113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170

  
17 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2015-002069-52-IE
(EUCTR)
11/12/202021/05/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
2EUCTR2017-004625-32-BG
(EUCTR)
03/12/202028/10/2020A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
150Phase 3United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden
3EUCTR2017-004625-32-SE
(EUCTR)
17/06/202009/08/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 3United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden
4NCT04179409
(ClinicalTrials.gov)
February 18, 202012/8/2019A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD DuplicationsA 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD DuplicationsDuchenne Muscular DystrophyDrug: Casimersen;Drug: Eteplirsen;Drug: GolodirsenKevin FlaniganSarepta Therapeutics, Inc.Active, not recruiting6 MonthsN/AMale3Phase 2United States
5EUCTR2015-002069-52-DK
(EUCTR)
26/11/201925/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
6EUCTR2017-004625-32-FR
(EUCTR)
05/02/201923/07/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 3United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;France;Australia;Bulgaria;Germany
7EUCTR2017-004625-32-BE
(EUCTR)
20/12/201810/09/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
8EUCTR2017-004625-32-ES
(EUCTR)
07/09/201810/09/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
150Phase 3United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
9EUCTR2015-002069-52-BG
(EUCTR)
06/08/201811/06/2018A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden
10EUCTR2017-004625-32-GB
(EUCTR)
26/07/201823/01/2019A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 3United States;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Sweden
11EUCTR2015-002069-52-CZ
(EUCTR)
11/09/201714/03/2017A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
12EUCTR2015-002069-52-BE
(EUCTR)
28/04/201705/09/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Georgia;Norway;Germany;Sweden
13EUCTR2015-002069-52-DE
(EUCTR)
07/02/201724/10/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden
14NCT02500381
(ClinicalTrials.gov)
September 28, 201614/7/2015Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: SRP-4045;Drug: SRP-4053;Drug: PlaceboSarepta Therapeutics, Inc.NULLActive, not recruiting6 Years13 YearsMale229Phase 3United States;Argentina;Australia;Belgium;Bulgaria;Canada;Czechia;Denmark;France;Germany;Greece;Hungary;India;Ireland;Israel;Italy;Korea, Republic of;Mexico;Poland;Russian Federation;Serbia;Spain;Sweden;United Kingdom
15EUCTR2017-004625-32-PL
(EUCTR)
07/11/2019A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
260Phase 3United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;Australia;France;Bulgaria;Germany
16EUCTR2017-004625-32-CZ
(EUCTR)
09/09/2019A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
260 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
17EUCTR2017-004625-32-DE
(EUCTR)
26/07/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
260Phase 3United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden