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 | EUCTR2019-004426-24-IT (EUCTR) | 06/07/2020 | 15/06/2021 | Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers. | Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers. - TREAT-LMNA | Congenital Muscolar distrophy LMNA related MedDRA version: 20.0;Level: LLT;Classification code 10003718;Term: Atrophy skeletal muscle;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Deflazacort Product Code: [Deflazacort] INN or Proposed INN: deflazacort Trade Name: Lucen Product Name: Lucen Product Code: [-] INN or Proposed INN: esomeprazolo Other descriptive name: esomeprazole | AZIENDA OSPEDALIERO-UNIVERSITARIA PISANA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Italy | ||
| 2 | EUCTR2018-004740-36-DK (EUCTR) | 29/10/2019 | 25/06/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;France;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 3 | EUCTR2018-004740-36-DE (EUCTR) | 14/10/2019 | 29/04/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 4 | EUCTR2018-004740-36-FR (EUCTR) | 19/08/2019 | 04/06/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;France;Canada;Denmark;Norway;Germany;Sweden | ||
| 5 | NCT03783923 (ClinicalTrials.gov) | May 15, 2019 | 19/12/2018 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | A Multicenter Open Label Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | Limb-Girdle Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 18 Years | N/A | All | 30 | Phase 3 | United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden |
| 6 | NCT02592941 (ClinicalTrials.gov) | March 1, 2019 | 29/10/2015 | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy | An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | Parexel;Dohmen Life Science Services | Approved for marketing | 5 Years | N/A | All | United States | ||
| 7 | NCT03642145 (ClinicalTrials.gov) | October 31, 2018 | 10/7/2018 | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) | A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged =2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension Period | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Withdrawn | 2 Years | 4 Years | Male | 0 | Phase 3 | United States |
| 8 | NCT02251600 (ClinicalTrials.gov) | December 2014 | 22/9/2014 | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | 16 Years | Male | 24 | Phase 1 | United States |
| 9 | NCT02295748 (ClinicalTrials.gov) | December 2014 | 18/11/2014 | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort | An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | N/A | Male | 24 | Phase 1 | United States |
| 10 | NCT04740554 (ClinicalTrials.gov) | March 1, 2013 | 1/2/2021 | Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy | Characterization of Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy - Influence of Corticosteroids and Betablockers | Duchenne Muscular Dystrophy | Behavioral: Duchenne Muscular Dystrophy group with Deflazacort;Behavioral: Duchenne Muscular Dystrophy group with Prednisone/Predisolone;Behavioral: Duchenne Muscular Dystrophy group without Corticosteroids therapy;Behavioral: Control Group Typically Developing | University of Sao Paulo | NULL | Completed | 11 Years | 18 Years | Male | 40 | NULL | |
| 11 | EUCTR2010-023744-33-IT (EUCTR) | 26/02/2013 | 28/12/2012 | Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen. | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen. - FOR-DMD | Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids. 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] | Trade Name: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DEFLAN*10CPR 6MG INN or Proposed INN: DEFLAZACORT | AZIENDA OSPEDALIERA DI PADOVA | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;United Kingdom;Italy | |||
| 12 | NCT01603407 (ClinicalTrials.gov) | January 2013 | 3/4/2012 | Finding the Optimum Regimen for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen | Duchenne Muscular Dystrophy | Drug: Prednisone;Drug: Deflazacort | University of Rochester | Newcastle University;University Medical Center Freiburg;National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 4 Years | 7 Years | Male | 196 | Phase 3 | United States;Canada;Germany;Italy;United Kingdom |
| 13 | EUCTR2010-023744-33-GB (EUCTR) | 12/11/2012 | 20/09/2012 | Trial to find best steroid treatment for Duchenne muscular dystrophy | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen - FOR-DMD | Duchenne muscular dystrophy (DMD) 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] | Trade Name: Deflazacort Product Name: Deflazacort 6 mg Tablets INN or Proposed INN: Deflazacort Trade Name: Prednisone Product Name: Prednisone 5 mg tablets INN or Proposed INN: Prednisone | University of Rochester | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;Germany;Italy;United Kingdom |