3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 217 / Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 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 | 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 |
| 2 | 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 |
| 3 | 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 |
| 4 | 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 |
| 5 | 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 |
| 6 | 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 |
| 7 | 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 |