Informed consent considerations
|
| • General knowledge of the rationale and approach |
| • General knowledge about AAV vectors |
|
| • Short-term safety: infusion reactions, transaminase, and CPK elevations |
| • Likelihood/plan for immune modulation and glucocorticoid side effects |
| • Long-term safety considerations: unclear implications on liver health and theoretical risk of genotoxicity |
|
| • Expression profile and durability |
| • Range of phenotypic improvement at 1 year vs longitudinally; frequency of therapeutic response vs nonresponse |
| • Development of anti-AAV neutralizing antibodies and anticipated inability to receive future AAV therapies unless there are scientific developments to overcome anti-AAV neutralizing antibodies. |
|
| • Infusion day |
| • Anticipated short-term follow-up and monitoring; baseline liver ultrasound and blood tests |
| • Anticipated long-term follow-up and monitoring, including recommendations around long-term liver health and minimizing potential liver toxicities (eg, alcohol use, nonalcoholic fatty liver disease); planned long-term liver health evaluations and durability of expression |
| • Vector shedding precautions for clinic visits and close contacts (see supplemental Table 1) as well as contraindications for blood and organ donation after valoctocogene roxaparvovec |
|
| • General knowledge of the rationale and approach |
| • General knowledge about AAV vectors |
|
| • Short-term safety: infusion reactions, transaminase, and CPK elevations |
| • Likelihood/plan for immune modulation and glucocorticoid side effects |
| • Long-term safety considerations: unclear implications on liver health and theoretical risk of genotoxicity |
|
| • Expression profile and durability |
| • Range of phenotypic improvement at 1 year vs longitudinally; frequency of therapeutic response vs nonresponse |
| • Development of anti-AAV neutralizing antibodies and anticipated inability to receive future AAV therapies unless there are scientific developments to overcome anti-AAV neutralizing antibodies. |
|
| • Infusion day |
| • Anticipated short-term follow-up and monitoring; baseline liver ultrasound and blood tests |
| • Anticipated long-term follow-up and monitoring, including recommendations around long-term liver health and minimizing potential liver toxicities (eg, alcohol use, nonalcoholic fatty liver disease); planned long-term liver health evaluations and durability of expression |
| • Vector shedding precautions for clinic visits and close contacts (see supplemental Table 1) as well as contraindications for blood and organ donation after valoctocogene roxaparvovec |