Ps524    (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4

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.
4 / 567 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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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
2EUCTR2011-005042-35-IT
(EUCTR)
20/06/201322/03/2013A 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: 15.1;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: PRO053
Product Code: PRO053
INN or Proposed INN: PS524
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
3EUCTR2011-005042-35-NL
(EUCTR)
13/06/201315/08/2013A 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: 16.0;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: PRO053
Product Code: PRO053
INN or Proposed INN: PS524
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
4EUCTR2011-005042-35-FR
(EUCTR)
18/09/2015A 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: 18.0;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: PRO053
Product Code: PRO053
INN or Proposed INN: PS524
Prosensa Therapeutics BVNULLNAFemale: 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