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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
25 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2017-004625-32-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;Australia;Bulgaria;Germany;Sweden
2EUCTR2017-004625-32-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;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
3EUCTR2015-002069-52-NO
(EUCTR)
07/04/202030/09/2019A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Germany;Norway;Sweden
4EUCTR2015-002069-52-GR
(EUCTR)
29/11/201911/09/2019A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
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]
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Denmark;Australia;Bulgaria;Norway;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2015-002069-52-PL
(EUCTR)
11/12/201813/09/2018A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
7EUCTR2017-004625-32-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
8EUCTR2015-002069-52-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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Norway;Germany;Sweden;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy
9EUCTR2017-004625-32-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
10EUCTR2015-002069-52-CZ
(EUCTR)
11/09/201714/03/2017A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2015-002069-52-SE
(EUCTR)
26/06/201720/09/2016A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;United States;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
12EUCTR2015-002069-52-ES
(EUCTR)
11/05/201710/03/2017A 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: 19.1;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United States;Canada;Spain;Israel;Germany;United Kingdom
13EUCTR2015-002069-52-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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Spain;Ukraine;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Denmark;Australia;Bulgaria;Germany;Sweden
14EUCTR2015-002069-52-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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
15EUCTR2015-002069-52-GB
(EUCTR)
23/01/201705/09/2016A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
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;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2015-002069-52-IT
(EUCTR)
19/01/201707/02/2018A 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
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 45
Product Name: SRP-4053
Product Code: SRP-4053
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 53
SAREPTA THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3France;Czech Republic;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
17NCT02500381
(ClinicalTrials.gov)
September 28, 201614/7/2015Study of SRP-4045 and SRP-4053 in DMD PatientsA 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.NULLRecruiting7 Years13 YearsMale222Phase 3United States;Australia;Belgium;Bulgaria;Canada;Czechia;France;Germany;Greece;Hungary;Israel;Italy;Poland;Spain;Sweden;United Kingdom
18EUCTR2014-002008-25-FR
(EUCTR)
06/07/201611/01/2017A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 19.1;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]
Sarepta Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
48Phase 1;Phase 2France;Italy;United Kingdom
19NCT02310906
(ClinicalTrials.gov)
January 13, 20153/12/2014Phase I/II Study of SRP-4053 in DMD PatientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 SkippingDuchenne Muscular DystrophyDrug: Placebo;Drug: SRP-4053Sarepta Therapeutics, Inc.Institut de Myologie, France;Consultants for Research in Imaging and Spectroscopy;Great Ormond Street Hospital for Children NHS Foundation Trust;Catholic University of the Sacred Heart;Royal Holloway University;SYSNAV;University College, London;University of Newcastle Upon-TyneCompleted6 Years15 YearsMale39Phase 1;Phase 2United States;France;Italy;United Kingdom
20EUCTR2014-002008-25-IT
(EUCTR)
13/11/201410/09/2014A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
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: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not Available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 1;Phase 2France;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2014-002008-25-GB
(EUCTR)
10/09/201408/09/2014A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 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]
Sarepta Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
48Phase 1;Phase 2France;United States;Italy;United Kingdom
22EUCTR2017-004625-32-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;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
23EUCTR2015-002069-52-FR
(EUCTR)
12/01/2017A 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: 19.1;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: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
99Phase 3United States;France;Czech Republic;Canada;Spain;Belgium;Germany;United Kingdom
24EUCTR2015-002069-52-IE
(EUCTR)
21/05/2019A 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: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
222Phase 3United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
25EUCTR2017-004625-32-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;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Bulgaria;Germany;Sweden