337. ホモシスチン尿症 Homocystinuria Clinical trials / Disease details

臨床試験数 : 16 / 薬物数 : 19 - (DrugBank : 7) / 標的遺伝子数 : 2 - 標的パスウェイ数 : 23

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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	ChiCTR2100054613	2022-08-01	2021-12-21	Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ...	Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ...	Methylmalonic acidemia	Experimental group:PEG-rhGH;Control Group:None;	Zhengzhou Children's Hospital	NULL	Pending	0	3	Both	Experimental group:30;Control Group:30;	Phase 4	China
2	NCT05462132 (ClinicalTrials.gov)	July 7, 2022	7/7/2022	Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers	A Phase 1, Dose-escalation, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, ...	Homocystinuria	Drug: SYNB1353	Synlogic	NULL	Completed	18 Years	64 Years	All	31	Phase 1	United States
3	NCT04015557 (ClinicalTrials.gov)	February 11, 2022	28/5/2019	Effect of Acetaminophen and N-Acetylcysteine on Liver Metabolism on Homocystinuria	Functional Consequences and Therapeutic Intervention in Hampered Production of Cysteine, Glutathione ...	CBS Deficiency	Drug: Acetaminophen;Drug: N-acetylcysteine	Hospital de Clinicas de Porto Alegre	Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil	Suspended	18 Years	65 Years	All	10	Phase 1/Phase 2	Brazil
4	NCT05154890 (ClinicalTrials.gov)	May 13, 2021	20/8/2021	A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency	A Phase 1/2 Multiple Ascending-Dose Study in Subjects With HomocystinuriaDue to Cystathionine ß-Synt ...	Homocystinuria Due to Cystathionine Beta-Synthase Deficiency	Drug: Pegtarviliase	Aeglea Biotherapeutics	NULL	Recruiting	12 Years	N/A	All	36	Phase 1/Phase 2	United States;Australia;United Kingdom
5	EUCTR2019-004791-19-GB (EUCTR)	11/05/2020	14/01/2020	A Phase 1/2 Study of ACN00177 in Subjects With HomocystinuriaDue to Cystathionine ß-Synthase (CBS) D ...	A Phase 1/2 Multiple Ascending-Dose Study in Subjects With HomocystinuriaDue to Cystathionine ß-Synt ...	HomocystinuriaDue to Cystathionine ß-Synthase (CBS) Deficiency MedDRA version: 20.0;Level: PT;Classi ...	Product Code: ACN00177	Aeglea Biotherapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	25	Phase 1	United Kingdom
6	NCT04287881 (ClinicalTrials.gov)	October 31, 2019	22/2/2020	Methylenetetrahydrofolate Reductase Deficiency in Ischemic Stroke	Investigation the Incidence of Methylenetetrahydrofolate Reductase Deficiencyin Ischemic Cerebrovasc ...	Hyperhomocysteinemia, Thrombotic, Cbs-Related;Ischemic Stroke;Epilepsy	Genetic: Methylenetetrahydrofolate reductase (MTHFR) deficiency	Istanbul Medeniyet University	NULL	Completed	N/A	N/A	All	32	N/A	Turkey
7	NCT03406611 (ClinicalTrials.gov)	January 22, 2019	5/12/2017	Pegtibatinase as an Enzyme Therapy for Patients With HomocystinuriaCaused by Cystathionine Beta-Synt ...	A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, ...	Homocystinuria	Drug: Pegtibatinase;Drug: Placebo	Travere Therapeutics, Inc.	NULL	Recruiting	12 Years	65 Years	All	32	Phase 1/Phase 2	United States
8	NCT02404337 (ClinicalTrials.gov)	July 2015	6/3/2015	Betaine METABOLISM OF PATIENTS With Homocystinuria	Betaine METABOLISM OF PATIENTS With Homocystinuria	Homocystinuria	Drug: Betaine	Assistance Publique - Hôpitaux de Paris	NULL	Completed	1 Year	18 Years	All	12	Phase 2	France
9	NCT01793090 (ClinicalTrials.gov)	January 2013	8/2/2013	EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological Impairment	Phase 2, Double-Blind, Placebo Controlled Clinical Trial of EPI-743 in Subjects With Cobalamin C Def ...	Methylmalonic Aciduria and Homocystinuria,Cblc Type;Genetic Disease;Retinopathy	Drug: Epi-743;Other: Placebo supplementation	Bambino Gesù Hospital and Research Institute	Catholic University of the Sacred Heart	Completed	1 Year	20 Years	All	30	Phase 2	Italy
10	NCT01192828 (ClinicalTrials.gov)	January 2010	30/8/2010	Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine	Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine	Homocystinuria	Drug: taurine	University of Colorado, Denver	NULL	Completed	8 Years	49 Years	All	15	Phase 1/Phase 2	United States

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