3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75
Showing 1 to 10 of 12 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 | NCT05386680 (ClinicalTrials.gov) | January 12, 2023 | 18/5/2022 | Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101Ad ... | Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to 12 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®) Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Effi ... | Spinal Muscular Atrophy | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Recruiting | 2 Years | 12 Years | All | 28 | Phase 3 | Canada;Japan;Netherlands;Spain |
| 2 | EUCTR2021-003474-31-GR (EUCTR) | 03/01/2023 | 15/09/2022 | Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER) Safety and efficacy of Intrathecal OAV101in Pediatric Patients with Spinal Muscular Atrophy (SMA) (S ... | A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are = 2 to < 18 years of age, treatment naive, sitting, and never ambulatory - STEER A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal ... | 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 INN or Proposed INN: Onasemnogene abeparvovec Other descriptive name: previously termed sc.AAV9.CB.SMN and AVXS-101 Trade Name: Okrido 6mg/mL oral solution Product Name: Okrido INN or Proposed INN: PREDNISOLONE SODIUM PHOSPHATE Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE Trade Name: Zolgensma INN or Proposed INN: Onasemnogene abeparvovec Other descriptive name: previously ... | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 125 | Phase 3 | United States;United Arab Emirates;Saudi Arabia;Taiwan;Greece;Thailand;Russian Federation;Colombia;Italy;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Viet Nam;Denmark;South Africa;China United States;United Arab Emirates;Saudi Arabia;Taiwan;Greece;Thailand;Russian Federation;Colombia;I ... | ||
| 3 | NCT05335876 (ClinicalTrials.gov) | December 19, 2022 | 12/4/2022 | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101IT or OAV101IV in Cl ... | Spinal Muscular Atrophy (SMA) | Biological: onasemnogene abeparvovec | Novartis Pharmaceuticals | NULL | Recruiting | N/A | N/A | All | 260 | Phase 3 | Australia;Belgium |
| 4 | NCT05089656 (ClinicalTrials.gov) | January 12, 2022 | 11/10/2021 | Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) Efficacy and Safety of Intrathecal OAV101(AVXS-101) in Pediatric Patients With Type 2 Spinal Muscula ... | A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are = 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal ... | Type 2 Spinal Muscular Atrophy | Genetic: OAV101;Procedure: Sham control | Novartis Pharmaceuticals | NULL | Recruiting | 2 Years | 17 Years | All | 125 | Phase 3 | United States;China;Denmark;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;Vietnam United States;China;Denmark;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailan ... |
| 5 | NCT05073133 (ClinicalTrials.gov) | November 4, 2021 | 20/9/2021 | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Safety and Efficacy of Intravenous OAV101(AVXS-101) in Pediatric Patients With Spinal Muscular Atrop ... | A Phase IV 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 From Latin America and Canada With Spinal Muscular Atrophy (SMA) - OFELIA A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerabili ... | Muscular Atrophy, Spinal | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 24 Months | All | 16 | Phase 4 | Argentina;Brazil |
| 6 | 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 Atrop ... | 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 ... | ||
| 7 | 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 Atrop ... | 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 ... | ||
| 8 | NCT04851873 (ClinicalTrials.gov) | September 8, 2021 | 6/4/2021 | 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 Atrop ... | 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) A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerab ... | Spinal Muscular Atrophy | Genetic: OAV101 | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 17 Years | All | 24 | Phase 3 | United States;Australia;Belgium;Canada;France;Italy;Portugal;Taiwan;United Kingdom;Switzerland |
| 9 | 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 Atrop ... | 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;Belgium;Australia;Germany;United Kingdom;Italy;Switzerland Portugal;France;United States;Taiwan;Canada;Belgium;Australia;Germany;United Kingdom;Italy;Switzerla ... | ||
| 10 | 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 Atrop ... | 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 ... |