treatment group:GC101 intrathecal injection

2 trials

Sponsors

The Seventh Medical Center of PLA General Hospital, The seventh medical center of PLA General Hospital

Conditions

type 1 spinal muscular atrophy

Phase 1

Single arm, single center, high-dose clinical study on the safety, tolerance and efficacy of GC101 adeno-associated virus injection in patients with type 1 spinal muscular atrophy

RecruitingChiCTR2200063230

The Seventh Medical Center of PLA General Hospitaltype 1 spinal muscular atrophy

Start: 2022-08-19Updated: 2023-04-17

Phase 2

Multi center, open, single arm, single dose increasing phase I/IIa clinical trial to evaluate the safety, tolerance and initial efficacy of GC101 adeno-associated virus injection in the treatment of type 1 SMA patients

Active, not recruitingChiCTR2200066399

The seventh medical center of PLA General Hospitaltype 1 spinal muscular atrophy

Start: 2022-12-05Updated: 2023-05-15