113. 筋ジストロフィー
[臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151]
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 | EUCTR2007-004819-54-SE (EUCTR) | 19/03/2008 | 31/01/2008 | A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | Belgium;Netherlands;Sweden | ||
2 | EUCTR2007-004819-54-NL (EUCTR) | 22/02/2008 | 07/01/2008 | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: PRO051 Product Code: h51AON23 | Prosensa Holding B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1;Phase 2 | Netherlands;Belgium;Sweden | ||
3 | EUCTR2007-004819-54-BE (EUCTR) | 22/01/2008 | 14/12/2007 | A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | Belgium;Netherlands;Sweden |