Phase 1, SQY51 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
113筋ジストロフィー1

113. 筋ジストロフィー


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05753462
(ClinicalTrials.gov)
March 20236/2/2023Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular DystrophyPhase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Phase 1, SQY51;Drug: Phase 2a, SQY51 (cohort 1);Drug: Phase 2a, SQY51 (cohort 2);Drug: Phase 2a, SQY51 (cohort 3)Sqy TherapeuticsBiotrialRecruiting6 YearsN/AMale12Phase 1/Phase 2France