113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


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

  
3 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-005023-92-Outside-EU/EEA
(EUCTR)
28/03/2017Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
40Phase 2United States
2EUCTR2016-005002-19-Outside-EU/EEA
(EUCTR)
28/03/2017Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
160Phase 3United States
3EUCTR2016-005024-28-Outside-EU/EEA
(EUCTR)
28/03/2017Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
24Phase 2United States