113. 筋ジストロフィー
[臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2011-005042-35-BE
(EUCTR)
26/06/201321/03/2013A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 18.1;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]
Product Name: BMN053
Product Code: BMN053
INN or Proposed INN: PS524
BioMarin Nederland B.V.NULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
2NCT01957059
(ClinicalTrials.gov)
June 20132/7/2013A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyDrug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing ExtensionBioMarin PharmaceuticalNULLTerminated5 Years18 YearsMale9Phase 1;Phase 2Belgium;France;Italy;Netherlands;United Kingdom