251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details

臨床試験数 : 48 / 薬物数 : 52 - (DrugBank : 13) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 29

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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	EUCTR2021-001081-38-ES (EUCTR)	12/01/2022	31/08/2021	A Multi-dose Study of an Investigational Treatment to Evaluate Safety, Tolerability, and Ability to ...	Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Stu ...	Ornithine transcarbamylase deficiency MedDRA version: 21.1;Level: LLT;Classification code 10013373;T ...	Product Name: ARCT-810 Product Code: ARCT-810 INN or Proposed INN: Not assigned Other descriptive name: ...	Arcturus Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	24	Phase 2	Belgium;Spain;United Kingdom
2	EUCTR2021-000824-36-ES (EUCTR)	09/12/2021	11/06/2021	An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in ...	An open-label, controlled, multi-site, Phase I clinical trial to assess the ureagenesis capacity in ...	Subject has a genetically confirmed diagnosis of any of the following urea cycle disorders: ASS, CPS ...	Product Name: 15N ammonium chloride (15NH4Cl) INN or Proposed INN: Ammonium (15N) chloride Other descr ...	Unicyte AG	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	30	Phase 1	Portugal;Czechia;Saudi Arabia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Be ...
3	NCT05076318 (ClinicalTrials.gov)	March 1, 2021	26/8/2021	Dysregulated Urea-synthesis at Terminal Uremia	Dysregulated Urea-synthesis at Terminal Uremia	Urea Cycle Disorder;Uremia	Other: Aminoacid, alanine-infusion	University of Aarhus	NULL	Recruiting	18 Years	N/A	All	16	N/A	Denmark
4	NCT04442347 (ClinicalTrials.gov)	November 3, 2020	19/6/2020	Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Trans ...	A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety ...	Ornithine Transcarbamylase Deficiency	Biological: ARCT-810;Other: Placebo	Arcturus Therapeutics, Inc.	NULL	Recruiting	18 Years	N/A	All	12	Phase 1	United States
5	EUCTR2018-000156-18-FR (EUCTR)	02/11/2020	31/01/2020	A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine T ...	A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Sero ...	Ornithine transcarbamylase deficiency MedDRA version: 20.0;Level: LLT;Classification code 10071107;T ...	Product Name: DTX301 INN or Proposed INN: Not yet assigned Other descriptive name: DTX301	Ultragenyx Pharmaceutical, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	18	Phase 1;Phase 2	United States;France;Canada;Spain;United Kingdom
6	NCT04416126 (ClinicalTrials.gov)	June 1, 2020	28/5/2020	Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects	A Phase 1 Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, ...	Ornithine Transcarbamylase Deficiency	Biological: ARCT-810;Other: Placebo	Arcturus Therapeutics, Inc.	NULL	Recruiting	18 Years	65 Years	All	30	Phase 1	New Zealand
7	EUCTR2018-004842-40-FR (EUCTR)	14/01/2020	18/11/2019	International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ...	A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cyc ...	Subjects with a Urea Cycle Disorderwith Inadequate Control on Standard of Care MedDRA version: 20.1; ...	Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195	Kaleido Biosciences	NULL	Not Recruiting			Female: yes Male: yes	29	Phase 2	United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United King ...
8	NCT03767270 (ClinicalTrials.gov)	December 2019	28/11/2018	Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With ...	A Phase 1/2 Single Ascending Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and P ...	Ornithine Transcarbamylase Deficiency	Biological: MRT5201;Other: Placebo	Translate Bio, Inc.	NULL	Withdrawn	18 Years	N/A	All	0	Phase 1/Phase 2	NULL
9	EUCTR2018-004842-40-DE (EUCTR)	25/11/2019	04/06/2019	International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ...	A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycl ...	Subjects with a Urea Cycle Disorderwith Inadequate Control on Standard of Care MedDRA version: 20.1; ...	Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195	Kaleido Biosciences	NULL	Not Recruiting			Female: yes Male: yes	29	Phase 2	United States;France;Saudi Arabia;Mexico;Canada;Spain;Belgium;Turkey;Netherlands;Germany;United King ...
10	EUCTR2018-004842-40-ES (EUCTR)	06/11/2019	11/06/2019	International multicenter study to test the efficacy and safety of a new drug, KB195, in subjects wi ...	A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cyc ...	Subjects with a Urea Cycle Disorderwith Inadequate Control on Standard of Care MedDRA version: 20.1; ...	Product Name: KB195 Product Code: KB195 INN or Proposed INN: KB195	Kaleido Biosciences	NULL	Not Recruiting			Female: yes Male: yes	29	Phase 2	United States;France;Saudi Arabia;Mexico;Canada;Belgium;Spain;Turkey;Netherlands;Germany;United King ...

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