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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2018-001762-42-NL (EUCTR) | 20/07/2020 | 21/07/2020 | A 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 152 | Phase 3 | United 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 | ||
| 2 | NCT03992430 (ClinicalTrials.gov) | July 13, 2020 | 18/6/2019 | A 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 Skipping | Muscular Dystrophy, Duchenne | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Recruiting | 7 Years | 13 Years | Male | 152 | Phase 3 | United States;Canada;Korea, Republic of;Taiwan |
| 3 | EUCTR2018-001762-42-SE (EUCTR) | 27/04/2020 | 26/09/2019 | A 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: 20.0;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. | NULL | Not Recruiting | Female: no Male: yes | 152 | Phase 3 | United 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 | ||
| 4 | EUCTR2018-001762-42-IE (EUCTR) | 03/04/2020 | 01/07/2019 | A 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: 20.0;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. | NULL | Not Recruiting | Female: no Male: yes | 122 | Phase 3 | United 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 | ||
| 5 | NCT04179409 (ClinicalTrials.gov) | February 18, 2020 | 12/8/2019 | A 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 Duplications | A 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 Duplications | Duchenne Muscular Dystrophy | Drug: Casimersen;Drug: Eteplirsen;Drug: Golodirsen | Kevin Flanigan | Sarepta Therapeutics, Inc. | Enrolling by invitation | 6 Months | N/A | Male | 6 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | EUCTR2018-001762-42-DK (EUCTR) | 18/12/2019 | 30/10/2019 | A 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: 20.0;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. | NULL | Not Recruiting | Female: no Male: yes | 122 | Phase 3 | United 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 | ||
| 7 | EUCTR2018-001762-42-ES (EUCTR) | 29/10/2019 | 09/08/2019 | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 152 | Phase 3 | Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;Korea, Republic of | |||
| 8 | EUCTR2018-001762-42-GB (EUCTR) | 17/09/2019 | 04/07/2019 | A 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: 20.0;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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 122 | Phase 3 | United 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 | ||
| 9 | EUCTR2019-000337-39-GB (EUCTR) | 28/06/2019 | 23/04/2019 | A research study of a new investigational medicinal product for thetreatment of Duchenne Muscular Dystrophy patients who have completedstudy 4658-102 | An 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, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||
| 10 | NCT03985878 (ClinicalTrials.gov) | June 26, 2019 | 11/6/2019 | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy | 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 | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Enrolling by invitation | 2 Years | 5 Years | Male | 15 | Phase 2 | Belgium;France;Italy;United Kingdom |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2019-000337-39-BE (EUCTR) | 03/06/2019 | 08/04/2019 | 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] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||
| 12 | NCT03218995 (ClinicalTrials.gov) | August 16, 2017 | 9/7/2017 | Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping | An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Active, not recruiting | 6 Months | 48 Months | Male | 12 | Phase 2 | Belgium;France;Italy;United Kingdom;Germany |
| 13 | EUCTR2016-000951-29-DE (EUCTR) | 13/07/2017 | 13/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An 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. | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 2 | France;Belgium;Germany;Italy;United Kingdom | |||
| 14 | EUCTR2016-000951-29-GB (EUCTR) | 22/06/2017 | 04/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;Germany;Italy;United Kingdom | ||
| 15 | EUCTR2016-000951-29-IT (EUCTR) | 13/06/2017 | 07/02/2018 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 2 | France;Belgium;Germany;United Kingdom;Italy | ||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | EUCTR2016-000951-29-FR (EUCTR) | 01/06/2017 | 17/07/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An 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, Inc | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1 | France;Belgium;Germany;Italy;United Kingdom | ||
| 17 | EUCTR2016-000951-29-BE (EUCTR) | 29/05/2017 | 11/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An 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 | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;Germany;Italy;United Kingdom | ||
| 18 | NCT02420379 (ClinicalTrials.gov) | June 30, 2015 | 10/4/2015 | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 4 Years | 6 Years | Male | 33 | Phase 2 | United States |
| 19 | NCT02255552 (ClinicalTrials.gov) | November 17, 2014 | 25/9/2014 | Study of Eteplirsen in DMD Patients | An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 16 Years | Male | 109 | Phase 3 | United States |
| 20 | NCT02286947 (ClinicalTrials.gov) | November 2014 | 30/10/2014 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 21 Years | Male | 24 | Phase 2 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT01540409 (ClinicalTrials.gov) | February 27, 2012 | 23/2/2012 | Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects 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 | Duchenne Muscular Dystrophy (DMD) | Drug: AVI-4658 (Eteplirsen) | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 13 Years | Male | 12 | Phase 2 | United States |
| 22 | NCT01396239 (ClinicalTrials.gov) | July 2011 | 8/7/2011 | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients | A 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 Dystrophy | Duchenne Muscular Dystrophy | Drug: AVI-4658 (Eteplirsen);Other: Placebo | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 13 Years | Male | 12 | Phase 2 | United States |
| 23 | EUCTR2016-005023-92-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | An 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. | NULL | NA | Female: no Male: yes | 40 | Phase 2 | United States | |||
| 24 | EUCTR2019-000337-39-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, Inc | NULL | NA | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||||
| 25 | EUCTR2018-001762-42-DE (EUCTR) | 01/10/2019 | A 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: 20.0;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. | NULL | NA | Female: no Male: yes | 152 | Phase 3 | United 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 | |||
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | EUCTR2016-005000-26-Outside-EU/EEA (EUCTR) | 16/03/2017 | A 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. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 27 | EUCTR2016-005024-28-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | An 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. | NULL | NA | Female: no Male: yes | 24 | Phase 2 | United States | |||
| 28 | EUCTR2016-005002-19-Outside-EU/EEA (EUCTR) | 28/03/2017 | Confirmatory 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. | NULL | NA | Female: no Male: yes | 160 | Phase 3 | United States | |||
| 29 | EUCTR2016-005001-39-Outside-EU/EEA (EUCTR) | 16/03/2017 | A 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. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 30 | EUCTR2018-001762-42-FR (EUCTR) | 08/07/2019 | A 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. | NULL | NA | Female: no Male: yes | 152 | Phase 3 | Taiwan;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 |