Table 1.
Adverse events during treatment of a patient with Canavan disease with recombinant adeno-associated virus serotype 9-mediated human aspartoacylase (rAAV9-hASPA) gene transfer therapy
| Event | Patient identifier | Date enrolled in study | Date of AE | Serious (Y/N) | Unanticipated (Y/N) | Related to study procedure (Y/N/cannot R/O) | Date reported to IRBa | Outcome |
|---|---|---|---|---|---|---|---|---|
| Red rash around eyes and minor swelling – reaction to first rituximab infusion | C-001 | 4/3/17 | 4/3/17 | N | N | Y (related to rituximab infusion) | CR-2017 | resolved |
| Emesis | C-001 | 4/3/17 | 4/4/17 | N | Y | N | CR-2017 | resolved |
| Bug bite on right foot | C-001 | 4/3/17 | 4/11/17 | N | Y | N | CR-2017 | resolved |
| Emesis following first dose of prednisolone oral solution; changed dosing plan to resolve AE | C-001 | 4/3/17 | 4/27/17 | N | Y | Y (related to prednisolone administration) | CR-2017 | resolved |
| Maculopapular rash on trunk, back, head, and thigh | C-001 | 4/3/17 | 5/24/17 | N | Y | N | CR-2017 | resolved |
| CSF leak and inflammation of surgical site | C-001 | 4/3/17 | 5/26/17 | Y (SAE) | Y | Y | 5/26/17 | resolved |
| Rash (apparently drug related [linezolid]) during hospitalization for SAE | C-001 | 4/3/17 | 5/30/17 | N | Y | N | CR-2017 | resolved |
| Bug bite on left lateral neck area | C-001 | 4/3/17 | 6/5/17 | N | Y | N | CR-2017 | resolved |
| Gastric reflux | C-001 | 4/3/17 | 7/12/17 | N | Y | N | CR-2017 | ongoing |
| Febrile illness | C-001 | 4/3/17 | 9/12/17 | N | Y | N | CR-2017 | resolved |
| Vomiting | C-001 | 4/3/17 | 9/12/17 | N | Y | N | CR-2017 | resolved |
| Acute viral syndrome (coronavirus) | C-001 | 4/3/17 | 9/13/17 | N | Y | N | CR-2017 | resolved |
| Constipation | C-001 | 4/3/17 | 10/2/17 | N | Y | N | CR-2017 | resolved |
| Gastrointestinal tube removal (accidentally removed by mother) | C-001 | 4/3/17 | 10/11/17 | N | Y | N | CR-2017 | resolved |
| Fever | C-001 | 4/3/17 | 10/31/17 | N | Y | N | CR-2017 | resolved |
| IgG infusion inpatient | C-001 | 4/3/17 | 11/3/17 | N | N | N | CR-2017 | resolved |
| Hypogammaglobulinemia | C-001 | 4/3/17 | 11/3/17 | N | Y | N | CR-2018 | resolved |
| Fever | C-001 | 4/3/17 | 11/3/17 | N | Y | N | CR-2018 | resolved |
| Hypogammaglobulinemia | C-001 | 4/3/17 | 11/9/17 | Y | Y | cannot R/O | 11/9/17 | resolved |
| Posterior humeral fracture | C-001 | 4/3/17 | 6/29/18 | N | Y | N | CR-2018 | ongoing |
AE, adverse event; CR, continuing review; CSF, cerebrospinal fluid; IRB, institutional review board; R/O, rule out; SAE, serious AE; Y, yes; N, no.
a
Date reported or reported with continuing review.