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

  
13 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-004426-24-IT
(EUCTR)
06/07/202015/06/2021Deflazacort 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 PISANANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Italy
2EUCTR2018-004740-36-DK
(EUCTR)
29/10/201925/06/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;France;Canada;Denmark;Russian Federation;Norway;Germany;Sweden
3EUCTR2018-004740-36-DE
(EUCTR)
14/10/201929/04/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden
4EUCTR2018-004740-36-FR
(EUCTR)
19/08/201904/06/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA 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.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;France;Canada;Denmark;Norway;Germany;Sweden
5NCT03783923
(ClinicalTrials.gov)
May 15, 201919/12/2018A 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 DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted18 YearsN/AAll30Phase 3United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden
6NCT02592941
(ClinicalTrials.gov)
March 1, 201929/10/2015Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyAn Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsParexel;Dohmen Life Science ServicesApproved for marketing5 YearsN/AAllUnited States
7NCT03642145
(ClinicalTrials.gov)
October 31, 201810/7/2018A 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 PeriodDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLWithdrawn2 Years4 YearsMale0Phase 3United States
8NCT02295748
(ClinicalTrials.gov)
December 201418/11/2014An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortAn 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 DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted4 YearsN/AMale24Phase 1United States
9NCT02251600
(ClinicalTrials.gov)
December 201422/9/2014A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyA 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 DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted4 Years16 YearsMale24Phase 1United States
10NCT04740554
(ClinicalTrials.gov)
March 1, 20131/2/2021Heart Rate Variability in Individuals With Duchenne Muscular DystrophyCharacterization of Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy - Influence of Corticosteroids and BetablockersDuchenne Muscular DystrophyBehavioral: 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 DevelopingUniversity of Sao PauloNULLCompleted11 Years18 YearsMale40NULL
11EUCTR2010-023744-33-IT
(EUCTR)
26/02/201328/12/2012Duchenne 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 PADOVANULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
300United States;Canada;United Kingdom;Italy
12NCT01603407
(ClinicalTrials.gov)
January 20133/4/2012Finding the Optimum Regimen for Duchenne Muscular DystrophyDuchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid RegimenDuchenne Muscular DystrophyDrug: Prednisone;Drug: DeflazacortUniversity of RochesterNewcastle University;University Medical Center Freiburg;National Institute of Neurological Disorders and Stroke (NINDS)Completed4 Years7 YearsMale196Phase 3United States;Canada;Germany;Italy;United Kingdom
13EUCTR2010-023744-33-GB
(EUCTR)
12/11/201220/09/2012Trial to find best steroid treatment for Duchenne muscular dystrophyDuchenne 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 RochesterNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
300United States;Canada;Germany;Italy;United Kingdom