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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2014-005296-81-BE (EUCTR) | 02/05/2016 | 19/11/2015 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 104 | Phase 3 | United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden | ||
| 2 | EUCTR2015-001955-54-DE (EUCTR) | 13/01/2016 | 18/08/2015 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;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 | ||
| 3 | EUCTR2015-001955-54-BE (EUCTR) | 06/01/2016 | 21/09/2015 | A 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. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;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 | ||
| 4 | EUCTR2015-001955-54-NL (EUCTR) | 08/12/2015 | 09/07/2015 | A 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 BV | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;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 | ||
| 5 | NCT02636686 (ClinicalTrials.gov) | November 27, 2015 | 9/12/2015 | Extension Study of Drisapersen in DMD Subjects | An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | No longer available | 5 Years | 80 Years | Male | United 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. | 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 |
| 6 | EUCTR2015-001955-54-ES (EUCTR) | 24/11/2015 | 05/10/2015 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 220 | Portugal;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 | |||
| 7 | NCT01890798 (ClinicalTrials.gov) | January 2014 | 27/6/2013 | Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol | A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study | Muscular Dystrophies | Drug: Drisapersen | GlaxoSmithKline | NULL | Withdrawn | 5 Years | N/A | Male | 0 | Phase 3 | NULL |
| 8 | NCT01803412 (ClinicalTrials.gov) | May 1, 2013 | 28/2/2013 | A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects | An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy. | Muscular Dystrophies | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | N/A | Male | 53 | Phase 3 | United States;Canada |
| 9 | 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 | ||
| 10 | NCT01910649 (ClinicalTrials.gov) | March 2008 | 2/8/2012 | A 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 Administration | 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 | Muscular Dystrophies | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 16 Years | Male | 12 | Phase 2 | NULL |
| 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 |
| 11 | 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 |