113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 622 / 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 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 | EUCTR2011-005042-35-GB (EUCTR) | 23/08/2013 | 18/03/2013 | A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-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: BMN 053 Product Code: BMN 053 INN or Proposed INN: PS524 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 45 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 2 | EUCTR2011-005042-35-BE (EUCTR) | 26/06/2013 | 21/03/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A 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. | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 3 | EUCTR2011-005042-35-IT (EUCTR) | 20/06/2013 | 22/03/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 4 | EUCTR2011-005042-35-NL (EUCTR) | 13/06/2013 | 15/08/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 5 | NCT01957059 (ClinicalTrials.gov) | June 2013 | 2/7/2013 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 18 Years | Male | 9 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom |
| 6 | EUCTR2011-005042-35-FR (EUCTR) | 18/09/2015 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A 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 BV | NULL | NA | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan |