3. Spinal muscular atrophy
179 clinical trials, 102 drugs (DrugBank: 26 drugs), 52 drug target genes, 78 drug target pathways
Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
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 | EUCTR2019-002663-10-GB (EUCTR) | 17/12/2020 | 01/09/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal MuscularAtrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nursinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: Nursinersen Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany | ||
| 2 | EUCTR2019-002663-10-ES (EUCTR) | 06/05/2020 | 10/05/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 3 | EUCTR2019-002663-10-HU (EUCTR) | 23/04/2020 | 05/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 4 | EUCTR2019-002663-10-LV (EUCTR) | 27/03/2020 | 12/02/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2;Phase 3 | United States;Saudi Arabia;Estonia;Spain;Ireland;Lebanon;Russian Federation;Chile;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | ||
| 5 | EUCTR2019-002663-10-IE (EUCTR) | 04/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;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 |
| 6 | EUCTR2019-002663-10-PL (EUCTR) | 11/03/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Taiwan;Saudi Arabia;Estonia;Spain;Lebanon;Ireland;Turkey;Russian Federation;Chile;Israel;Colombia;Italy;Hungary;Mexico;Canada;Argentina;Poland;Brazil;Australia;Latvia;Korea, Republic of | |||
| 7 | EUCTR2019-002663-10-GR (EUCTR) | 02/11/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | Canada;Argentina;Brazil;Poland;Germany;United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico | |||
| 8 | EUCTR2019-002663-10-DE (EUCTR) | 23/10/2020 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM | Biogen Idec Research Limited | NULL | NA | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Estonia;Saudi Arabia;Taiwan;Greece;Spain;Ireland;Lebanon;Israel;Chile;Russian Federation;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany |