18. 脊髄小脳変性症（多系統萎縮症を除く。） Spinocerebellar degeneration Clinical trials / Disease details

臨床試験数 : 71 / 薬物数 : 99 - (DrugBank : 30) / 標的遺伝子数 : 45 - 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	NCT03761511 (ClinicalTrials.gov)	April 2022	27/11/2018	Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia	A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy an ...	Friedreich Ataxia	Drug: Nicotinamide;Drug: Placebo	RWTH Aachen University	Assistance Publique - Hôpitaux de Paris	Not yet recruiting	18 Years	N/A	All	225	Phase 2	Austria;France;Germany;Italy;Spain;United Kingdom
2	NCT05285540 (ClinicalTrials.gov)	March 7, 2022	9/3/2022	Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia	A Phase 1a, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose Study of DT-216 in A ...	Friedreich Ataxia	Drug: DT-216;Drug: DT-216 matching Placebo	Design Therapeutics	NULL	Recruiting	18 Years	55 Years	All	25	Phase 1	United States
3	NCT04801303 (ClinicalTrials.gov)	August 25, 2021	1/3/2021	Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patien ...	Pilot Trial About the Effects of Calcitriol's Treatment in the Neurological Function and Frataxin's ...	Friedreich Ataxia	Drug: Calcitriol;Other: Blood analysis for frataxin's level measurement;Diagnostic Test: Blood analy ...	Berta Alemany	Institut de Recerca Biomèdica de Lleida;Universitat de Lleida;Institut d'Investigació Biomèdica de G ...	Active, not recruiting	16 Years	65 Years	All	20	Phase 4	Spain
4	NCT04921930 (ClinicalTrials.gov)	July 1, 2021	31/5/2021	Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)	Evaluation of the Effect of Artesunate in Friedreich Ataxia(FA) Phase I-II Efficacy-Toxicity of Arte ...	Friedreich Ataxia	Drug: Artesunate Oral Product	Institut National de la Santé Et de la Recherche Médicale, France	Imagine Institute	Not yet recruiting	16 Years	65 Years	Male	20	Phase 1/Phase 2	NULL
5	EUCTR2020-002812-36-ES (EUCTR)	02/06/2021	19/11/2020	A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological c ...	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Asse ...	Friedreich Ataxia(FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ...	Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: ...	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	126	Phase 2;Phase 3	United States;France;Canada;Brazil;Spain;Australia;Germany;Italy
6	EUCTR2020-002812-36-IT (EUCTR)	14/04/2021	04/06/2021	A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological c ...	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Asse ...	Friedreich Ataxia(FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ...	Product Name: Vatiquinone Product Code: [PTC743] INN or Proposed INN: Vatiquinone	PTC THERAPEUTICS INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	126	Phase 2;Phase 3	France;United States;Canada;Spain;Brazil;Australia;Germany;Italy
7	NCT04817111 (ClinicalTrials.gov)	April 10, 2021	10/3/2021	NAD+ Precursor Supplementation in Friedreich's Ataxia	A Phase 2a Study of NAD+ Precursor Supplementation in Friedreich's Ataxia	Friedreich Ataxia	Drug: MIB-626	Metro International Biotech, LLC	Children's Hospital of Philadelphia	Recruiting	18 Years	64 Years	All	10	Phase 2	United States
8	EUCTR2021-000171-36-FR (EUCTR)	23/03/2021	22/01/2021	PHASE I-II EFFICACY-TOXICITY OF ARTESUNATE IN FRIEDREICH ATAXIA (FA)	PHASE I-II EFFICACY-TOXICITY OF ARTESUNATE IN FRIEDREICH ATAXIA	Friedreich Ataxia MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ata ...	Product Name: artesunate	INSERM	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	22	Phase 1;Phase 2	France
9	EUCTR2020-002812-36-FR (EUCTR)	18/03/2021	15/01/2021	A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological c ...	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Asse ...	Friedreich Ataxia(FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ...	Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: ...	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	126	Phase 2;Phase 3	United States;France;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand
10	EUCTR2020-002812-36-DE (EUCTR)	18/02/2021	06/11/2020	A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological c ...	A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study with Open-Label Extension to Asse ...	Friedreich Ataxia(FA) MedDRA version: 20.0;Level: PT;Classification code 10017374;Term: Friedreich's ...	Product Name: Vatiquinone Product Code: PTC743 INN or Proposed INN: VATIQUINONE Other descriptive name: ...	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	126	Phase 2;Phase 3	France;United States;Canada;Spain;Brazil;Australia;Germany;Italy;New Zealand

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