3. Spinal muscular atrophy
179 clinical trials, 102 drugs (DrugBank: 26 drugs), 52 drug target genes, 78 drug target pathways
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 | EUCTR2019-002611-26-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 | United States;France;Taiwan;Canada;Spain;Belgium;Australia;United Kingdom;Japan;Italy;Korea, Republic of | ||
| 2 | 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 | ||
| 3 | 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 for Spinal Muscular Atrophy Receiving AVXS-101 - not applicable | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 308 | Phase 3 | France;United States;Taiwan;Canada;Belgium;Spain;Australia;Japan;Italy;United Kingdom;Korea, Republic of | |||
| 4 | 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 Pharmaceuticals | NULL | Enrolling by invitation | N/A | N/A | All | 308 | Phase 4 | United States;Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;Korea, Republic of |
| 5 | 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 Pharmaceuticals | PRA Health Sciences | Active, not recruiting | N/A | 6 Months | All | 2 | Phase 3 | Japan;Korea, Republic of;Taiwan |
| 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 | 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] | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan | |||
| 7 | 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] | AveXis, 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 | |||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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] | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | |||
| 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 | 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 | ||
| 12 | NCT03461289 (ClinicalTrials.gov) | August 29, 2018 | 4/3/2018 | Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 | European, 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 | AveXis, Inc. | NULL | Completed | N/A | 6 Months | All | 33 | Phase 3 | Belgium;France;Italy;United Kingdom;Germany;Netherlands;Spain;Sweden |
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;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 |
| 16 | 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 Pharmaceuticals | PRA Health Sciences | Active, not recruiting | N/A | 42 Days | All | 30 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom |
| 17 | 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 Pharmaceuticals | NULL | Suspended | 6 Months | 60 Months | All | 51 | Phase 1 | United States |
| 18 | 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 | AveXis, Inc. | NULL | Completed | N/A | 180 Days | All | 22 | Phase 3 | United States |
| 19 | 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 Pharmaceuticals | NULL | Active, not recruiting | N/A | N/A | All | 13 | United States | |
| 20 | 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 | AveXis, Inc. | NULL | Completed | N/A | 6 Months | All | 15 | Phase 1 | United States |
| 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 |
| 21 | 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 | |||
| 22 | 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 | |||
| 23 | EUCTR2019-002611-26-BE (EUCTR) | 20/01/2020 | 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;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | AveXis, Inc. | NULL | NA | Female: yes Male: yes | 308 | Phase 3 | France;United States;Taiwan;Canada;Spain;Belgium;Australia;Japan;Italy;United Kingdom;Korea, Republic of |