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Exondys 51    (DrugBank: -)

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
113筋ジストロフィー9

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.
9 / 567 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-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
2EUCTR2019-000337-39-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
3EUCTR2016-000951-29-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
4EUCTR2016-000951-29-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
5EUCTR2016-005000-26-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
6EUCTR2016-005023-92-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
7EUCTR2016-005001-39-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
8EUCTR2016-005024-28-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
9EUCTR2016-005002-19-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

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