients With Pantothenate Kin ...	Pantothenate Kinase-Associated Neurodegeneration	Drug: Deferiprone oral solution;Drug: Placebo	ApoPharma	Food and Drug Administration (FDA)	Completed	4 Years	N/A	All	89	Phase 3	United States;Germany;Italy;United Kingdom;Poland
9	EUCTR2012-000845-11-DE (EUCTR)	16/07/2012	30/03/2012	An international trial of deferiprone in patients with iron storage brain disorders	A randomized, double-blind, placebo-controlled trial of deferipronein patients with pantothenate kin ...	Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous Sys ...	Product Name: Deferiprone80 mg/mL oral solution INN or Proposed INN: Deferiprone Other descriptive nam ...	ApoPharma Inc.	NULL	Not Recruiting			Female: yes Male: yes	90	Phase 3	United States;Poland;Germany;United Kingdom;Italy
10	EUCTR2008-003059-56-IT (EUCTR)	29/07/2008	18/07/2008	Phase II trial to assess safety and efficacy of Iron chelating agent Deferipronein patients with Pan ...	Phase II trial to assess safety and efficacy of Iron chelating agent Deferipronein patients with Pan ...	PKAN MedDRA version: 9.1;Level: HLT;Classification code 10042259;Term: Structural brain disorders NE ...	Trade Name: FERRIPROX*100CPR RIV 500MG INN or Proposed INN: Deferiprone	ISTITUTO NEUROLOGICO CARLO BESTA	NULL	Not Recruiting			Female: yes Male: yes		Phase 2	Italy

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122. 脳表ヘモジデリン沈着症

臨床試験数 : 3 / 薬物数 : 3 - (DrugBank : 2) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

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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-UMIN000021218	2024/09/30	26/02/2016	Evaluation of iron chelator administration, and cochlear implantation for superficial siderosis (ECH ...	Evaluation of iron chelator administration, and cochlear implantation for superficial siderosis (ECH ...	superficial siderosis of CNS	deferiprone administration cochlear implantation	Department of Neurology, Tokyo Medical and Dental University	Department of Neurology, Kouchi University Hospital	Recruiting	20years-old	Not applicable	Male and Female	5	Not applicable	Japan
2	JPRN-jRCTs031180438	01/04/2019	27/03/2019	Evaluation of iron chelator administration, and cochlear implantation for superficial siderosis	Evaluation of iron chelator administration, and cochlear implantation for superficial siderosis - EC ...	Superficial siderosis siderosis, cerebellar ataxia, hearing loss, superficial siderosis	1. Administration of Deferiproneorally 30mg/kg after every meal for 36 months. 2. Artificial cochlear ...	Sanjo Nobuo	NULL	Recruiting	>= 20age old	Not applicable	Both	50	N/A	Japan

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233. ウォルフラム症候群

臨床試験数 : 11 / 薬物数 : 17 - (DrugBank : 8) / 標的遺伝子数 : 12 - 標的パスウェイ数 : 41

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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	NCT02882477 (ClinicalTrials.gov)	December 2016	17/7/2016	Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy	Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone, and Incretin Based Therapy	Diabetes Mellitus;Iron Metabolism Disorders;Gastroduodenal Ulcer;Optic Atrophy;Sensorineural Hearing ...	Drug: Deferiprone;Drug: Acetylcysteine;Drug: Sitagliptin and Metformin	Hadassah Medical Organization	NULL	Not yet recruiting	3 Years	N/A	Both	20	Phase 2/Phase 3	Israel

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