RAAV9-CK8-h-µD5 ( 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 : 622 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 168
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-002213-60-GB
(EUCTR)
08/01/201911/12/2018A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophyA randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SGT-001
Product Code: rAAV9-CK8-h-µD5
INN or Proposed INN: SGT-001
Other descriptive name: rAAV9-CK8-h-µD5
Solid Biosciences Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom