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"
12 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02958202
(ClinicalTrials.gov)
April 201627/10/2016Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kgBioMarin PharmaceuticalNULLTerminated5 YearsN/AMale7Phase 2Belgium;Italy;Netherlands;Sweden
2EUCTR2015-003681-87-BE
(EUCTR)
12/01/201616/11/2015A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study)A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy
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: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Product Name: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
50Phase 2United States;Belgium;Netherlands;Italy;Sweden
3EUCTR2013-003605-26-NL
(EUCTR)
13/04/201519/11/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy 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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
4EUCTR2013-003605-26-BE
(EUCTR)
23/01/201504/12/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
5EUCTR2013-003605-26-IT
(EUCTR)
02/12/201411/09/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy 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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02329769
(ClinicalTrials.gov)
December 201422/12/2014Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kgBioMarin PharmaceuticalNULLTerminated9 Years20 YearsMale15Phase 2Belgium;Italy;Netherlands;Sweden
7EUCTR2013-003605-26-SE
(EUCTR)
26/11/201426/09/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
BioMarin Nederland B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
8EUCTR2009-013762-63-NL
(EUCTR)
16/04/201025/08/2009A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy Duchenne Muscular DystrophyProduct Name: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188
Prosensa Therapeutics B.VNULLNot RecruitingFemale: no
Male: yes
18Phase 1;Phase 2aBelgium;Netherlands;Italy;Sweden
9EUCTR2009-013762-63-IT
(EUCTR)
25/02/201027/04/2010A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - NDA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND Duchenne Muscular Dystrophy
MedDRA version: 9.1;Level: LLT;Classification code 10013801
Product Name: PRO044
Product Code: PRO044
PROSENSA THERAPEUTICS BVNULLNot RecruitingFemale: no
Male: yes
18Phase 1;Phase 2aBelgium;Netherlands;Italy;Sweden
10EUCTR2009-013762-63-SE
(EUCTR)
19/02/201003/11/2009A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne DiseaseA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.1;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: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188
Prosensa Therapeutics B.VNULLNot RecruitingFemale: no
Male: yes
24Phase 1;Phase 2aBelgium;Netherlands;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2009-013762-63-BE
(EUCTR)
09/12/200903/11/2009A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy Duchenne Muscular DystrophyProduct Name: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188
Prosensa Therapeutics B.VNULLNot RecruitingFemale: no
Male: yes
24Phase 1;Phase 2Belgium;Netherlands;Italy;Sweden
12NCT01037309
(ClinicalTrials.gov)
December 200921/12/2009Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PRO044 SC;Drug: PRO044 IVBioMarin PharmaceuticalNULLCompleted5 Years16 YearsMale18Phase 1;Phase 2Belgium;Italy;Netherlands;Sweden