[Translation] A multi-center, open-label, single-arm, single-dose ascending phase I/IIa clinical trial to evaluate the safety, tolerability and preliminary efficacy of GC101 adeno-associated virus injection in the treatment of patients with type 1 SMA.
I期(耐受性试验)
评价GC101腺相关病毒注射液单次给药治疗1型SMA患者的安全性和耐受性。IIa期(初步疗效探索)
主要目的:
评价GC101腺相关病毒注射液单次给药治疗1型SMA患者的初步疗效。
次要目的:
评价GC101腺相关病毒注射液单次给药治疗1型SMA患者的安全性、AAV病毒载量及免疫原性。
[Translation] Phase I (Tolerance Trial)
To evaluate the safety and tolerability of a single administration of GC101 adeno-associated virus injection in the treatment of patients with type 1 SMA. Phase IIa (preliminary efficacy exploration)
main purpose:
To evaluate the preliminary efficacy of a single administration of GC101 adeno-associated virus injection in the treatment of patients with type 1 SMA.
Secondary purpose:
To evaluate the safety, AAV viral load and immunogenicity of a single administration of GC101 adeno-associated virus injection in the treatment of patients with type 1 SMA.