HMI-103 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
240フェニルケトン尿症1

240. フェニルケトン尿症


臨床試験数 : 143 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05222178
(ClinicalTrials.gov)
June 3, 202221/12/2021Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH DeficiencyA Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH DeficiencyPhenylketonurias;PAH Deficiency;PhenylketonuriaDrug: HMI-103Homology Medicines, IncNULLRecruiting18 Years55 YearsAll9Phase 1United States