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
| 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 | 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) | 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 | 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] | 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 | 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 | ||
| 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) | 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 | 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] | 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 | 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 | ||
| 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) | 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 | 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] | 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 | 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 | ||
| 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) | 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 | 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] | 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 | 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 | ||
| 5 | 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 | 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 | ||
| 6 | 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 | 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] | 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 | ||
| 7 | EUCTR2019-002611-26-GB (EUCTR) | 30/03/2020 | 31/12/2019 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | 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 | United States;France;Taiwan;Canada;Spain;Belgium;Australia;Japan;Italy;United Kingdom;Korea, Republic of | ||
| 8 | EUCTR2019-002611-26-ES (EUCTR) | 28/02/2020 | 14/02/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | 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 | Not Recruiting | Female: yes Male: yes | 308 | Phase 3 | Italy;Korea, Republic of;United States;France;Taiwan;Canada;Belgium;Japan;United Kingdom;Australia;Spain | ||
| 9 | NCT04042025 (ClinicalTrials.gov) | February 10, 2020 | 31/7/2019 | Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi | A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 | Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMA | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Enrolling by invitation | N/A | N/A | All | 308 | Phase 4 | United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;Spain |
| 10 | NCT03837184 (ClinicalTrials.gov) | May 15, 2019 | 8/2/2019 | Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | Spinal Muscular Atrophy Type I | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | PRA Health Sciences | Completed | N/A | 6 Months | All | 2 | Phase 3 | Japan;Korea, Republic of;Taiwan |
| 11 | EUCTR2017-000266-29-NL (EUCTR) | 25/03/2019 | 05/06/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 12 | EUCTR2017-004087-35-NL (EUCTR) | 28/02/2019 | 09/07/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan | ||
| 13 | EUCTR2017-004087-35-BE (EUCTR) | 31/01/2019 | 16/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 14 | EUCTR2017-000266-29-BE (EUCTR) | 31/01/2019 | 23/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;United Kingdom;Italy;Sweden | ||
| 15 | EUCTR2017-004087-35-IT (EUCTR) | 24/12/2018 | 23/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 16 | EUCTR2017-004087-35-ES (EUCTR) | 21/12/2018 | 20/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Australia;Germany;Netherlands;Japan;Belgium;Canada;Italy;United Kingdom;Israel;Korea, Democratic People's Republic of;Spain;Taiwan;United States | ||
| 17 | EUCTR2017-000266-29-ES (EUCTR) | 18/12/2018 | 28/05/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 18 | EUCTR2017-000266-29-DE (EUCTR) | 05/11/2018 | 05/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 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] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 19 | EUCTR2017-004087-35-GB (EUCTR) | 24/08/2018 | 08/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | Novartis Gene Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||
| 20 | NCT03461289 (ClinicalTrials.gov) | August 16, 2018 | 4/3/2018 | Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | SMA | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Completed | N/A | 6 Months | All | 33 | Phase 3 | Belgium;France;Italy;United Kingdom;Germany;Netherlands;Spain;Sweden |
| 21 | EUCTR2017-000266-29-GB (EUCTR) | 12/06/2018 | 12/02/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 3 | France;Spain;Belgium;Netherlands;Italy;United Kingdom;Sweden | ||
| 22 | EUCTR2017-000266-29-IT (EUCTR) | 21/05/2018 | 09/06/2020 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion - N/A | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 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] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 | AVEXIS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 23 | NCT03505099 (ClinicalTrials.gov) | April 10, 2018 | 13/4/2018 | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 | Spinal Muscular Atrophy | Biological: onasemnogene abeparvovec-xioi | Novartis Gene Therapies | PRA Health Sciences | Completed | N/A | 42 Days | All | 30 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom |
| 24 | NCT03381729 (ClinicalTrials.gov) | December 14, 2017 | 13/12/2017 | Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy | Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Completed | 6 Months | 60 Months | All | 32 | Phase 1 | United States |
| 25 | NCT03306277 (ClinicalTrials.gov) | October 24, 2017 | 2/10/2017 | Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | SMA - Spinal Muscular Atrophy;Gene Therapy | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Completed | N/A | 180 Days | All | 22 | Phase 3 | United States |
| 26 | NCT03421977 (ClinicalTrials.gov) | September 21, 2017 | 30/1/2018 | Long-Term Follow-up Study for Patients From AVXS-101-CL-101 | A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101 | Spinal Muscular Atrophy 1 | Biological: Onasemnogene Abeparvovec-xioi | Novartis Gene Therapies | NULL | Active, not recruiting | N/A | N/A | All | 13 | United States | |
| 27 | NCT02122952 (ClinicalTrials.gov) | May 5, 2014 | 23/4/2014 | Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101 | Spinal Muscular Atrophy 1 | Biological: AVXS-101 | Novartis Gene Therapies | NULL | Completed | N/A | 6 Months | All | 15 | Phase 1 | United States |
| 28 | EUCTR2017-004087-35-DE (EUCTR) | 06/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | NA | Female: yes Male: yes | 27 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | |||
| 29 | EUCTR2017-000266-29-SE (EUCTR) | 12/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | |||
| 30 | EUCTR2017-000266-29-FR (EUCTR) | 05/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 | Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | |||
| 31 | EUCTR2019-002611-26-BE (EUCTR) | 20/01/2020 | not applicable | A Long-term Follow-up Study of Patients in the Clinical Trials forSpinal Muscular Atrophy Receiving AVXS-101 - not applicable | 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 | Novartis Gene Therapies, Inc. | NULL | NA | Female: yes Male: yes | 308 | Phase 3 | Japan;Italy;United Kingdom;Australia;Belgium;Spain;Canada;Taiwan;United States;France | |||
| 32 | EUCTR2021-003474-31-DK (EUCTR) | 02/11/2021 | Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER) | 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 | 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] | Trade Name: Zolgensma Product Name: OAV101 Product Code: OAV101 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 | Novartis Pharma AG | NULL | NA | Female: yes Male: yes | 125 | Phase 3 | United States;United Arab Emirates;Saudi Arabia;Taiwan;Thailand;Russian Federation;Colombia;Italy;Vietnam;India;Egypt;Mexico;Brazil;Malaysia;Singapore;Denmark;South Africa;China |