| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05335876 | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials | PHASE3 | RECRUITING | 175 | — | — | Dec 19, 2022 | Feb 27, 2031 | Jan 20, 2026 | 32 | United States, Australia +17 |
An SAE is defined as any adverse event \[appearance of (or worsening of any pre-existing)\] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * fatal * life-threatening * results in persistent or significant disability/incapacity * constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect * requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition * is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.
| Arm | Type | Description |
|---|---|---|
| Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec | EXPERIMENTAL | Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302) |
| Name | Type | Description |
|---|---|---|
| onasemnogene abeparvovec | BIOLOGICAL | Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight. |
Inclusion Criteria: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. Exclusion Criteria: There are no exclusion criteria for this study.