113. Muscular dystrophy
567 clinical trials,   442 drugs   (DrugBank: 93 drugs),   55 drug target genes,   151 drug target pathways
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.
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | EUCTR2014-005296-81-BE (EUCTR) | 02/05/2016 | 19/11/2015 | A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability. | A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study | Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: Drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 104 | Phase 3 | United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden | ||
2 | EUCTR2015-001955-54-DE (EUCTR) | 13/01/2016 | 18/08/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan | ||
3 | EUCTR2015-001955-54-BE (EUCTR) | 06/01/2016 | 21/09/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan | ||
4 | EUCTR2015-001955-54-ES (EUCTR) | 24/11/2015 | 05/10/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 220 | Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan |