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 34 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 |
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1 | EUCTR2020-005995-37-PT (EUCTR) | 15/10/2021 | 19/07/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Zolgensma2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Cod ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3 | United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy United States;France;Portugal;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
2 | EUCTR2020-005995-37-IT (EUCTR) | 28/09/2021 | 17/08/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE Trade Name: Zolgensma Product Name: OAV101 Product Code: [AVXS-101] INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: Previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Prednisolone Product Name: Prednisolone Product Code: [-] INN or Proposed INN: PREDNISOLONE ... | NOVARTIS PHARMA AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
3 | EUCTR2020-005995-37-BE (EUCTR) | 03/09/2021 | 28/06/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | Trade Name: Zolgensma 2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Zolgensma2 x 1013 vector genomes/mL solution for infusion Product Name: OAV101 Product Cod ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 24 | Phase 3 | Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy Portugal;France;United States;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
4 | EUCTR2020-005995-37-FR (EUCTR) | 25/08/2021 | 21/06/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART) Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atro ... | A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerab ... | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | Trade Name: Zolgensma Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Zolgensma Product Name: OAV101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABE ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerland;Italy Portugal;United States;France;Taiwan;Canada;Spain;Belgium;Australia;Germany;United Kingdom;Switzerla ... | ||
5 | 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 Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abep ... | 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 Ona ... | 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 | EUCTR2020-003492-18-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 Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abep ... | 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 Ona ... | 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 | ||
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 Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abep ... | 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 Ona ... | 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 | 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 Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abep ... | 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 Ona ... | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | NULL | Recruiting | 2 Months | 36 Months | All | 60 | Phase 4 | United States;Germany;Israel;Italy;Spain |
9 | EUCTR2019-002611-26-FR (EUCTR) | 16/09/2020 | 10/03/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 Product Code: AVXS-101 INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | Korea, Republic of;Italy;Japan;United Kingdom;Australia;Belgium;Spain;Canada;Taiwan;France;United States Korea, Republic of;Italy;Japan;United Kingdom;Australia;Belgium;Spain;Canada;Taiwan;France;United St ... | ||
10 | EUCTR2019-002611-26-IT (EUCTR) | 27/04/2020 | 03/08/2021 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - not applicable A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving ... | 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] - Nervous System Diseases [C10] Spinal Muscular Atrophy MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal mus ... | Product Name: AVXS-101 Product Code: [AVXS-101] INN or Proposed INN: ONASEMNOGENE ABEPARVOVEC | AVEXIS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | France;United States;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of France;United States;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republi ... |