3. 脊髄性筋萎縮症
［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］
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 | EUCTR2015-001870-16-GB (EUCTR) | 04/10/2016 | 29/10/2015 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 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 | EUCTR2015-001870-16-BE (EUCTR) | 29/07/2016 | 23/05/2016 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 7 | EUCTR2015-001870-16-SE (EUCTR) | 26/05/2016 | 22/01/2016 | A study for participants with Spinal Muscular Atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Sweden;Korea, Republic of | ||
| 8 | EUCTR2015-001870-16-DE (EUCTR) | 08/03/2016 | 28/10/2015 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;Italy;United Kingdom;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | ||
| 9 | EUCTR2014-003657-33-DE (EUCTR) | 21/09/2015 | 05/05/2015 | A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA). | A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE | Spinal Muscular Atrophy 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United States;Germany;United Kingdom | ||
| 10 | EUCTR2014-002098-12-DE (EUCTR) | 05/08/2015 | 09/04/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE | Spinal Muscular Atrophy (SMA) 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: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Canada;Turkey;Australia;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 |
| 11 | EUCTR2014-002098-12-GB (EUCTR) | 10/06/2015 | 30/03/2015 | A Study of Multiple Doses of ISIS ISIS 396443 Delivered to Infants withGenetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE | Spinal Muscular Atrophy (SMA) 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 Product Code: ISIS 396443 (BIIB058) INN or Proposed INN: Nusinersen Other descriptive name: ISIS 396443 (BIIB058) | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 25 | Phase 2 | United States;Qatar;Taiwan;Turkey;Australia;Germany;Italy;United Kingdom | ||
| 12 | 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 | |||
| 13 | 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 | |||
| 14 | 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 | |||
| 15 | 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 |