3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
Showing 1 to 10 of 55 diseases
| 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 | NCT05067790 (ClinicalTrials.gov) | January 21, 2022 | 24/9/2021 | A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam A Study to Evaluate Higher Dose (HD) Nusinersen(BIIB058) in Participants With Spinal Muscular Atroph ... | A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam A Phase 3b Study to Evaluate Higher Dose Nusinersen(BIIB058) in Patients With Spinal Muscular Atroph ... | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | NULL | Recruiting | 5 Years | 39 Years | All | 135 | Phase 3 | United States |
| 2 | JPRN-jRCT2061200040 | 03/08/2021 | 25/12/2020 | 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 Escalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal ... | Spinal Muscular Atrophy | - Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersen intrathecally on Days 1, 15 and 29 followed by maintenance doses of 28 mg on Days 149 and 269. - Active Comparator Part B: Participants with infantile- or later-onset SMA will receive loading doses of 12 mg of nusinersen intrathecally on Days 1, 15, 29, and 64 followed by maintenance doses of 12 mg on Days 183 and 279. Sham procedure will be administered on Day 135. - 50/28 mg Group Part B: Participants with infantile- or later-onset SMA will receive loading doses of 50 mg of nusinersen intrathecally on Days 1 and 15 followed by maintenance doses of 28 mg on Days 135 and 279. Sham procedure will be administered on Days 29, 64 and 183. - Part C: Participants who have been receiving the approved dose for at least 1 year prior to entry in this study, will receive a single bolus dose of 50 mg of nusinersen intrathecally on Day 1 (4 months after their most recent maintenance dose) followed by maintenance doses of 28 mg on Days 121 and 241. - Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersenintrath ... | Berger Zdenek | NULL | Recruiting | Not applicable | Not applicable | Both | 152 | Phase 2-3 | Spain;Taiwan;United States;Japan;Estonia;Ireland;Latvia;Colombia |
| 3 | EUCTR2020-004708-32-HU (EUCTR) | 15/06/2021 | 17/04/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen Extension Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy Who Previously P ... | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD A Long-Term Extension Study of Nusinersen(BIIB058) Administered at Higher Doses in Participants With ... | 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] Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mu ... | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descr ... | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 3 | United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany United States;Taiwan;Saudi Arabia;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chi ... | ||
| 4 | EUCTR2020-003492-18-DE (EUCTR) | 07/06/2021 | 18/03/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec A Study of NusinersenAmong Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abepa ... | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy A Phase 4 Study of Nusinersen(BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onas ... | 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] Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mu ... | Trade Name: Spinraza 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 | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 5 | EUCTR2020-003492-18-ES (EUCTR) | 02/06/2021 | 01/09/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec A Study of NusinersenAmong Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abepa ... | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy A Phase 4 Study of Nusinersen(BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onas ... | 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] Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mu ... | Trade Name: Spinraza 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 | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 6 | EUCTR2020-004708-32-ES (EUCTR) | 05/05/2021 | 09/02/2021 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen Extension Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy Who Previously P ... | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen - ONWARD A Long-Term Extension Study of Nusinersen(BIIB058) Administered at Higher Doses in Participants With ... | 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] Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mu ... | Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descriptive name: NUSINERSEN SODIUM Product Name: Nusinersen Product Code: ISIS 396443, BIIB058 INN or Proposed INN: NUSINERSEN Other descr ... | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 3 | United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chi ... | ||
| 7 | EUCTR2020-003492-18-IT (EUCTR) | 26/04/2021 | 08/06/2021 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec A Study of NusinersenAmong Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abepa ... | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy A Phase 4 Study of Nusinersen(BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onas ... | Muscular Atrophy, Spinal;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: . Product Code: [.] INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSEN SODIUM Trade Name: Spinraza Product Name: . Product Code: [.] INN or Proposed INN: Nusinersen Other descriptive ... | BIOGEN IDEC RESEARCH LIMITED | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 4 | United States;Spain;Israel;Germany;Italy | ||
| 8 | NCT04729907 (ClinicalTrials.gov) | April 19, 2021 | 11/12/2020 | Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen Extension Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy Who Previously P ... | A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen A Long-Term Extension Study of Nusinersen(BIIB058) Administered at Higher Doses in Participants With ... | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Enrolling by invitation | N/A | N/A | All | 172 | Phase 3 | United States;Brazil;Colombia;Estonia;Hungary;Spain;Taiwan;Argentina;Australia;Canada;Chile;France;Germany;Greece;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Turkey;United Kingdom United States;Brazil;Colombia;Estonia;Hungary;Spain;Taiwan;Argentina;Australia;Canada;Chile;France;G ... |
| 9 | EUCTR2019-002663-10-DE (EUCTR) | 18/02/2021 | 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 Escalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal ... | 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] Muscular Atrophy, Spinal MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mu ... | 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 Trade Name: Spinraza INN or Proposed INN: Nusinersen Other descriptive name: NUSINERSENSODIUM Product N ... | Biogen Idec Research Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 152 | Phase 2;Phase 3 | United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chile;Colombia;Italy;France;Australia;Latvia;Korea, Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Argentina;Brazil;Poland;Germany United States;Saudi Arabia;Taiwan;Estonia;Greece;Spain;Lebanon;Ireland;Russian Federation;Israel;Chi ... | ||
| 10 | NCT04488133 (ClinicalTrials.gov) | January 7, 2021 | 20/7/2020 | A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec A Study of NusinersenAmong Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abepa ... | A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec A Phase 4 Study of Nusinersen(BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onas ... | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Recruiting | 2 Months | 36 Months | All | 60 | Phase 4 | United States;Germany;Israel;Italy;Spain |