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"
30 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-001762-42-NL
(EUCTR)
20/07/202021/07/2020A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne MuscularDystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
152Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Switzerland;France;Mexico;Canada;Poland;Belgium;Denmark;Australia;Norway;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
2NCT03992430
(ClinicalTrials.gov)
July 13, 202018/6/2019A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 SkippingMuscular Dystrophy, DuchenneDrug: EteplirsenSarepta Therapeutics, Inc.NULLRecruiting7 Years13 YearsMale152Phase 3United States;Canada;Korea, Republic of;Taiwan
3EUCTR2018-001762-42-SE
(EUCTR)
27/04/202026/09/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
152Phase 3United States;Taiwan;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Canada;Argentina;Belgium;Peru;Australia;Denmark;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
4EUCTR2018-001762-42-IE
(EUCTR)
03/04/202001/07/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
122Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Switzerland;France;Mexico;Canada;Poland;Belgium;Denmark;Australia;Norway;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
5NCT04179409
(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.Enrolling by invitation6 MonthsN/AMale6Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2018-001762-42-DK
(EUCTR)
18/12/201930/10/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
122Phase 3United States;Taiwan;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
7EUCTR2018-001762-42-ES
(EUCTR)
29/10/201909/08/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;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
152Phase 3Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;Korea, Republic of
8EUCTR2018-001762-42-GB
(EUCTR)
17/09/201904/07/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
122Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;Switzerland;United Kingdom;France;Mexico;Canada;Poland;Belgium;Australia;Denmark;Norway;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
9EUCTR2019-000337-39-GB
(EUCTR)
28/06/201923/04/2019A research study of a new investigational medicinal product for thetreatment of Duchenne Muscular Dystrophy patients who have completedstudy 4658-102An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen inPatients with Duchenne Muscular Dystrophy Who Have Completed Study4658-102 Duchenne Muscular Dystrophy Amenable to Exon 51 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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;United Kingdom
10NCT03985878
(ClinicalTrials.gov)
June 26, 201911/6/2019A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular DystrophyAn Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102Duchenne Muscular DystrophyDrug: EteplirsenSarepta Therapeutics, Inc.NULLEnrolling by invitation2 Years5 YearsMale15Phase 2Belgium;France;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2019-000337-39-BE
(EUCTR)
03/06/201908/04/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;United Kingdom
12NCT03218995
(ClinicalTrials.gov)
August 16, 20179/7/2017Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingAn Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDuchenne Muscular DystrophyDrug: EteplirsenSarepta Therapeutics, Inc.NULLActive, not recruiting6 Months48 MonthsMale12Phase 2Belgium;France;Italy;United Kingdom;Germany
13EUCTR2016-000951-29-DE
(EUCTR)
13/07/201713/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 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
12Phase 2France;Belgium;Germany;Italy;United Kingdom
14EUCTR2016-000951-29-GB
(EUCTR)
22/06/201704/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;Germany;Italy;United Kingdom
15EUCTR2016-000951-29-IT
(EUCTR)
13/06/201707/02/2018A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping - N/A Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
SAREPTA THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
12Phase 2France;Belgium;Germany;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
16EUCTR2016-000951-29-FR
(EUCTR)
01/06/201717/07/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
12Phase 1France;Belgium;Germany;Italy;United Kingdom
17EUCTR2016-000951-29-BE
(EUCTR)
29/05/201711/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;Germany;Italy;United Kingdom
18NCT02420379
(ClinicalTrials.gov)
June 30, 201510/4/2015Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: eteplirsenSarepta Therapeutics, Inc.NULLCompleted4 Years6 YearsMale33Phase 2United States
19NCT02255552
(ClinicalTrials.gov)
November 17, 201425/9/2014Study of Eteplirsen in DMD PatientsAn Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: eteplirsenSarepta Therapeutics, Inc.NULLCompleted7 Years16 YearsMale109Phase 3United States
20NCT02286947
(ClinicalTrials.gov)
November 201430/10/2014Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: EteplirsenSarepta Therapeutics, Inc.NULLCompleted7 Years21 YearsMale24Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT01540409
(ClinicalTrials.gov)
February 27, 201223/2/2012Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyOpen-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201Duchenne Muscular Dystrophy (DMD)Drug: AVI-4658 (Eteplirsen)Sarepta Therapeutics, Inc.NULLCompleted7 Years13 YearsMale12Phase 2United States
22NCT01396239
(ClinicalTrials.gov)
July 20118/7/2011Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsA Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: AVI-4658 (Eteplirsen);Other: PlaceboSarepta Therapeutics, Inc.NULLCompleted7 Years13 YearsMale12Phase 2United States
23EUCTR2018-001762-42-FR
(EUCTR)
08/07/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Sarepta Therapeutics, Inc.NULLNA Female: no
Male: yes
152Phase 3Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;New Zealand;Korea, Republic of
24EUCTR2016-005023-92-Outside-EU/EEA
(EUCTR)
28/03/2017Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystropy Amenable to Exon 51 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]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
40Phase 2United States
25EUCTR2016-005000-26-Outside-EU/EEA
(EUCTR)
16/03/2017A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy.A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability, and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administered Over 28 Weeks in the Treatment of Ambulant Subjects with Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: Exondys 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
12Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2016-005024-28-Outside-EU/EEA
(EUCTR)
28/03/2017Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
24Phase 2United States
27EUCTR2016-005001-39-Outside-EU/EEA
(EUCTR)
16/03/2017A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy.Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201 Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: Exondys 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
12Phase 2United States
28EUCTR2016-005002-19-Outside-EU/EEA
(EUCTR)
28/03/2017Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI)An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 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]
Trade Name: EXONDYS 51
Product Name: Eteplirsen Injection
Product Code: AVI-4658
INN or Proposed INN: AVI-4658
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
160Phase 3United States
29EUCTR2018-001762-42-DE
(EUCTR)
01/10/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
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: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNAFemale: no
Male: yes
152Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Switzerland;France;Mexico;Canada;Poland;Belgium;Denmark;Australia;Norway;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
30EUCTR2019-000337-39-FR
(EUCTR)
05/04/2019 A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 Duchenne Muscular Dystrophy Amenable to Exon 51 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, IncNULLNA Female: no
Male: yes
15Phase 2France;Belgium;United Kingdom