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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
11 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2014-005296-81-BE
(EUCTR)
02/05/201619/11/2015A 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.NULLNot RecruitingFemale: no
Male: yes
104Phase 3United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden
2EUCTR2015-001955-54-DE
(EUCTR)
13/01/201618/08/2015A 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;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
3EUCTR2015-001955-54-BE
(EUCTR)
06/01/201621/09/2015A 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.NULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;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
4EUCTR2015-001955-54-NL
(EUCTR)
08/12/201509/07/2015A 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
BioMarin Nederland BVNULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;United States;Belarus;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;Sweden
5NCT02636686
(ClinicalTrials.gov)
November 27, 20159/12/2015Extension Study of Drisapersen in DMD SubjectsAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyDrug: DrisapersenBioMarin PharmaceuticalNULLNo longer available5 Years80 YearsMaleUnited States;Argentina;Australia;Belgium;Bulgaria;Czechia;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom;Czech Republic
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-001955-54-ES
(EUCTR)
24/11/201505/10/2015A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
220Portugal;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
7NCT01890798
(ClinicalTrials.gov)
January 201427/6/2013Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment ProtocolA Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen StudyMuscular DystrophiesDrug: DrisapersenGlaxoSmithKlineNULLWithdrawn5 YearsN/AMale0Phase 3NULL
8NCT01803412
(ClinicalTrials.gov)
May 1, 201328/2/2013A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian SubjectsAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.Muscular DystrophiesDrug: DrisapersenBioMarin PharmaceuticalNULLTerminated5 YearsN/AMale53Phase 3United States;Canada
9EUCTR2007-004819-54-SE
(EUCTR)
19/03/200831/01/2008A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne DiseaseA phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration Duchenne Muscular Dystrophy
MedDRA version: 17.0;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: PRO051
INN or Proposed INN: PRO051
Product Name: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
12Phase 1;Phase 2Belgium;Netherlands;Sweden
10NCT01910649
(ClinicalTrials.gov)
March 20082/8/2012A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of AdministrationA Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of AdministrationMuscular DystrophiesDrug: DrisapersenBioMarin PharmaceuticalNULLTerminated5 Years16 YearsMale12Phase 2NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2007-004819-54-BE
(EUCTR)
22/01/200814/12/2007A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne DiseaseA phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration Duchenne Muscular Dystrophy
MedDRA version: 17.1;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: PRO051
INN or Proposed INN: PRO051
Product Name: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
12Phase 1;Phase 2Belgium;Netherlands;Sweden