18. Spinocerebellar degeneration Clinical trials / Disease details

Clinical trials : 76 / Drugs : 98 - (DrugBank : 31) / Drug target genes : 44 - Drug target pathways : 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 2023	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	NCT05515536 (ClinicalTrials.gov)	December 8, 2022	23/8/2022	A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia	Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedre ...	Friedreich Ataxia	Drug: Vatiquinone	PTC Therapeutics	NULL	Enrolling by invitation	N/A	N/A	All	140	Phase 3	United States;Australia;Brazil;Canada;France;Germany;Italy;New Zealand;Spain
3	NCT05573698 (ClinicalTrials.gov)	September 27, 2022	6/10/2022	Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich ...	A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose Stu ...	Friedreich Ataxia	Drug: DT-216;Drug: Placebo	Design Therapeutics	NULL	Recruiting	18 Years	55 Years	All	38	Phase 1	United States
4	NCT05579691 (ClinicalTrials.gov)	September 21, 2022	5/10/2022	A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedr ...	A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose Exploration Study to Assess the Safety ...	Friedreich Ataxia	Biological: CTI-1601;Other: Placebo	Larimar Therapeutics, Inc.	NULL	Recruiting	18 Years	N/A	All	15	Phase 2	United States
5	NCT05445323 (ClinicalTrials.gov)	August 24, 2022	23/6/2022	Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia	A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants With Cardiomyopa ...	Friedreich Ataxia;Cardiomyopathy, Secondary	Genetic: Low dose LX2006;Genetic: High Dose LX2006	Lexeo Therapeutics	NULL	Recruiting	18 Years	40 Years	All	10	Phase 1/Phase 2	United States
6	NCT05485987 (ClinicalTrials.gov)	August 15, 2022	1/8/2022	A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia	An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children Wi ...	Friedreich Ataxia	Drug: Vatiquinone	PTC Therapeutics	NULL	Recruiting	N/A	6 Years	All	5	Phase 2	United States
7	NCT05285540 (ClinicalTrials.gov)	March 11, 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
8	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
9	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
10	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

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