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

  
2 trials found
No.TrialIDDate_
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agemin
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PhaseCountries
1NCT05154890
(ClinicalTrials.gov)
May 13, 202120/8/2021A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS DeficiencyA Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177Homocystinuria Due to Cystathionine Beta-Synthase DeficiencyDrug: PegtarviliaseAeglea BiotherapeuticsNULLRecruiting12 YearsN/AAll36Phase 1/Phase 2United States;Australia;United Kingdom
2EUCTR2019-004791-19-GB
(EUCTR)
11/05/202014/01/2020A Phase 1/2 Study of ACN00177 in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) DeficiencyA Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 - SA notification 100D New site and PI Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency
MedDRA version: 20.0;Level: PT;Classification code 10020365;Term: Homocystinuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Code: ACN00177Aeglea Biotherapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
25Phase 1United Kingdom