113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 622 / Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168
| 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02958202 (ClinicalTrials.gov) | April 2016 | 27/10/2016 | Extension 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 Dystrophy | Duchenne Muscular Dystrophy | Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | N/A | Male | 7 | Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 2 | EUCTR2015-003681-87-BE (EUCTR) | 12/01/2016 | 16/11/2015 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 50 | Phase 2 | United States;Belgium;Netherlands;Italy;Sweden | ||
| 3 | EUCTR2013-003605-26-NL (EUCTR) | 13/04/2015 | 19/11/2014 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 4 | EUCTR2013-003605-26-BE (EUCTR) | 23/01/2015 | 04/12/2014 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 5 | EUCTR2013-003605-26-IT (EUCTR) | 02/12/2014 | 11/09/2014 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Italy;Sweden | ||
| 6 | NCT02329769 (ClinicalTrials.gov) | December 2014 | 22/12/2014 | Open 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 Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kg | BioMarin Pharmaceutical | NULL | Terminated | 9 Years | 20 Years | Male | 15 | Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 7 | EUCTR2013-003605-26-SE (EUCTR) | 26/11/2014 | 26/09/2014 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 8 | EUCTR2009-013762-63-NL (EUCTR) | 16/04/2010 | 25/08/2009 | A 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 | A 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 Dystrophy | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188 | Prosensa Therapeutics B.V | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 9 | EUCTR2009-013762-63-IT (EUCTR) | 25/02/2010 | 27/04/2010 | A 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 | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 10 | EUCTR2009-013762-63-SE (EUCTR) | 19/02/2010 | 03/11/2009 | A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne Disease | 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 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.V | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 11 | EUCTR2009-013762-63-BE (EUCTR) | 09/12/2009 | 03/11/2009 | A 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 | A 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 Dystrophy | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188 | Prosensa Therapeutics B.V | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 1;Phase 2 | Belgium;Netherlands;Italy;Sweden | ||
| 12 | NCT01037309 (ClinicalTrials.gov) | December 2009 | 21/12/2009 | Phase 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 Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC;Drug: PRO044 IV | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 16 Years | Male | 18 | Phase 1/Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 13 | EUCTR2015-003681-87-IT (EUCTR) | 19/01/2021 | A study to assess the effect of BMN 044 in subjects with Duchennemuscular 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 - NA | Duchenne muscular dystrophy MedDRA version: 20.0;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: BMN 044 Product Code: BMN 044 Other descriptive name: PS188 Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 Other descriptive name: PS188 | BIOMARIN PHARMACEUTICAL INC. | NULL | NA | Female: no Male: yes | 50 | Phase 2 | Netherlands;Italy;Sweden;United States;Belgium |